White Paper

Pharma R&D
Annual Review 2024
Look back at key pipeline trends from the last
12 months so you can forecast success in 2024
 Contents

Welcome 03

Introduction: Total Pipeline Size 05

Pleasant outlook for pharma as things continue to heat up 

Developing Systems: The 2024 Pipeline by Phase 08

Clinical development advances still face a stiff headwind 

Top Companies 11
Some enjoy a moment in the sun, but for others the future looks more hazy 

Therapies and Diseases 19

Cancer still taking R&D by storm 

Regional Variations 31
Where the outlook seems to be brightest

Mechanisms and Targets 54

Immuno-oncology makes hay while the sun shines

Types of Pipeline Drugs 59

Is the continued move into biotech starting to meet a frosty reception?

What’s the Forecast for Pharma? 62

Plenty of rays of sunshine should hold off any storm clouds on the horizon 

About the Author  65

 Welcome

Welcome to Pharmaprojects’ 2024 review of trends in

pharmaceutical R&D

Welcome to Pharmaprojects’ 2024 review Since our early cave-dwelling days, the activities
of trends in pharmaceutical R&D. For over of humans have been somewhat governed
30 years now, I’ve been taking an annual by the vagaries of the weather. We needed to
look at the evolution of pharma R&D, and in know when conditions were best for hunting,
this article I’ll take the temperature of the and whether our crops would flourish or fail. The
industry at the start of 2024. We’ll assess earliest attempts to predict the weather were
industry trends by examining the pipeline by largely unscientific and based on prophecy,
company, therapeutic area, disease, target, astrology, or prayers to invisible weather gods.
and drug type, using data primarily from As with so many things, the ancient Greeks were
Pharmaprojects, part of the Citeline suite the first to turn superstition into science, and
of products, which has been tracking global in fact the term “meteorology” comes from the
drug development since 1980. This report will Greek meaning “the study of things high in the
be followed up by our annual supplement air.” The acknowledged father of the science
reviewing the New Active Substance (NAS) is Aristotle, who published a treatise on the
launches for the year just passed. But here, subject as early as 350 BC. Of course, accurate
we’ll examine how the pharma R&D climate is instruments to measure weather-related
changing, look at where the pressure is rising phenomena only really came along with the
and falling, and try to determine where the Enlightenment, but weather forecasts were still
outlook is sunny and where storm clouds are notoriously unreliable, especially in countries like
gathering. Hopefully, it will help you to take the UK where I am based, as little as 50 years
the industry’s temperature and forecast ago. It was only with the growth of computing
its outlook. power, and with it, computer modeling
algorithms, that forecasters were able to provide
Regular readers of this report (which has been predictions with any measure of certainty. The
running since 1993, so is presented here in its key, as always, is data — and lots of it. My hope
32nd edition) will know that, in recent years, I’ve is that this report will fulfill the same function:
threaded a different theme through each edition, it will provide enough information for you to
to highlight points, to draw analogies, and to determine which parts of the industry are
add a little character into what could otherwise likely to be basking in unbroken sunshine,
be a rather lengthy narrative through a parade and which are preparing for a period of
of statistics, charts, and tables — duller than a unsettled conditions.
December day in Doncaster. Themes selected so
far have included astronomy, movies, the natural
world, music, food and drink, science fiction,
travel, and, last year, literature. This year, as you
may have guessed, I decided to hang my (rain)
hat on something that affects us all every day:
the weather, and the allied most important issue
of our times, climate change.

3 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

While our primary focus is naturally on the The pharma industry, as we shall see, has been
short term; i.e., whether we need to put on an heating up for some time now, despite the mini-
extra jumper or to bring our umbrellas, the ice age which COVID presented. Within that
long-term trends are what exercise the minds overall trend, though, there are ever-shifting
of both climate scientists and pharma industry patterns, more complex than weather pressure
followers. In the past few decades, the topic of charts. Drug development has its own areas of
the weather has grown from being a parochial high and low pressure, its own fronts; in some
consideration to something threatening our very places, it is experiencing headwinds, in others,
existence. But, in fact, the idea that our impact the air is calm; and some therapeutics are most
on the planet is changing it irrevocably has been definitely hotter than others. In this report, we
around longer than you might think. It was at will present to you our meteorological map of
the end of the 19th century when scientists first pharmaceutical R&D as it is now, and compare
predicted that changes in atmospheric carbon that to our charts from a year ago, to get the
dioxide levels could substantially alter a planet’s best view of where things are warming up and
surface temperature through the greenhouse where a deep depression may be setting in.
effect, and just prior to World War II when Guy We will also occasionally look further back to
Callendar connected carbon dioxide increases define longer-term trends; after all, you don’t
in Earth’s own atmosphere to global warming. always just need to know whether it will rain
Particularly since the turn of the millennium, the tomorrow, you need to know whether to pack
evidence seems irrefutable that, unless action is your sunscreen or your Wellington boots for your
taken and taken fast, we are building towards a forthcoming holiday. In that sense, our pharma
threat to our continued existence. The pharma industry review will try to provide both a weather
industry has of course weathered a more acute forecast and an insight into broader changes in
existential threat recently — the COVID-19 the climate.
pandemic, which it helped to deliver us from —
and has emerged to enjoy a moment in the sun. I hope this introduction has broken the ice and
But both climate change and the coronavirus set you up to ride along the trade winds of data
crisis are evidence of the fact that we need to we have on today’s pharma industry. So, take
identify — and act on — emerging trends very a rain check from whatever you’re doing, find a
quickly. We have the data; it’s what we do with place in the sun, and read on to find out where
it that counts. the industry is basking in unbroken sunshine,
and where the outlook is more overcast. We
Of course, changes in climate patterns predate won’t try to boil the ocean, but we will provide
humans and the transformational effect of the a clear view to the horizon so that you can see
industrial revolution. Within the past million which way the wind is blowing in drug R&D.
years, there have been at least eight cycles of We’ve cooked up a veritable storm of statistics
ice ages and warmer periods, along with shorter and will help you to navigate any choppy waters
cycles such as the Little Ice Age in the North they stir up. Hopefully, after reading this report,
Atlantic between the 16th and 19th centuries, understanding drug R&D trends will be a breeze.
which famously led to London’s River Thames
freezing over and the Victorian “Frost Fairs” held
there. While climate is bound to have inherent
variability, this change has been variously
attributed to cyclical lows in solar radiation,
heightened volcanic activity, changes in ocean
circulation, and variations in Earth’s orbit and
axial tilt, so the human race hasn’t always
historically been behind climate change. It’s
clear now, however, that our effect extends far
beyond such natural cycles, and is potentially
sending our planet towards irreversible disaster,
at least as far as life is concerned. It’s theorized
that other planets such as Mars have already
been through this process, and that the dead
planet we see today is potentially a spooky view
into our own future.

4 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 Introduction: Total Pipeline Size
Pleasant outlook for pharma as things continue to heat up
As a foreword to this year’s almanac, let’s We’ve also consistently seen increases in the
define the climate system in which our number of drugs in development year-on-
changing weather patterns analyses will year — is “pharma warming” still occurring in
take place, by looking at the total number 2024? Well, Figure 1 confirms that the overall
of drugs currently in the R&D pipeline. All pipeline size has again hit a new high, and
the analyses in this report will be focusing the rate of expansion is on the up again.
on this set of drugs, so it’s worth starting 2024’s headline figure is 22,825 drugs, an
off with a definition of what exactly we expansion of 7.20% from last year’s figure
mean by the term “pipeline.” Here, we of 21,292. The rate of growth represents an
are counting all drugs in development by increase from the previous year’s 5.89%,
pharmaceutical companies, from those at which places the 2024 rate nicely in the
the preclinical stage, through the various middle of that rate and the year before
stages of clinical testing and regulatory (8.22%). It also places this year’s rate pretty
approval, and up to and including launch. much dead on the average of the past five
Launched drugs are still counted, but years, which is 7.14%. So it would seem that
only if they are still in development for this year is at least right on trend.
additional indications or markets. Drugs
whose development has been terminated,
or is complete, are not included. All data
were collected on Jan. 2, 2024.

Are things still heating up in pharma R&D,

as well as the climate? Records for extremes
of high temperature have repeatedly been
broken in recent years. In the UK, the
summer of 2022 saw the mercury peak
at 40.3°C (104.5°F), while Australia hit
its own record just a few months earlier,
hitting a blistering 50.7°C (123.3°F).
Perhaps surprisingly, the highest verifiable
temperature humans have recorded globally
thus far dates back over a century to
July 1913, when the appropriately named
Furnace Creek, California, recorded a face-
melting 56.7°C (134°F). Globally, all 10 of
the warmest years on record have occurred
post-2000, with 2023 recently confirmed
to be the warmest yet. It’s now well
documented that the overall trend for global
temperatures is climbing at an alarmingly
accelerating rate, with average global
temperatures already having increased by
about 1°C (1.7°F) since 1880, and projected
to warm by 1.5°C (2.7°F) by 2050, and
potentially by 2–4°C (3.6–7.2°F) by 2100.

5 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 Figure 1: Total R&D pipeline size by year, 2001­­—­24
25,000

22,825

21,292

20,109
20,000
18,582
17,737

16,181

15,267
14,872
15,000
DRUG COUNT

13,718

12,300

11,307
10,452 10,479
9,605 9,737 9,713
10,000
9,217

7,737
7,360 7,406
6,994
6,416
5,995 6,198

5,000

0
20
20

20

20

20

20

20

20

20

20

20

20

20

20

20

20

20

20

20

20

20

20

20

20
05
01

02

03

04

06

07

08

09

10

11

12

13

14

15

16

17

18

19

20

21

22

23

24
YEAR
Source: Pharmaprojects®, January 2024

In real terms, the size of the pipeline has thus the big hitters point to a slightly broader spread
increased by 1,533 drugs, representing a bigger of candidates by therapeutic area joining drug
increase than the 1,183 seen during 2022–23, companies’ portfolios last year; as with weather,
but almost exactly the same as the 1,527 uptick a mixed, balanced picture is healthier, rather
reported over 2021–22. But this doesn’t tell the than too much of one feature causing a flood
whole story. During 2023, a total of 5,428 drugs or a drought somewhere else.
were added to the Pharmaprojects database,
considerably more than the 5,082 added during
the previous year. So how come the total
pipeline size only grew by 1,533? The answer is
that a total of 3,895 candidates left the active
pipeline during the same period, a figure which
again almost exactly matches the one from the
previous year. This indicates that, once again,
there has been considerable turnover within
the pipeline in the course of a single year,
but the amount of change has at least
remained constant.

Let’s look a little deeper into the types of drugs

that entered the pipeline during 2023. By far
the largest chunk of drugs fall into the oncology
bucket, which provided 38.0% of all new
candidates — interestingly, a little down from
2022’s 40.7%. Neurologicals came in second,
with 12.7%, also down from 13.5% the previous
year. Taken together, these slight declines for

6 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

There might be some subtle shifts in wind continuously increasing drug R&D pipeline sizes
direction this year, which are exemplified by are not wholly a good thing. As we’ll see shortly,
a look at which companies added the most the vast majority of the drugs in this number
new drugs to the pipeline. This year, Pfizer are not yet — and, in the majority of cases, will
tops the list with 32 new candidates, pushing never be — launched. Thus, they are a cost to the
Jiangsu Hengrui Pharmaceuticals into second industry, and a risk. Might the bloated pipeline
place with 30. The leading Chinese firm topped reach a tipping point where it no longer becomes
the equivalent ranking last year, somewhat sustainable, in the same way that global warming
eclipsing the more traditional Western big might? Just as switching to renewable energy
pharmas. However, in terms of the location of sources might save the planet, the way in which
development of 2023’s new intake of drugs, the industry renews itself and keeps progressing
there seems further evidence of China’s is by successfully launching new drugs, which
continued advance. The US is still the pre-
earn revenue. We are still gathering and finessing
eminent territory this year, with 1,856 new
data on NAS launches for 2023 as I write, but
candidates (up from 1,840), but China is closing
early indications are it’s likely that, while it was
the gap, with 1,627 neophyte drugs, up from
a less successful year than 2021, which produced
1,457 in 2022. The varying strengths of Eastern
a record haul on 97 NAS launches, it surpassed
and Western trade winds is a subject we’ll be
returning to later in this report. 2022’s 74 to become the second best year
ever. Be sure to check out the forthcoming NAS
However, just as ever-warming temperatures Supplement for the final tally, and confirmation
might lead to nicer summers but have some of whether the pharma industry is continuing
potential downsides (droughts, forest fires, to reap a sizable harvest, or whether drought
the total extinction of the human race, etc.), conditions might be setting in.

7 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 Developing Systems: The 2024 Pipeline by Phase
Clinical development advances still face a stiff headwind
Weather, like new drugs, doesn’t just Drug development of course follows an
appear fully formed — it develops. And, orderly pattern, as candidates move from
like a healthy human, it relies on many preclinical development, through the various
interrelated pieces working together to phases of clinical trials, into registration, and
function. The large-scale structure of the (hopefully) finally onto the market. But just
Earth’s atmospheric circulation systems, as the sun rising into a cloudless sky doesn’t
like the drug development process, mean it will be fine all day, it might stay
remains fairly constant, but smaller-scale clear, or conditions might deteriorate; a drug
systems such as mid-latitude depressions can have a clear run to market but, more
or tropical convective cells occur often than not, it will get lost in the fog and,
chaotically and are impossible to predict before you know it, it’s rain stopped play.
accurately beyond around 10 days ahead. Let’s take a look at how the pipeline breaks
down by phase of development, and try to
Chaos theory plays a large part in climate determine whether the outlook is improving.
modeling algorithms and unpredictability
is innate, in much the same way as we
cannot predict accurately whether a drug in
preclinical development will make it through
the development process. There are as
many variables along the path to produce a
successfully marketed drug versus a failed
one as there are between a weather system
developing into a hurricane versus a spell
of light drizzle. In the pursuit of maximizing
success in both fields, increases in computer
power to better model such variables are an
important step forward.

8 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 Figure 2: Pipeline by development phase, 2024 vs. 2023
14,000

12,485
11,835
12,000

10,000

8,000
DRUG COUNT

6,000

3,703
4,000
3,263 3,374
3,131

2,000 1,425
1,229 1,248 1,297

254 270 135 174 63 120 85 85

0
Preclin Phase I Phase II Phase III Pre-reg Registered Launched Suspended N/A

2023 2024

(N/A = not applicable and is applied to companion diagnostics prelaunch)

Source: Pharmaprojects®, January 2024

Figure 2 breaks down the 2024 pipeline by the Phase I then progress serenely to Phase II.
drugs’ current global statuses. Global status There is considerable attrition between the two
is the most advanced stage of development phases, but as Phase II development generally
a drug has reached in any country, for any takes much longer, drugs pile up at the Phase II
disease, and by any company, so each drug is stage, so that at any one time, there are more
counted only once here. You can immediately drugs in Phase I than there are in Phase II.
see that a considerable amount of the increase
in pipeline size growth occurred by adding
drugs at the preclinical stage, which posted a
total up by 650. However, in percentage terms,
this is a 5.5% increase; lower than the overall
rate of increase in the pipeline as a whole, but
higher than 2023’s equivalent number of 4.3%.
In percentage terms, it is the number of drugs
in Phase I which has increased the most, rising
by an impressive 13.5%, accelerating from the
10.7% increase reported last year. Rises at Phase
II also showed a positive trend, with the number
of drugs at that phase up by 7.8%, compared to
7.2% last year. Note that while the numbers in
Phase I being higher than those in Phase II may
at first seem counterintuitive, this is a function
of these data being a snapshot in time. The fact
that there are roughly the same numbers of
drugs in Phase II as there are in Phase I in no
way means that virtually all drugs undergoing

9 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 But there is cause for concern when we move to the 2017–21 period failed to keep pace with
the Phase III number, which seems in contrast equivalent rises seen in Phase I and II, indicating
somewhat becalmed. It’s wiser here to take worsening attrition. But the sun had appeared
a deeper dive into our historical records, as to be breaking through the clouds here during
Figure 3 pulls out the numbers of drugs in the 2022 and 2023, with Phase III numbers on the up
clinical development stages across a longer time again. This year’s data indicate darkening skies
period, going back to 2007. You can see here again, which could be a cause for concern for
how the increases in Phase III numbers during the industry.

Figure 3: Clinical phase trends, 2007–24

4,000
3,703
2007
3,500 3,374 2008
3,263
3,131 2009

3,000 2010
2011
2012
2,500
DRUG COUNT

2013
2014
2,000
2015
2016
1,500 2017
1,248
1,229
2018
1,000 2019
2020

500 2021
2022
2023
0
Phase I Phase II Phase III 2024
CLINICAL STAGE OF DEVELOPMENT
Source: Pharmaprojects®, January 2024

It’s undoubtedly true that if you are spending

more on pushing more drugs through Phase
I and Phase II trials, you want more drugs
to make it into Phase III. However, there is
a caveat to this. Phase III trials are by some
measure the most expensive part of drug
development, so it’s only a good thing to
have more drugs in Phase III if the industry is
consequently able to bring more drugs to the
market. Indeed, data from industry analysts
such as Deloitte suggest that the year-on-year
cost of developing drugs continues to rise,
while return on investment is falling. This, like
the ongoing rise in global temperatures, clearly
cannot be sustainable.

10 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 Top Companies
Some enjoy a moment in the sun, but
for others the future looks more hazy

Which companies in 2024 are enjoying extensive sunny periods, and which are undergoing
a sudden cold snap? In this section, we’ll take a look at the leading companies enjoying fair
weather, plus the industry as a whole, which is composed not just of big fluffy clouds, but many
tiny raindrops. Like the atmosphere, the pharma industry is composed of a number of distinct
layers, and we’ll be peering through the fog at each.

Table 1: Top 25 pharma companies by size of pipeline

POSITION
NO. OF DRUGS IN NO. OF ORIGINATED
2024 COMPANY TREND
PIPELINE 2024 (2023) DRUGS 2024 (2023)
(2023)
1 (1) Roche 218 (194) 125 ↔
2 (5) Pfizer 205 (171) 127 ↑
3 (7) AstraZeneca 166 (155) 103 ↔
4 (10) Eli Lilly 159 (135) 90 ↑
5 (4) Bristol-Myers Squibb 158 (175) 90 ↓
6 (2) Novartis 154 (191) 96 ↓
7 (6) Johnson & Johnson 150 (156) 81 ↓
Jiangsu Hengrui ↑↑
8 (13) 147 (106) 138
Pharmaceuticals
9 (8) Merck & Co. 145 (151) 69 ↓
10 (9) Sanofi 142 (145) 79 ↓
11 (11) GlaxoSmithKline 138 (123) 70 ↔
12 (3) Takeda 130 (178) 50 ↓
13 (12) AbbVie 111 (122) 46 ↓
14 (14) Boehringer Ingelheim 111 (99) 80 ↔
15 (27) Sino Biopharmaceutical 103 (60) 84 ↑↑
16 (15) Bayer 91 (93) 61 ↔
Shanghai Fosun ↑
17 (24) 90 (64) 62
Pharmaceutical (Group)
18 (16) Gilead Sciences 86 (86) 46 ↔
19 (17) Otsuka Holdings 85 (85) 45 ↔
20 (20) Eisai 81 (74) 46 ↔
21 (18) Amgen 79 (79) 47 ↔
22 (26) Astellas Pharma 78 (63) 40 ↑
23 (19) Novo Nordisk 76 (77) 53 ↔
24 (23) CSPC Pharmaceutical 73 (68) 60 ↔
25 (21) Regeneron 71 (73) 53 ↔
Source: Pharmaprojects®, January 2024

11 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

The upper level of the atmosphere is the Most manufactured satellites orbit in the
exosphere. At this rarified level, there’s no exosphere, and some of the larger and more
weather as such, but the lower parts can reflective ones are sometimes visible from
host that most spectacular of atmospheric the Earth’s surface as they catch the sunlight.
phenomena, the aurora. Shimmering on the Undoubtedly, one of the brightest “stars” in
upper echelons of the pharma industry are the the pharma exosphere this year has to be
Top 25 companies by pipeline size, which are Jiangsu Hengrui Pharmaceuticals. The company
listed in Table 1. Shining brightest for the second becomes the first ever Chinese firm to make
year in a row is the Swiss-based multinational it into the top 10 in terms of pipeline size,
Roche, which deposed its Basel-based neighbor recording a 38.7% increase in its pipeline to burst
Novartis last year. The latter slips notably this into the upper firmament at number 8. It also
year with a pipeline which has considerably has by some measure the biggest proportion
slimmed down. Taking the runner-up position of “homemade” drugs, with almost 94% of its
this year is Pfizer, whose portfolio is about 20% drugs being self-originated. In fact, by this latter
bigger than it was this time last year, a fact measure it is at number 1, having more drugs
which can largely be attributed to its completion in the pipeline which it discovered itself than
of its acquisition of Seagen (the former Seattle any other company, period. This is a landmark
Genetics) in December 2023. In a year which moment for the Chinese pharmaceutical
saw an unusual degree of turbulence in the industry, which, 20 years ago, had little to no
upper atmosphere, it’s just one of the significant homegrown pharma R&D focused on original
changes in a top 10 which, in recent years, has compounds.
exhibited a high level of stability. AstraZeneca
hops up to number 3, having itself acquired a The next level of the atmosphere is the
couple of small firms, Neogene Therapeutics thermosphere, another rarified layer devoid of
and CinCor Pharma, while Eli Lilly shoots up clouds and water vapor, but where you will find
from number 10 to number 4, having taken over the International Space Station. Our next level,
Versanis Bio, DICE Therapeutics, and Sigilon the companies numbering 11 to 25 by pipeline
Therapeutics during a particularly acquisitive size, also has some interesting features in it, and
year (it also subsumed POINT Biopharma, again it’s China that is lighting up the sky here.
although that completed too late in the year Sino Biopharmaceutical leapfrogs its compatriot
to be included in the Jan. 2, 2024 data cut). Shanghai Fosun to become the country’s
Away from this year’s top four, though, the second biggest R&D powerhouse, bursting into
rest of last year’s top 10 all report pipelines of the upper echelons this year with a massive
smaller size this year. While Bristol Myers Squibb 72% increase in its pipeline size, all via organic
and Johnson & Johnson steered clear of M&A growth. Similarly, Shanghai Fosun also reported
activity during 2023, Novartis, Merck & Co., decent growth with a 41% bigger R&D portfolio.
Sanofi, and most dramatically Takeda report These were by far the most significant changes
smaller pipelines despite having acquired some in companies at this level; most other firms had
(admittedly small) companies last year. pipelines of pretty similar sizes to last year.

With the possible exception of Pfizer’s Seagen

acquisition, our Top 25 is not really affected
significantly by M&A activity through 2023.
Overall, M&A activity across the industry
appears to have slightly risen from last year’s
nadir, with 88 mergers and acquisitions reported
by Pharmaprojects to have closed during 2023,
slightly up from the 81 noted in 2022. There has
now been a long period of calm weather after
the stormy period of the late 1990s and early
2000s when tempestuous megamergers of
pharmaceutical giants were commonplace.
Like storms, these big mergers may have
cleared the air, but many ultimately may
have inflicted significant damage.

12 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

Most of the Earth’s weather occurs in the lowest minnows comprise a large part of the industry.
level of the atmosphere, the troposphere. Almost Figure 4 shows that these minute, emerging
all clouds that are generated by weather are companies produce just over 18% of the drugs
found here, with the exception of cumulonimbus in development, a slightly bigger slice than last
thunder clouds, whose tops can rise into the year, but roughly the same percentage as 10
lowest parts of the neighboring stratosphere. years ago. This is a larger tranche than either the
It’s perhaps surprising how much activity occurs
Top 10 or the Top 25 companies. It’s interesting
at the lowest level of the pharma industry too
to note, however, that this year seems to have
­­— at those companies that are only developing
one or two drugs. This year, we are reporting 931 witnessed a small reversal to the long-term
companies with only two drugs in their pipelines trend whereby the biggest pharmaceutical firms
(up from 825 last year), and a huge 2,249 (up were contributing smaller and smaller shares of
from 2,083) with just a single candidate. Just drugs to the overall pipeline. The Top 10 are now
as it takes many tiny water droplets to make providing 4.4% of drugs, up from 4.1%, while the
up a large cloud, taken together, these pharma Top 25 are delivering 8.2%, rising from 7.8%.

Figure 4: Share of the pipeline contributed by top 10 companies,

top 25 companies, and companies with just one or two drugs, 2011—24

Top 10 Top 25 Companies with 1 or 2 drugs

24
% OF TOTAL PIPELINE

19

14

4
2011 2012 2013 2014 2015 2016 2017 2018 2019 2020 2021 2022 2023 2024

YEAR
Source: Pharmaprojects®, January 2024

13 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

The Top 10 companies are continuing to display
interests across a wide variety of therapeutic
areas, as illustrated in Figure 5. In fact, the
top five firms have drugs in development in
every single one of the 14 therapeutic areas,
while numbers 6 to 10 only miss out on one
of the smaller areas each (antiparasitics
for one, hormonal drugs for the other four).
This indicates that, despite received wisdom
suggesting that companies are refining
their efforts to specialize in just a handful of
disciplines, for the very largest companies at
least, this simply isn’t the case. There are some
interesting diversifications within that, however,
with Eli Lilly once again bucking the trend by
being the only Top 10 company that doesn’t
have anticancers as its largest therapeutic focus
(it instead prefers alimentary/metabolic drugs).
Meanwhile, Bristol Myers Squibb is the company
with the most skewed pipeline, with nearly 50%
of its drugs having an oncology focus, whereas
interest in other areas languishes at single-digit
percentages. Sanofi almost matches its cancer
portfolio size with that of its anti-infectives,
with Pfizer and Merck & Co. also having sizable
portfolios in that therapeutic area.

Figure 5: Disease focus areas of the Top 10 pharma companies

2024
100%
90%
80%
70%
60%
50%
40%
30%
20%
10%
0%
Pfizer Roche AstraZeneca Novartis Bristol-Myers Sanofi Eli Lilly Takeda Johnson Merck
Squibb & Johnson & Co.

Alimentary/Metabolic Anticancer Anti-infective

Antiparasitic Blood and Clotting Cardiovascular
Dermatological Genitourinary (including sex hormones) Hormonal (excluding sex hormones)
Immunological Musculoskeletal Neurological
Respiratory Sensory

Source: Pharmaprojects®, January 2024

14 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

Rare diseases also continue to excite those
at the helms of the biggest pharma beasts,
with all the Top 10 companies featuring in our
table of the 20 companies with the most drugs
against rare diseases in their pipelines (Table
2). Novartis comes out on top by this measure,
pushing Pfizer back into second place. Novartis
has the second-highest proportion of its pipeline
focused on rare diseases, its 53.6% being just
edged out by Biogen’s 53.7%. Elsewhere, only
Bristol Myers Squibb also breaks the 50% barrier.
At the other end of the scale, the company
with the lowest percentage is the previously
highlighted Chinese company Jiangsu Hengrui,
with just 24.8%. Eli Lilly comes pretty close to
that figure, with just 25.4% of its pipeline hitting
rare diseases, further cementing the view that
the company is something of an anomaly
among the big hitters.

Table 2: Top 20 pharma companies with a rare disease focus

NUMBER OF DRUGS
COMPANY % OF PIPELINE
FOR RARE DISEASES

Novartis 118 53.6

Pfizer 112 40.1
Bristol-Myers Squibb 110 50.9
Roche 102 36.8
Sanofi 85 40.5
AstraZeneca 81 36.2
Takeda 80 39.8
Johnson & Johnson 71 37.0
GlaxoSmithKline 68 36.0
Merck & Co. 59 30.9
AbbVie 58 37.2
Amgen 52 46.8
Eli Lilly 51 25.4
Eisai 42 45.7
Bayer 40 34.2
Astellas Pharma 39 37.1
Jiangsu Hengrui Pharmaceuticals 37 24.8
Biogen 36 53.7
Otsuka Holdings 36 34.6
Sino Biopharmaceutical 36 34.3

Source: Pharmaprojects®, January 2024

15 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 The total number of companies involved in the there were definitely more companies entering
pharma R&D ecosystem has now hit 6,124, the fray during 2023, with 921 being added to
as evidenced by Figure 6, continuing another our database, considerably more than the 809
upward trend. Unfortunately, a calculation discovered the previous year. This would suggest
error means that the equivalent figure for 2023 that around 500 companies were removed from
published last year was incorrect, so we have our database over the past 12 months, either
replaced this with an estimated total of 5,700. having been acquired, having folded, or, in the
If accurate, this would give us a growth in the majority of cases, seemingly having become
total number of companies developing drugs of inactive. It’s possible that, for some of these
7.4%. Either way, it’s clear that the sun continues companies, while their current status is somewhat
to shine
7,000 on the industry, with no shortage of new hazy, they will yet emerge from the mist and
companies bubbling up in its sky. We can say that rejoin the active set for next year.

Figure 6: Total number of companies with active pipelines, 2001—24

6,124

6,000

5,700

5,416

5,099

5,000
4,816

4,323

4,134
4,003
4,000
COMPANY COUNT

3,687

3,286

2,984
3,000
2,745
2,705

2,387
2,207
2,084
1,965
2,000
1,769
1,633
1,576 1,621
1,503

1,313
1,198

1,000

0
2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018 2019 2020 2021 2022 2023 2024
YEAR
*Estimated figure
Source: Pharmaprojects®, January 2024

16 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

Which way is the wind blowing in terms of has increased once again, up from 13% to 16%.
geographies for drug development? Figure This represents an acceleration in the latter’s
7 suggests that the pharma pressure map growth, with 1,008 Chinese-headquartered
indicates that the breeze is continuing to flow companies reported this year, up from 808 last
from west to east. We can see that, while 43% year and 792 the year before. Whereas last
of all companies involved in pharma R&D a year we had suggested that growth in China
year ago were headquartered in the US, this might be slowing, in 2024, there appears to be a
figure has taken a significant dip in 2024 to 39%. strengthening of the air currents in this direction
Meanwhile, the proportion of China-based firms from gentle to gusty.

Figure 7: Distribution of R&D companies by HQ country/region, 2023 and 2024

1%

4%

13%

3%
2%
3%
2023 43%
USA

UK
11%

Rest of Europe

Rest of Asia-Pacific
15%
5%
Japan

1% Germany

3%
France

16% China

Canada

3% C and S America/Africa
39%
2024 2%
3%

13%

5%
15%

Source: Pharmaprojects®, January 2024

17 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

Similar trends can be observed in Table 3, which The data for 2024 reinforce trends seen over
examines the locations where drug development a number of years in that R&D is experiencing
is actually occurring. While the US still dominates, an easterly airflow. This year, we have our first
its presence is in decline. In all, 49.1% of all drugs Chinese Top 10 pharma company, and the US
in development have some US R&D activity; this losing its majority as the favored place for
is down from 51.1% last year, and falls below the pharma R&D. Our forecast here is that such
50% mark for the first time. Again, this ground climactic changes are likely to continue for
has been ceded largely to China, which increases several years to come.
its share from 23.6% to 26.7%. Another Asian
country, South Korea, has snuck into third place,
pushing the UK into fourth. Note that the table
only lists countries where 5% or more of drugs
are in development. In total, our database
reports active R&D in 162 countries this year.

Table 3: Where is R&D actually occurring?

DRUG DEVELOPMENT COUNTRY NUMBER OF DRUGS % OF PIPELINE

USA 11,200 49.1

China 6,098 26.7
South Korea 3,233 14.2
UK 3,156 13.8
Germany 2,479 10.9
Canada 2,387 10.5
Australia 2,372 10.4
France 2,363 10.4
Spain 2,259 9.9
Japan 2,041 8.9
Netherlands 1,850 8.1
Italy 1,823 8.0
Belgium 1,783 7.8
Poland 1,724 7.6
Sweden 1,562 6.8
Denmark 1,536 6.7
Switzerland 1,504 6.6
Czech Republic 1,406 6.2
Hungary 1,398 6.1
Austria 1,369 6.0
Taiwan, China 1,362 6.0
Bulgaria 1,244 5.5
Finland 1,178 5.2
Portugal 1,174 5.1
Greece 1,171 5.1
Ireland 1,158 5.1
Romania 1,153 5.1
Israel 1,147 5.0
Norway 1,146 5.0
Source: Pharmaprojects®, January 2024

18 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 Therapies and Diseases
Cancer still taking R&D by storm

How many different types of weather are there? However, over the years we have added both to
the broad therapeutic areas and to the individual
There doesn’t seem to be an internationally
250+ therapeutic categories to more closely reflect
agreed answer, and even in the various weather modern drug development.
apps you probably have on your phone, there
will probably be some discord. Try this for a We start our analysis in this area by looking at how
basic list: sunny, partly cloudy, cloudy, drizzle, numbers have changed year-on-year across those
rain, stormy, foggy, windy, snowy. But, of 14 therapeutic areas, along with the two additional
course, many of these are not mutually classes that we created, biotechnology products
exclusive. Similarly, drug development for and reformulations. This shows anticancer drugs
disease types can cross multiple therapeutic reaching stratospheric levels again, with 9,142
areas, and we likewise assign them into a drugs in development for oncology, up from 8,480
number of big buckets. last year, an expansion of 7.8%, slightly ahead of
the global pipeline expansion rate we reported
In this section, we move to looking at trends earlier in this review. Neurologicals also marginally
across the 14 major therapeutic area types for beat the average, with a 7.5% growth rate, but
which drugs are under development. Just like alimentary/metabolics put in a really impressive
with weather, there is no internationally agreed performance, swelling the pipeline by 13.1%.
classification for therapeutic areas, with several Meanwhile, growth in anti-infective R&D, which
ontologies such as ICD-10 and MeSH coexisting. saw a boost during the COVID-19 pandemic but
Our own system has its origins in the EphMRA had since fallen to a below-average growth rate,
classification system, which itself is closely has now almost stalled completely, increasing by
aligned with the well-known ATC code taxonomy. a paltry 0.8% this year.

Figure 8: R&D pipeline by therapeutic area, 2023 and 2024

10,000

9,142 9,100
9,000
8,480
8,394

8,000

7,000

6,000

5,000

4,000
3,713
3,454
3,319
2,951
2,933 2,927
3,000

2,210 2,074
2,066
1,914
2,000
1,453
1,262 1,219 1,215 1,157 1,087
1,130 1,143 1,126 988 820 783
1,000
750 741
275
247 112
110
0
ns
ic

ar
al

al
e

g
y

y
l

al

c
ry
r

ca

al
y
io
ol

iv
og

tin
ce

iti
ar
ic

ic

ul
or
et

to

on
ab

ct

at
gi

og

og

as
sc

in
an

ol

ot
ns
el

ira
fe

ul

m
lo

ur
et

va
hn

ar
sk

ol

ol

Cl
tic

Se
m
In
ro

or
sp
/M

ito
un

at

tip
lo

io
ec

&
ru
An

ti-
eu

H
Re
cu

rd

en
m

An
ot

ry

d
fo
An
N

er

oo
Im

Ca
us
ta

G
Bi

Re

D
M

Bl
en
im
Al

Source: Pharmaprojects®, January 2024 2023 2024

19 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 Oncology’s above-average result has meant a
further grab of its overall share of the pipeline,
which Figure 9 shows. However, the rate at which
its advance has slowed might be some comfort
for the other therapeutic areas, which must be
concerned to see their metaphorical polar ice
caps continuing to recede.

Figure 9: Proportion of the pipeline in development for cancer, 2010—24

45.0

39.8 40.1
40.0 39
% OF PIPELINE WHICH IS ONCOLOGY

37.5
36.7
35.2
35.0 34.1

32.6

30.1 30.4
29.5 29.3 29.3
30.0
28
26.8

25.0

20.0
2010 2011 2012 2013 2014 2015 2016 2017 2018 2019 2020 2021 2022 2023 2024

YEAR
Source: Pharmaprojects®, January 2024

20 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

Table 4 zooms in on this analysis further, but there is further evidence of interest here
providing the Top 25 of the 243 individual peaking, with a growth rate of just 3.3% this
therapeutic categories used to classify drugs year, down from 6.3% last year and 23.3% the
in Pharmaprojects and the Citeline suite of year before. The Top 10 of this chart is once
products. Unsurprisingly, there are big boosts for again pretty stable, as if an anticyclone has
the top two anticancer categories: anticancer, settled over therapeutic category changes,
immunological (+8.2%); and anticancer, other leading to a long period of calm weather.
(+8.6%). Gene therapy again comes in third,

Table 4: Top therapeutic categories

POSITION 2024 NUMBER OF ACTIVE

THERAPY TREND
(2023) COMPOUNDS 2024 (2023)

1 (1) Anticancer, immunological 4,860 (4,492) ↑↑

2 (2) Anticancer, other 3,935 (3,622) ↑↑

3 (3) Gene therapy 2,151 (2,083) ↔

4 (4) Monoclonal antibody, other 1,595 (1,395) ↑

5 (6) Neurological 1,125 (1,045) ↔

6 (5) Prophylactic vaccine, anti-infective 1,115 (1,064) ↔

7 (7) Ophthalmological, other 1,054 (984) ↔

8 (8) Antiviral, other 935 (983) ↓

9 (9) Immunosuppressant 899 (797) ↑

Cellular therapy, chimaeric antigen ↔

10 (10) 795 (792)
receptor
11 (11) Antidiabetic 772 (747) ↔

12 (14) Musculoskeletal 767 (677) ↑

13 (12) Anti-inflammatory 747 (722) ↔

14 (13) GI inflammatory/bowel disorders 740 (705) ↔

15 (15) Cognition enhancer 702 (641) ↑

16 (17) Monoclonal antibody, humanized 672 (624) ↑

17 (16) Respiratory 654 (632) ↔

18 (18) Cardiovascular 645 (599) ↑

19 (19) Neuroprotective 632 (595) ↔

20 (20) Hepatoprotective 618 (594) ↔

21 (21) Dermatological 589 (559) ↔

22 (22) Urological 578 (519) ↑

23 (24) Antiparkinsonian 570 (516) ↑

24 (32) Antibody-drug conjugate 551 (405) ↑↑

25 (23) Analgesic, other 547 (517) ↔

Source: Pharmaprojects®, January 2024

21 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 Despite the fact that gene therapy and cell
therapy growth appears from this table to
be slowing, they both remain major drivers
of drug development. Figure 10 shows this,
but also reinforces the fact that their rates of
expansion are slowing down from the frankly
remarkable snowballing of R&D seen in the last
decade. Of course, these two categorizations
are not mutually exclusive; indeed, 1,192 drugs
are classified as both — these are generally
therapeutics whereby cells are removed from
the body, genetically altered ex vivo, and then
reintroduced to the patient (such as with CAR-T
therapies). This means there are 959 gene
therapies which are not also cell therapies;
these will be primarily in vivo gene therapies,
where genes are delivered in vivo to cells via a
vector, or where genes are edited using newer,
cutting-edge technologies (of which more later).
This figure has grown from 886 last year. Cell
therapies without a gene therapy component
now stand at 916, up from 852.

Figure 10: The ongoing rise of gene and cell therapies

2,500

2,151
2,108
2,083
2,048
1,960
2,000

1,936
1,836
NUMBER OF DRUGS

1,589

1,500
1,427
1,273
1,140

Gene Cell
1,000
867

862
722

687
610

564
479

500
416
327
302
275

276
255

242
249

208

239

238
206
223

217

232
221

220
196

204

209
191

191

194

193

168
158
142

150
122

117
115
114

106

93
64
54
54
99

39
32
19
16

0
1996

2006

2016
2000

2010

2020
1995

1998

1999

2002

2003

2004

2005

2008

2009

2012

2013

2014

2015

2018

2019

2022

2023
2024
1997

2007

2017
2001

2011

2021

YEAR
Source: Pharmaprojects®, January 2024

22 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

Within the umbrella term of gene therapy, a projects, including two of the six launched gene
multitude of different technologies are covered, therapies using viral vectors. They also have
with one major variation being how the genetic significant numbers of drugs at Phase I, II, and
change is introduced into cells. Whereas III, although much of the work here is still at the
sometimes this is achieved via chemical preclinical stage of development. Lentiviruses
methods or using plasmids, by far the most come in second; this type of vector is more
common method is to use a viral vector. The commonly used for ex vivo transfection of cells.
breakdown of the viral vector types is shown in Whereas the number of projects using AAV
Figure 11, which further breaks each down by vectors has increased this year, lentivirus use is
global phase of development. You can see here in decline, with 185 drugs this year, down from
that, by some measure, adeno-associated viral 241. The use of adenovirus took an even bigger
(AAV) vectors are both the most popular and tumble, falling from 109 to 43. AAV seems to be
the most mature. They account for just over 500 the weapon of choice.

Figure 11: Viral vectors used in gene therapies

600

500

400

300

200

100

0
Adeno-associated Lentivirus Adenovirus Retrovirus Herpes simplex Poxvirus Polyomavirus Rhabdovirus
virus virus

Preclinical Phase I Clinical Trial Phase II Clinical Trial Phase III Clinical Trial Pre-registration Registered Launched Suspended

Source: Pharmaprojects®, January 2024

23 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

The types of cells used for cell therapy are
considerably more diverse, with in total 24
different kinds of cells currently being employed.
T cells remain the most popular, due to their
predominance in the popular CAR-T-based
therapeutics, although there are somewhat
fewer this year. In contrast, the temperature
is rising for stem cells, which have posted
a modest increase, along with blood cells
and antigen-presenting cells. Figure 12 has
the details.

Figure 12: Cell types used in cell therapies

900

800

700

600

500

400

300

200

100

0
ll
l

ll

ll
ll

ll
ll
ll

ll
el
ll

e
ce

ce

ce
ce

ce
ce
ce

ce
ce

om
rc

ed
r

tic

ow
em

d
T

ille

ito
os
in

su
oo

ea

ifi
nt

r
Ex
tis
k
St

en

ar
Bl

ec
se

r
al

nc
og

m
e

sp
ur

re

tiv

Pa

ne
Pr
at

-p

er
ec

Bo
N

en

th
nn

O
tig

Co
An

Preclinical Phase I Clinical Trial Phase II Clinical Trial Phase III Clinical Trial Pre-registration Registered Launched Suspended

Source: Pharmaprojects®, January 2024

24 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

We now move to what is always one of the most it should be noted that only specific diseases
interesting Top 25s — the individual diseases on are listed; non-specific indications such as
which the pharmaceutical industry is focusing its “Cancer, unspecified” have been removed
attention most. These are listed in Table 5, where from this analysis.

Table 5: Top 25 diseases/indications

POSITION 2024 NUMBER OF ACTIVE

THERAPY TREND
(2023) COMPOUNDS 2024 (2023)

1 (1) Cancer, breast 1,031 (965) ↑

2 (2) Cancer, lung, non-small cell 1,010 (925) ↑↑

3 (3) Cancer, colorectal 825 (741) ↑↑

4 (4) Cancer, pancreatic 740 (675) ↑

5 (6) Cancer, ovarian 625 (587) ↔

6 (5) Cancer, brain 580 (539) ↑

Infection, coronavirus, novel ↓

7 (7) 580 (653)
coronavirus
8 (8) Cancer, prostate 576 (523) ↑

9 (9) Alzheimer's disease 563 (529) ↔

10 (10) Cancer, liver 501 (444) ↑

11 (11) Diabetes, Type 2 498 (475) ↔

Cancer, leukaemia, acute ↔

12 (14) 497 (484)
myelogenous
13 (12) Cancer, melanoma 496 (476) ↔

14 (13) Cancer, gastrointestinal, stomach 473 (449) ↔

15 (15) Cancer, myeloma 459 (445) ↔

16 (17) Parkinson's disease 457 (407) ↑

17 (16) Arthritis, rheumatoid 449 (431) ↔

18 (18) Cancer, head and neck 432 (408) ↔

Infection, coronavirus, novel ↓
19 (19) 425 (444)
coronavirus prophylaxis
20 (20) Cancer, lymphoma, non-Hodgkin's 419 (399) ↔

21 (21) Non-alcoholic steatohepatitis 383 (369) ↔

22 (22) Psoriasis 348 (337) ↔

23 (24) Obesity 300 (230) ↑↑

24 (32) Cancer, renal 299 (281) ↔

25 (23) COVID-19 complications 285 (332) ↓

Source: Pharmaprojects®, January 2024

Breast cancer remains the hottest place on of the colorectal cancer pipeline has risen by
the temperature chart, with a pipeline that has 11.3%. Tumors of the pancreas, the ovary, and
warmed up by a further 6.8%, slightly below the the brain round off an all cancer top six this year.
overall expansion rate. As a result, it is in danger The incursion made into the top five by the novel
of being overtaken by second-placed non-small coronavirus has now ended, with this indication
cell lung cancer, which itself advanced by 9.2%. dropping to number 7 with a pipeline that
Even more impressively, at number 3, the size shrank by 11.2%. Nonetheless, with 580 drugs in

25 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 development against it, there is still significant events across the globe: temperatures plunging
effort being put into the ongoing battle with the to -28°C (-18.4°F) in Afghanistan, flooding
virus, which thankfully has now become endemic in California and South Korea, heat-induced
rather than pandemic. wildfires in Canada and Hawaii, and a record-
breaking cyclone in Brazil. Climate change
Elsewhere, that eternal tough nut to crack, appears to increase the frequency of extreme
Alzheimer’s disease, remains as the only other weather, a concern for us all. For most of us,
non-oncology disease in the Top 10, which also such catastrophic weather events can still be
sees a new entrant in the form of liver cancer, considered rare. But they nevertheless quite
up a significant 12.8%. Further down in the Top rightly grab our attention.
25, we see declines for two further COVID-19-
related categories, those covering vaccines There is also a big focus on rare diseases in
and complication therapies; but again, there
pharma R&D. Such syndromes may not affect
is still considerable activity in both. Perhaps
many of us, but for those unfortunate enough
the most striking change in an otherwise fairly
to suffer from them, they can be similarly
stable table is the 30% increase in the R&D of
devastating. This, and the big paydays pharma
drugs to treat obesity. This is no doubt related
can command for successfully developing drugs
to the success of Novo Nordisk’s Wegovy
(semaglutide), which was first launched for this for orphan indications, means that rare diseases
additional indication in mid-2021, setting off are a major focus for the pharmaceutical
a stampede for access that has led to supply industry too. In Pharmaprojects, a rare disease
issues. Note that this global picture masks some is defined as one with a prevalence of 1 in
regional variations in the most popular disease 2,000 people in the EU, or affecting fewer than
targets for drug R&D, which we will look at later 200,000 people in the US (equivalent to around
in this report. 1 in 1,600 people). Figure 13 demonstrates that
the number of individual rare diseases being
While sunshine, cloudy skies, or rain showers targeted by drug development continues to
are commonplace for most of us, there are grow, hitting 761 by the end of 2023, with slightly
some weather events which are thankfully more new additions to this pot over the course
rarer. 2023 saw a number of freak weather of that year than during the previous one.

Figure 13: Number of rare diseases being targeted by pharma

NUMBER OF NEWLY IDENTIFIED TARGETS

2023 761
2022 718
2021 677
2020 648
2019 608
2018 580
2017 546
2016 509
2015 476
2014 440
2013 389

0 100 200 300 400 500 600 700 800

YEAR
Source: Pharmaprojects®, January 2024

26 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

In total, the number of drugs under development metabolism. In contrast, cancer is only the fourth
for rare diseases at the start of 2024 hit 7,191, most common therapeutic area for rare diseases.
up 7.6% from last year’s 6,682, and representing Despite this, it still commands the most attention
31.5% of all drugs in the pipeline, up a shade in terms of drugs under development, with 39%
from 30.2% in 2023. As Figure 14 shows, the of all drugs in R&D for rare diseases being for
largest proportion of rare diseases, 19%, sit in a rare cancer. By this measure, alimentary/
the alimentary/metabolic area — this is not metabolic is pushed to joint third place, with
surprising, since there are many rare inherited a paltry 7% of the effort sitting there.
genetic conditions that cause disorders of the

Figure 14: Rare diseases by therapeutic area, by number of diseases and number of drugs

Rare Diseases by TA
Sensory Products
4%
3%
Alimentary/Metabolic Products
19%

Respiratory Products
Anticancer Products

16% Anti-infective Products

Neurological Products
Antiparasitic Products

Blood and Clotting Products

12%
Musculoskeletal Products
Cardiovascular Products
9%
Dermatological Products

Immunological Products
Genitourinary Products (including sex hormones)

6% Hormonal Products (excluding sex hormones)

13%
2% Hormonal Products (excluding
2% Immunological Products
3%
4% 5% 2%
Musculoskeletal Products
Genitourinary Products (includ
Neurological Products

Drugs for Rare Diseases by Project Respiratory Products

Dermatological
Sensory Products
Products
Sensory Products
4% 7%
7%
Cardiovascular
Respiratory Products
Products

12%
Blood and Clotting
Neurological Products
Products

5%
39% Antiparasitic Products
Musculoskeletal Products

7% Anti-infective Products
Immunological Products

1%
2%
AnticancerProducts
Hormonal Products(excluding s
1%
2% 5%
6%

2% Alimentary/Metabolic
Genitourinary ProductsProducts
(includi
Source: Pharmaprojects®, January 2024

27 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 Like all drug R&D, most of the rare disease
work is at the preclinical stage, some of it at
the cutting edge of development techniques.
But there is considerable clinical activity too.
Data from Trialtrove, another product in the
Citeline suite that tracks individual clinical trials,
shows that there were 2,430 clinical trial starts
associated with rare disease-focused drugs
during 2023 — up from 2022’s figure, but by
a somewhat lower percentage than in the
previous 12 months (Figure 15).

Figure 15: Industry-sponsored rare disease trials by start date, 2010—present

NUMBER OF TRIALS
3,000

2430
2,500
2335

2,000
1845 1862
1807
1704 1747

1540
1490
1,500
1298 1325
1236 1261
1202

1,000
sex hormones)

ng sex hormones)
500

0
2010 2011 2012 2013 2014 2015 2016 2017 2018 2019 2020 2021 2022 2023
Source: Trialtrove®, January 2024

28 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

While we’re on the subject of clinical trial a 7.7% rise on 2023’s figure, representing a
statistics rather than drugs, let’s take a look at slight slowdown in the growth rate from last
all of the ongoing (open, closed, or temporarily year’s 8.7%. CNS in second posted a similar
closed) clinical trials reported by Trialtrove growth rate of 7.6%. But infectious disease in
during 2023. Figure 16 breaks this down by third reported a decline of 3.1%, the strongest
trial phase and by therapeutic area (note that evidence yet that the spike caused by the
Trialtrove uses slightly different therapeutic COVID crisis is well and truly over (note that
areas from Pharmaprojects). The dominance infectious disease vaccines was the only other
of cancer is far more pronounced here, skewed therapeutic area to show a drop). Indeed, the
by the fact that oncological candidates may number of novel coronavirus-related ongoing
often be tested in multiple clinical trials across trials took a tumble this year, standing at 2,016,
different cancer types. There are 18,966 trials down from 2,384 last year.
under way in cancer at the start of 2024,

Figure 16: Ongoing clinical trials, by therapeutic area

2024
20,000
18,000
16,000
14,000
12,000
10,000
8,000
6,000
4,000
2,000
0
n
y
e

y
ar

y
y

D
io
og
as

ar

og
og

CN

ul

(I
at

in
ise

ol

sc

ol
ol

es
m

ur
rin

m
nc

va
D

in
ito

al
oc
O

us

io

cc
fla

th
en
rd
nd
io

Va
In

ph
G
ct

Ca
/E

e/

O
fe

ic

un
In

ol

m
ab

im
et

to
M

Au

I I/II II II/III III III/IV IV

Source: Trialtrove®, January 2024

29 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 The number of approvals of emergency
authorization — a strategy which came to the
fore during the pandemic — fell back too last
year. Figures for this metric, shown in Figure
17, would seem to exactly track the course of
the crisis. The amount of orphan drug and
expedited review designations also dropped
slightly in 2023.

Figure 17: Numbers of drugs receiving Orphan Drug status, Expedited Review designation*,
and Emergency Authorization**, 2013—23

500

450 434 425

394 397
400 378 381
369 367
348 338 341
350 326
316
NUMBER OF DRUGS

299 298 302

300 269
250
250

200 182 188

157
150

100
50 39
50 24 22
1
0
2013 2014 2015 2016 2017 2018 2019 2020 2021 2022 2023

YEAR
Orphan Drug Status Granted Expedited Review Status Granted Emergency Authorization Granted

*Data for 2013 not complete as we only began systematically recording the dates of these events mid-year.
**Emergency Authorizations only tracked from 2019.
Source: Pharmaprojects®, January 2024

So, we seem to be entering a sunshine-after-the-

rain period of drug development, as the clouds
of COVID, if not exactly dissipating, appear to
be scooting out to sea towards the horizon. And
the horizons for drug R&D are still expanding;
there are 1,514 individual diseases being
targeted by active drug development this year,
up from 1,452 a year ago. The pandemic actually
accelerated progress in drug development in
a number of areas, such as the use of mRNA-
based vaccine technology. In that sense, the
coronavirus-shaped cloud really does seem to
have had a silver lining.

30 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 Regional
Variations
Where the outlook
seems to be brightest

While understanding changes in

global climate is undoubtedly important,
such data are not likely to be of much
use in helping you to decide whether
you can wear shorts tomorrow or
whether you need to bring a woolly
hat. So, new for this year’s report, we
are introducing several regional sub-
analyses, looking at pipeline size growth
rates, leading companies, and top
diseases across a range of some of the
largest pharmaceutical markets across
the world. Is it sunny in China, but
tipping it down across Europe?

Let’s find out…

Source: Pharmaprojects®, January 2024

31 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline
 United States
It seems prudent to start our regional analyses in the largest of the world’s pharma markets, the
US. The number of drugs in active development in the US has consistently grown since 1995, with
an average pipeline increase of 284.8 drugs per year, corresponding to 4.8% year-on-year (YoY)
growth. While the number of active pipeline drugs continues to increase in the US, the rate
of growth has decelerated.

From 2023 to 2024, there was an increase of 393 active drugs in the US pipeline, equivalent to 3.6%
YoY growth. This is around half the overall global pipeline growth rate of 7.3%. The US has historically
been a powerhouse of drug development, but, as we have seen, has been ceding some ground in
recent years. Since 2021, the US’s annual pipeline growth rate has been well below the worldwide
growth rate (2.9 percentage points [pp] lower in 2021–22, 4.4pp lower in 2022–23, and 3.7pp lower
in 2023–24).

Figure 18: Total US R&D pipeline size, 1995—2024

11,197
12,000

10,804
10,679
10,271
9,477
9,140
10,000

8,535
8,370
7,907
8,000

7,034
DRUG COUNT

6,305
6,144
6,162
5,751
5,648
5,586
5,312

6,000
4,497
4,354

4,342
4,265
4,134
3,699

3,781
3,590
3,476
3,350
3,396

4,000
2,989
2,937

2,000

0
1996

2006

2016
2000

2010

2020
1995

1998

1999

2002

2003

2004

2005

2008

2009

2012

2013

2014

2015

2018

2019

2022

2023

2024
1997

2007

2017
2001

2011

2021

Source: Pharmaprojects®, January 2024 YEAR

32 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

As of January 2024, there were 2,609 companies Table 6: Top 10 US—headquartered
developing drugs that are headquartered in the companies by size of pipeline
US. Of these, 39.9% were developing one drug,
83.5% were developing ≤5 drugs, and 96.6% PIPELINE
COMPANY
DRUGS
were developing ≤10 drugs. The Top 10 companies
are listed in Table 6, alongside the size of Pfizer 204
their pipelines.
Johnson & Johnson 149
(Note: data in this chapter were pulled at a slightly later
date than the data used for Table 1, hence there may be Eli Lilly 144
minor differences in numbers.)
Merck & Co. 139
AbbVie 109
Bristol-Myers Squibb 106
Gilead Sciences 85
Amgen 76
Regeneron 74
Moderna 69
Source: Pharmaprojects®, January 2024

The Top 10 diseases in active development in the US are of course dominated by cancer indications,
but here, non-small cell lung cancer tops the table, pushing breast cancer into second. Two non-
cancer indications appear in the Top 10 disease list for the US: Alzheimer’s disease (ranked sixth with
236 drugs in development, higher than globally) and the novel coronavirus (ranked ninth with 224
drugs in development, lower than globally). The Top 10 diseases in development in the US are nearly
identical to the Top 10 worldwide list, the only exception being that acute myelogenous leukemia
appears in the US Top 10, replacing liver cancer from the worldwide list.

Table 7: The US’s Top 10 diseases for pipeline drugs

PIPELINE
POSITION DRUG DISEASE
DRUGS

1 Cancer, lung, non-small cell 414

2 Cancer, breast 356
3 Cancer, colorectal 287
4 Cancer, pancreatic 249
5 Cancer, ovarian 237
6 Alzheimer's disease 236
7 Cancer, brain 235
8 Cancer, prostate 225
9 Infection, coronavirus, novel coronavirus 224
10 Cancer, leukaemia, acute myelogenous 223

Source: Pharmaprojects®, January 2024

33 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 Japan
The size of Japan’s R&D pipeline has steadily increased since 2007, following an overall decrease in
pipeline size from 1995. The pipeline has increased by an average of 75.6 drugs per year since 2007,
with a mean 6.1% YoY growth rate. From 2023 to 2024, the number of drugs in active R&D in Japan
increased from 1,981 to 2,044, corresponding to a 3.2% growth rate. Based on these data, while the
number of pipeline drugs continues to increase in Japan, the rate of growth has begun to decelerate.
That said, since 2007, growth of Japan’s pipeline has largely kept pace with the rest of the world. The
average worldwide annual rate of pipeline growth since 2007 is 6.9%, only 0.8pp higher than in Japan.

Figure 19: Total Japan R&D pipeline size, 1995—2024

2,500

2,044
1,981
1,835
1,796
2,000

1,732
1,632
1,579
1,583
DRUG COUNT

1,567
1,413
1,350
1,500

1,248
1,237
1,207
1,181

1,173
1,147
1,084

1,028
977
981

886
883

859

1,000 840
814
811

785
767

759

500

0
1997

1999
1995
1996

1998

2011

2017
2012
2013

2019
2015
2016

2018
2010

2014

2021
2022
2023
2020
2001

2024
2007
2002
2003

2009
2005
2006

2008
2000

2004

Source: Pharmaprojects®, January 2024 YEAR

As of January 2024, there were 184 companies developing drugs that are headquartered in
Japan. Of these, 35.3% were developing one drug, 63.6% were developing ≤5 drugs, and 83.7%
were developing ≤10 drugs. The Top 10 Japanese companies are listed in Table 8, alongside the
size of their pipelines.

Table 8: Top 10 Japan-headquartered

companies by size of pipeline

COMPANY PIPELINE DRUGS

Takeda 130
Astellas Pharma 74
Eisai 73
Daiichi Sankyo 55
Ono Pharmaceutical 50
Shionogi 48
Otsuka Pharmaceutical 34
Sosei Heptares 32
Taiho 32
Mitsubishi Tanabe Pharma 31
Source: Pharmaprojects®, January 2024

34 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

Cancer indications dominate Japan’s Top 10 diseases in active development, taking seven out
of the 10 spots. However, while all the cancer indications are solid forms in the worldwide Top 10
diseases (breast, non-small cell lung, colorectal, pancreatic, ovarian, brain, prostate, and liver),
Japan’s Top 10 list also includes the hematological cancers of non-Hodgkin’s lymphoma and
myeloma. The non-cancer indications included in Japan’s Top 10 differ from both the worldwide
list and that of the US, being type 2 diabetes, ulcerative colitis, and rheumatoid arthritis.

Table 9: Japan’s Top 10 diseases for pipeline drugs

POSITION DRUG DISEASE PIPELINE DRUGS

1 Cancer, lung, non-small cell 97

2 Cancer, breast 56
3 Cancer, colorectal 53
4 Cancer, gastrointestinal, stomach 43
5 Diabetes, Type 2 40
6 Cancer, lymphoma, non-Hodgkin's 38
7 Cancer, pancreatic 34
8 Colitis, ulcerative 33
9 Arthritis, rheumatoid 32
10 Cancer, myeloma 31
Source: Pharmaprojects®, January 2024

35 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 China
Although Chinese R&D companies only emerged into the pharmaceutical market in 1997, with one
active drug in the market, they are currently responsible for developing 26.7% of globally active
drugs, as we’ve seen, making China the second-largest developer of new drugs. This reflects the
staggering growth China has gone through. The last five years have seen a boom for Chinese drug
development with an increase of 200.9% since 2019, as shown in Figure 20, partly due to the surge of
government investment in the pharmaceutical sector.

Figure 20: Total China R&D pipeline size, 1995—2024

7,000

6,119
NUMBER OF ACTIVE PIPELINE DRUGS

6,000

5,136
4,203
5,000

3,295
4,000

2,590
3,000

2,033
1,452
1,269
2,000

1,131
851
701
570
483
1,000
392
276
170

199
187
167
160
142
155

171
123
0
0

0
0
1

0
1995
1996
1997
1998
1999
2000
2001
2002
2003
2004
2005
2006
2007
2008
2009
2010
2011
2012
2013
2014
2015
2016
2017
2018
2019
2020
2021
2022
2023
2024
YEAR
Source: Pharmaprojects®, January 2024

There are a total of 1,270 pharmaceutical companies headquartered in China, which as previously
noted is now a total that is second only to the US. The Top 10 companies in China can be seen in
Table 10. From 2023 to 2024, the size of the active pipelines of nine of the Top 10 Chinese companies
increased, reflecting the growth seen across the Chinese pharmaceutical sector.

Table 10: Top 10 China-headquartered companies by size of pipeline

COMPANY PIPELINE DRUGS 2024

Jiangsu Hengrui Pharmaceuticals 147

Sino Biopharmaceutical 103
Shanghai Fosun Pharmaceutical (Group) 90
CSPC Pharmaceutical 73
BeiGene 58
Shanghai Junshi Biosciences 57
Shanghai Pharmaceuticals Holding 51
Innovent Biologics 50
Qilu Pharmaceutical 50
Huadong Medicine 44
Source: Pharmaprojects®, January 2024

36 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

As shown in Table 11, nine out of the Top 10 diseases in China are forms of cancer, with the top
three being non-small cell lung cancer, breast cancer, and gastrointestinal stomach cancer.

Table 11: China’s Top 10 diseases for pipeline drugs

POSITION DRUG DISEASE NUMBER OF DRUGS

1 Cancer, lung, non-small cell 383

2 Cancer, breast 325
3 Cancer, gastrointestinal, stomach 227
4 Cancer, colorectal 218
5 Cancer, liver 207
6 Diabetes, Type 2 187
7 Cancer, pancreatic 169
8 Cancer, lymphoma, non-Hodgkin's 168
9 Cancer, myeloma 159

Cancer, leukaemia, acute

10 141
myelogenous
Source: Pharmaprojects®, January 2024

37 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 India
In India, active pipeline growth saw its steepest change from 2007 to 2013, across which there was
a 309.9% increase in total active pipeline drugs. After 2013, the number of active drugs plateaued
at around 800; however, since 2021, a steady decrease has been observed. There was an 18.9%
decrease in the total number of active pipeline drugs observed from 2021 to 2023, as shown in
Figure 21. But this year, that decline was almost completely reversed.

Figure 21: Total India R&D pipeline size, 1995—2024

900

832

816
812

810
803
NO. OF ACTIVE PIPELINE DRUG

794
783

778
800

747

714

694
700

648

631
567
600

500

428
400
323
285

300
191

200
153

148
147
132
90

100
62
0
0
0
0
0
0

0
1997
1995
1996

1998
1999

2011

2017
2012
2013

2019
2015
2016

2018
2010

2014

2021
2022
2023
2020
2001

2024
2007
2002
2003

2009
2005
2006

2008
2000

2004

YEAR
Source: Pharmaprojects®, January 2024

In India, there are 120 pharmaceutical companies with their headquarters in that country, less than
10% of the total number of companies in China. Out of the Top 10 pharmaceutical companies in
India shown in Table 12, seven saw a decrease in their total pipeline size this year.

Table 12: Top 10 India-headquartered companies by size of pipeline

COMPANY NUMBER OF DRUGS

Dr. Reddy’s Laboratories 36

Lupin 29
Sun Pharmaceutical Industries 21
Serum Institute of India 20
Zydus Lifesciences 19
Biocon 18
Cipla 13
Intas Pharmaceuticals 12
Suven Life Sciences 12
Bharat Biotech 11
Source: Pharmaprojects®, January 2024

38 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

Table 13 shows that India’s top disease targets for R&D confound trends seen in other territories,
with type 2 diabetes coming out on top. Breast cancer and non-small cell lung cancer are relegated
to second and third place, respectively, here. They are two of only three cancer indications in
India’s Top 10, which has a wider variety of diseases across many therapeutic areas. These include
metabolic and autoimmune indications, such as rheumatoid arthritis and psoriasis, as well as
vaccines for diphtheria and tetanus.

Table 13: India’s Top 10 diseases for pipeline drugs

POSITION DRUG DISEASE NUMBER OF DRUGS

1 Diabetes, Type 2 175

2 Cancer, breast 143
3 Cancer, lung, non-small cell 105
4 Arthritis, rheumatoid 99
5 Cancer, colorectal 73
6 Infection, tetanus prophylaxis 61
7 Psoriasis 61
8 Infection, coronavirus, novel coronavirus 55
9 Infection, diphtheria prophylaxis 53
10 Pain, nociceptive, general 52
Source: Pharmaprojects®, January 2024

39 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 United Kingdom
In the UK at the start of 2024, there were 3,156 pipeline drugs, up from 3,015 in 2023. This 4.5%
increase is less than the 7.8% growth rate seen from 2022 to 2023. There was a slight dip from 2019
to 2020; however, since 2020, drug development in the UK has been steadily increasing.

Figure 22: Total UK R&D pipeline size, 1995—2024

3,156
3,500

3,015
2,779
2,690
2,704

2,637
3,000

2,590
2,564
2,483
DRUG COUNT

2,275
2,500

2,147
1,966
1,815
1,701
2,000

1,648
1,570

1,581
1,519

1,466

1,431
1,416

1,394
1,371
1,354
1,329
1,288

1,297
1,284
1,186

1,500
1,115

1,000

500

0
1996

2006

2016
2000

2010

2020
1995

1998

2002
2003

2012
2013
2014
2015

2018
1999

2004
2005

2008
2009

2019

2022
2023
2024
1997

2007

2017
2001

2011

2021
YEAR
Source: Pharmaprojects®, January 2024

There are 321 companies developing drugs headquartered in the UK. The Top 10 UK companies are
listed in Table 14, alongside the size of their pipelines. AstraZeneca and GSK dominate the UK pharma
industry, with their closest competitors having pipeline sizes that are an order of magnitude less.

Table 14: Top 10 UK-headquartered

companies by size of pipeline

COMPANY NUMBER OF DRUGS

AstraZeneca 163
GlaxoSmithKline 128
Mundipharma
20
International
Hikma Pharmaceuticals 19
Albumedix 17
Healx 15
ViiV Healthcare 14
Bicycle Therapeutics 13
PhoreMost 13
Oxford BioTherapeutics 12
Source: Pharmaprojects®, January 2024

40 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

We have seen how the global pipeline is dominated by drugs in development for various cancer
indications, and the data from early 2024 in the UK certainly reflect this, with the top seven of the
Top 10 diseases falling into the oncology space. This pattern broadly falls in line with the global
rankings; however, non-small cell lung cancer is the number 1 disease being targeted in the UK,
whereas breast cancer is number 1 globally. Three non-cancer indications appear in the Top 10
disease list for the UK: type 2 diabetes (ranked eighth), Alzheimer’s disease (ranked ninth), and
novel coronavirus vaccines (ranked 10th).

Table 15: The UK’s Top 10 diseases for pipeline drugs

POSITION DRUG DISEASE PIPELINE DRUGS

1 Cancer, lung, non-small cell 113

2 Cancer, breast 87
3 Cancer, colorectal 82
4 Cancer, prostate 63
5 Cancer, ovarian 57
6 Cancer, melanoma 52
7 Cancer, lymphoma, non-Hodgkin’s 51
8 Diabetes, Type 2 51
9 Alzheimer’s disease 49
10 Infection, coronavirus, novel coronavirus prophylaxis 48
Source: Pharmaprojects®, January 2024

41 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 Ireland
Since 1995, there has been an overall increasing trend in the total pipeline size in Ireland. In terms of
the most recent changes in pipeline size in Ireland, there are currently 1,158 pipeline drugs, which is
up from 1,099 in 2023. However, this 5.4% increase is smaller than the 6.9% growth from 2022 to 2023.

Figure 23: Total Ireland R&D pipeline size, 1995—2024

Blood and Clotting Pro
1,400

1,158
1,099
1,200 Antiparasitic Products

1,030

1,037
1,028
989
975
950
932
1,000
DRUG COUNT

860
Anti-infective Products

800
760
800

652
597
Anticancer Products

563
507

512
600
490

491
466
460

462
468
440
423
429
396
356
333

400
313

Alimentary/Metabolic
200

0
1996

2006

2016
2000

2010

2020
1995

1998

2008
1999

2002
2003
2004
2005

2009

2012
2013
2014
2015

2018
2019

2022
2023
2024
1997

2007

2017
2001

2011

2021
YEAR
Source: Pharmaprojects®, January 2024

There are 40 companies developing drugs headquartered in Ireland. The top 10 Irish companies are
listed in Table 16, alongside the size of their pipelines.

Table 16: Top 10 Ireland-headquartered

companies by size of pipeline

COMPANY PIPELINE DRUGS 2024

Jazz Pharmaceuticals 20
Alkermes 10
Mallinckrodt 9
Prothena 8
Perrigo 5
ONK Therapeutics 5
Shorla Pharma 4
GH Research 3
Inflection Biosciences 3
Orbsen Therapeutics 2

Source: Pharmaprojects®, January 2024

42 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

As we’ve seen, globally, the Top 10 diseases against which most drugs are in development are
concentrated among cancer indications, but the data from early 2024 for Ireland show that this
country is another that bucks the trend, with only four of the Top 10 diseases falling into the oncology
space. While the top two diseases for Ireland are non-small cell lung cancer and breast cancer,
the Top 10 indications in Ireland include a relatively higher number of metabolic and autoimmune
indications, including type 2 diabetes, rheumatoid arthritis, juvenile arthritis, psoriatic arthritis,
psoriasis, and ulcerative colitis.

Table 17: Ireland’s Top 10 diseases for pipeline drugs

POSITION DRUG DISEASE PIPELINE DRUGS

1 Cancer, lung, non-small cell 51

2 Cancer, breast 44
3 Arthritis, rheumatoid 34
4 Diabetes, Type 2 32
5 Arthritis, psoriatic 31
6 Psoriasis 31
7 Cancer, colorectal 28
8 Colitis, ulcerative 27
9 Arthritis, juvenile 25
10 Cancer, renal 24
Source: Pharmaprojects®, January 2024

43 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

Denmark
In Denmark, there are currently 1,536 active drugs, which is up from 1,444 in 2023. This 6.4% increase
is substantially less than the 11.9% YoY growth rate recorded from 2022 to 2023. The growth over the
past year is slightly above the mean YoY growth rate in the country since 1996 (5.1%). In comparison,
the progression of development in Denmark over the past year was slightly lower than the global
growth rate of 7.2%. The last two years have stood out in the recent history of Denmark’s drug
development landscape as the country has had its two best years of growth since 2016.

Figure 24: Total Denmark R&D pipeline size, 1995—2024

1,800

1,536
1,444
1,600

1,300
1,320
1,321

1,290
1,234
1,400

1,210
1,199
DRUG COUNT

1,080
1,200

1,029
944
1,000

867
809
739
680
800

674
583
599
605
607
594
557
503
516
490

600
468

405
412
399

400

200

0
1996

2006

2016
2000

2010

2020
1995

1998
1999

2002
2003
2004
2005

2008
2009

2012
2013
2014
2015

2018
2019

2022
2023
2024
1997

2007

2017
2001

2011

2021
YEAR
Source: Pharmaprojects®, January 2024

Continued pharmaceutical development growth in Denmark is likely dependent on the 70 companies

currently headquartered in the country, led by the Top 10 companies listed in Table 18. Novo Nordisk
is head and shoulders above the rest, but there are a few other well-known companies based in the
country, including Genmab, Lundbeck, and Leo Pharma.

Table 18: Top 10 Denmark-headquartered

companies by size of pipeline

COMPANY PIPELINE DRUGS

Novo Nordisk 68
Genmab 25
Lundbeck 16
Gubra 15
Bavarian Nordic 10
Leo Pharma 10
Zealand Pharma 10
Evaxion Biotech 8
ALK-Abello 7
Xellia Pharmaceuticals 7

Source: Pharmaprojects®, January 2024

44 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

One would expect the vast majority of developmental therapies to be indicated for various forms
of cancer or alimentary/metabolic diseases, and the data from early 2024 in Denmark confirm
this, with seven of the Top 10 diseases falling into these spaces. With only four cancer indications,
Denmark differs from the global rankings, where eight of the top 10 indications are cancer. The
other two diseases of focus in the global landscape are Alzheimer’s disease and novel coronavirus
infection; however, out of the 589 indications under study in Denmark, Alzheimer’s disease and novel
coronavirus infection rank 34th and 55th, respectively.

Table 19: Denmark’s Top 10 diseases for pipeline drugs

POSITION DRUG DISEASE NUMBER OF DRUGS

1 Cancer, lung, non-small cell 63

2 Cancer, breast 49
3 Cancer, colorectal 39
4 Diabetes, Type 2 38
5 Arthritis, rheumatoid 37
6 Psoriasis 32
7 Arthritis, psoriatic 31
8 Cancer, myeloma 30
9 Colitis, ulcerative 29
10 Crohn's disease 28
Source: Pharmaprojects®, January 2024

45 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 Sweden
From one Scandinavian country to another, we turn our focus to Sweden, which has similar development
metrics to Denmark. In Sweden, there are currently 1,563 active drugs, which is up from 1,467 in 2023.
This represents 6.5% YoY growth, which pales in comparison to the rate seen from 2022 to 2023, which
was 9.2%. However, the past year’s growth is still above the mean growth rate in Sweden since 1996
(5.2%), so drug development is clearly still growing in this market. Much like Denmark, the YoY growth
rates seen in the past two years in Sweden are the country’s highest since 2016.

Figure 25: Total Sweden R&D pipeline size, 1995—2024

1,800

1,563
1,467
1,600

1,309
1,389

1,353
1,351
1,309
1,301
1,263
1,400

1,172
DRUG COUNT

1,112
1,200

1,024
903
1,000

831
769
800

663
618
581

579

563
548

528
529
524

600
511
481
454

416
422
393

400

200

0
1996

2006

2016
2000

2010

2020
1995

1998
1999

2002
2003
2004
2005

2008

2018
2009

2012
2013
2014
2015

2019

2022
2023
2024
1997

2007

2017
2001

2011

2021
YEAR
Source: Pharmaprojects®, January 2024

Currently there are 136 companies headquartered in Sweden that are actively developing drugs.
Table 20 shows the Top 10 of these companies and the number of drugs they have in their current
pipeline. For Sweden to continue growing its pharmaceutical industry and remain productive in the
global development landscape, the 10 companies below will need to continue their recent progress.

Table 20: Top 10 Sweden-headquartered

companies by size of pipeline

COMPANY PIPELINE DRUGS

Ribocure
16
Pharmaceuticals
Anocca 15
Sobi 15
BioArctic 13
Medivir 9
Alligator Bioscience 8
Salipro Biotech 7
AlzeCure Pharma 6
BioInvent 6
Camurus 6
Source: Pharmaprojects®, January 2024

46 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

Sweden also lags in oncology development compared to globally, with only five cancer diseases in
its Top 10. Interestingly, comparing the top pipeline indications between Denmark and Sweden shows
that both countries heavily contribute to the development of therapies for arthritis and psoriasis,
diseases that are at least 10 places lower in the global landscape. The Scandinavian countries appear
to focus more on immunological diseases than many other countries around the world.

Table 21: Sweden’s Top 10 diseases for pipeline drugs

POSITION DRUG DISEASE PIPELINE DRUGS

1 Cancer, lung, non-small cell 62

2 Cancer, breast 50
3 Cancer, colorectal 40
4 Diabetes, Type 2 40
5 Arthritis, rheumatoid 38
6 Cancer, myeloma 35
7 Psoriasis 35
8 Arthritis, psoriatic 32
9 Alzheimer's disease 31
10 Cancer, prostate 30
Source: Pharmaprojects®, January 2024

47 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 France
We next move south to France, which dwarfs both Denmark and Sweden in the total number of
drugs, while also besting both countries in YoY growth, with a rate of 8.1%. The most recent growth
rate is down from 2022—23 (12.2%), but is also still above the global 2023—24 growth rate of 7.2%.
The past year’s growth is almost double the mean YoY growth rate since 1996 (4.3%), and is its
second largest YoY metric since 2016.

Figure 26: France’s R&D pipeline size, 1995—2024

2,365
2,500

2,188
1,951
1,955
1,923
1,911
1,870

1,891
1,869
2,000

1,706
1,589
DRUG COUNT

1,492
1,336
1,500

1,185
1,016
956
949
878

1,000
953
858
859
838
794
791
767

798

785

799
735

688

500

0
1996

2006

2016
2000

2010

2020
1995

1998
1999

2002
2003
2004
2005

2008
2009

2012
2013
2014
2015

2018
2019

2022
2023
2024
1997

2007

2017
2001

2011

2021
YEAR
Source: Pharmaprojects®, January 2024

With a large number of active drugs in the country, one would expect the companies headquartered in
France to have large current pipelines, and that is what we can see in Table 22. With big companies like
Sanofi and Ipsen leading the way, the development landscape in France looks to be in good hands for
future years of continued growth.

Table 22: Top 10 France-headquartered

companies by size of pipeline

COMPANY PIPELINE DRUGS

Sanofi 130
Ipsen 38
Servier 32
TheraVectys 22
Pierre Fabre 18
Valneva 11
Innate Pharma 10
OSE
10
Immunotherapeutics
Vectans Pharma 9
Xenothera 9
Source: Pharmaprojects®, January 2024

48 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

France’s active pipeline is more comparable to the Top 10 targeted indications across the globe than
those of many other countries, reporting nine out of the Top 10 diseases from the oncology space.
Of the nine cancer indications, four are in both the French and global Top 10s: non-small cell lung
cancer, breast cancer, colorectal cancer, and prostate cancer. As for the diseases that round out
the global Top 10, Alzheimer’s disease and novel coronavirus infection developmental drugs are not
nearly as prevalent in France, with Alzheimer’s disease placing 31st and novel coronavirus infection
placing 37th.

Table 23: France’s Top 10 diseases for pipeline drugs

POSITION DRUG DISEASE PIPELINE DRUGS

1 Cancer, lung, non-small cell 134

2 Cancer, breast 89
3 Cancer, colorectal 69
4 Cancer, melanoma 58
5 Cancer, lymphoma, non-Hodgkin's 55
6 Cancer, leukaemia, acute myelogenous 54
7 Cancer, myeloma 46
8 Cancer, prostate 44
9 Cancer, renal 40
10 Colitis, ulcerative 38
Source: Pharmaprojects®, January 2024

49 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 Germany
Since 1995, there has been an overall increasing trend in the total pipeline size in Germany. In
terms of the most recent changes in pipeline size, between 2023 and 2024 the total pipeline size in
Germany rose, with a 5.0% increase in the number of active drugs.

Figure 27: Germany’s R&D pipeline size, 1995—2024

2,479
2,360
2,246
2,207
2,500

2,231
2,188

2,175
2,187
2,151
1,982
1,870
2,000

1,703
1,570
DRUG COUNT

1,423
1,249
1,500

1,078
1,048
958
842

944
845

912
883

876
923
815
811

1,000
793
793

748

500

0
1996

2006

2016
2000

2010

2020
1995

1998

2012
2013
2014
2015

2018
1999

2002
2003
2004
2005

2008
2009

2019

2022
2023
2024
1997

2007

2017
2001

2011

2021
YEAR
Source: Pharmaprojects®, January 2024

There are 143 companies headquartered in Germany. The top 10 companies are listed in Table 24,
alongside the size of their pipelines.

Table 24: Top 10 Germany-headquartered

companies by size of pipeline

COMPANY PIPELINE DRUGS

Boehringer Ingelheim 103

Bayer 91
BioNTech 53
Evotec 49
Merck KGaA 36
CureVac 22
Grunenthal 12
Stada 11
Biotest 10
Medigene 10
Source: Pharmaprojects®, January 2024

50 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

The top three diseases in Germany are non-small cell lung cancer, breast cancer, and colorectal
cancer, which are also the top three drug diseases globally. The Top 10 German indications also
include a number of metabolic, dermatological, and autoimmune indications, such as type 2
diabetes, psoriasis, and rheumatoid arthritis.

Table 25: Germany’s Top 10 diseases for pipeline drugs

POSITION DRUG DISEASE PIPELINE DRUGS

1 Cancer, lung, non-small cell 109

2 Cancer, breast 71
3 Cancer, colorectal 60
4 Diabetes, Type 2 59
5 Psoriasis 46
6 Cancer, myeloma 44
7 Cancer, leukaemia, acute myelogenous 43
8 Arthritis, rheumatoid 41
9 Cancer, lymphoma, non-Hodgkin's 41
10 Cancer, melanoma 40
Source: Pharmaprojects®, January 2024

51 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 Switzerland
Since 1995, there has been an almost tripling of the total pipeline size in Switzerland. Between 2023
and 2024, the total pipeline size in Switzerland rose, with a 6.1% increase in the number of active
drugs this year.

Figure 28: Switzerland’s R&D pipeline size, 1995—2024

1,505
1,600

1,419
1,400

1,232
1,165
1,154

1,127
1,120

1,113
1,089
1,200

1,071
1,035
955
1,000

872
DRUG COUNT

774
800

673
582
591

564
577

540
558

536
537
531
523

519

600
494
464

457
425

400

200

0
1996

2006

2016
2000

2010

2020
1995

1998
1999

2002
2003
2004
2005

2008

2012
2013
2014
2015

2018
2009

2019

2022
2023
2024
1997

2007

2017
2001

2011

2021
YEAR
Source: Pharmaprojects®, January 2024

There are 188 companies headquartered in Switzerland. The Top 10 companies are listed in Table 26,
alongside the size of their pipelines.

Table 26: Top 10 Switzerland-headquartered

companies by size of pipeline

COMPANY PIPELINE DRUGS

Roche30: Top 10 Switzerland-headquartered

Table 217
companies
Novartis by size of pipeline 153
Medicines for Malaria
32
Venture
DNDi 29
CRISPR Therapeutics 23
Debiopharm 18
AC Immune 17
Sandoz 17
Light Chain Bioscience 14
Idorsia Pharmaceuticals 14

Source: Pharmaprojects®, January 2024

52 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

The top three diseases for Switzerland are non-small cell lung cancer, breast cancer, and colorectal
cancer, which are also the top three drug diseases globally. As with the global pipeline, the Swiss
pipeline is dominated by drugs in development for different cancer indications.

Table 27: Switzerland’s Top 10 diseases for pipeline drugs

POSITION DRUG DISEASE NUMBER OF DRUGS

1 Cancer, lung, non-small cell 46

2 Cancer, breast 43
3 Cancer, colorectal 33
4 Cancer, melanoma 32
5 Cancer, brain 27
6 Alzheimer's disease 26
7 Cancer, myeloma 26
8 Diabetes, Type 2 23
9 Infection, malaria 21
10 Cancer, leukaemia, acute myelogenous 19
Source: Pharmaprojects®, January 2024

That concludes our whistle-stop

review of the R&D climates across
a range of countries with significant
pharma industries. It suggests
that while some territories are
experiencing fair weather, for others
the skies are darkening somewhat.
It also demonstrates how different
countries have different emphases
in terms of their disease focuses, as
every company continues to search
for the pot of gold at the end of
the rainbow.

53 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 Mechanisms and Targets
Immuno-oncology makes hay while the sun shines
What makes weather work? The Earth’s climate system is complex, but a small change
to temperature, atmospheric pressure, cloud formation, wind, humidity, or rain patterns
can have a significant effect on the development of weather systems. It is the interaction
of all these different components, in an environment where chaos theory also plays
its part, that produces our weather in all its infinite variety. Everything is very finely
balanced, hence how an external force, for example human interventions such as
the release of greenhouses gases into the atmosphere, can perturb the status quo,
potentially irrevocably.

What makes drugs work is the subject of Beginning with mechanisms, Table 28
this section of our report, which looks at lists the current Top 25 mechanisms in
drugs’ mechanisms of action and their Citeline’s proprietary mechanism of action
targets. It’s a similarly complex ecosystem. classification. It’s important to note that
Just as we can model the weather to this is hierarchical, one effect of which is to
get predictions of the outcome of what’s make the broader terms at the top of the
happening now, we can predict from drugs’ hierarchy crop up most commonly. This is
mechanisms and targets what will happen because, with over half of the pipeline still
when they are administered, but both are at the preclinical phase, where often full
still incredibly inexact sciences. The whole mechanistic information is yet unknown
point of the drug R&D process is to test or undisclosed, there tend to be a lot of
hypotheses and gather evidence, so that drugs where only a broad mechanistic
we can have confidence in the outcome of class can be ascribed. As drugs move up
taking a drug, just like we want to be able through development stages, these general
to trust our weather forecasts. categorizations are often replaced by
something more precise. There are also
a number of “umbrella” terms, created to
permit searching across mechanisms in
certain broader categories. The immuno-
oncology (IO) category is one such of these
and is applied to drugs in the IO class even
if more specific mechanistic information
is also known, so that all IO drugs can be
found in a single search. This is one of the
reasons that it is applied to so many drugs.
These caveats should be borne in mind
when examining the Top 25 terms.

54 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

Table 28: Top 25 mechanisms of action (pharmacologies)
POSITION
NUMBER OF DRUGS 2024 % AT PR, R
2024 MECHANISM OF ACTION TREND
(2023) OR L
(2023)
1 (1) Immuno-oncology therapy 3,932 (3,393) 2.7 ↑↑

2 (2) Immunostimulant 1,812 (1,472) 10.3 ↑

3 (3) T cell stimulant 1,148 (1,091) 1.6 ↔

4 (4) Immune checkpoint inhibitor 986 (618) 4.0 ↑↑

5 (5) Gene expression inhibitor 354 (283) 1.7 ↑

6 (15) Immune checkpoint stimulant 303 (150) 1.3 ↑↑

7 (7) Protein degrader 292 (221) 1.0 ↑

8 (6) Genome editing 290 (274) 0.3 ↔

9 (9) Radiopharmaceutical 277 (192) 7.2 ↑↑

10 (11) Natural killer cell stimulant 223 (186) 0 ↑

11 (10) Angiogenesis inhibitor 192 (192) 28.1 ↔

12 (8) CD3 agonist 190 (198) 5.3 ↓

13 (12) PD-L1 antagonist 178 (181) 6.2 ↓

14 (13) Immunosuppressant 174 (179) 38.5 ↓

Microbiome modulator, live ↑

15 (17) 167 (128) 1.2
microorganisms
16 (14) PD-1 antagonist 165 (152) 12.7 ↔

Glucagon-like peptide 1 receptor ↑

17 (18) 156 (111) 5.8
agonist
Vascular endothelial growth factor ↔
18 (16) 152 (142) 26.3
receptor antagonist
19 (352) Ubiquitin ligase E3 stimulant 125 (11) 0 ↑↑

20 (21) K-Ras inhibitor 108 (87) 2.8 ↑

21 (31) DNA topoisomerase I inhibitor 98 (67) 6.1 ↑

22 (19) Apoptosis stimulant 90 (103) 14.4 ↓

23 (34) Tubulin inhibitor 89 (62) 7.9 ↑

24 (24) DNA inhibitor 87 (82) 32.2 ↔

25 (20) ErbB-2 antagonist 87 (97) 20.7 ↓

Abbreviations used in table: PR = pre-registration R = registered L = launched

Source: Pharmaprojects®, January 2024

55 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

This certainly helps to explain the table’s number But there is also an internal reason for the
1 category being IO therapy. There are now 3,932 stratospheric increase, as a review of such
drugs that fall into that bucket, meaning more drugs led to us adding this mechanism to quite
than one in six of all drugs in development can a few existing PROTAC drugs which didn’t have
be categorized in this way — a striking statistic this assigned before. But despite the ongoing
considering how relatively new this technique, interest in protein degradation and E3 ligase
whereby the body’s own immune system signaling, development remains firmly rooted
is trained to attack cancer cells, is. Further at the preclinical to early clinical stage. To
evidence of how the pharmaceutical industry is date, there are only six protein degrader drugs
boldly placing a lot of faith in this strategy can approved worldwide, with just two new launches
be seen by looking at the column to the right in 2023. Another big increase within the Top 10
of the number of drugs column in this table: the was in radiopharmaceuticals, which recorded a
percentage of drugs using this strategy which 44% rise, with imaging agents making up over
have made it to the later stages (pre-registration, half of this increase.
registered, or launched) of development. Only
2.7% of drugs in this category have made it this There was stagnation in the development
far, so the overwhelming majority of candidates of some of the more traditional anticancer
are still in clinical or preclinical studies. As more cytotoxic drugs, such as DNA intercalators,
products do progress, though, this is looking tubulin binding agents, and angiogenesis
less like an enormous leap of faith and more like inhibitors. This is perhaps indicative of the
an exciting new front. With a remarkable 15.9% continued push towards targeted therapy as a
increase in candidates, this looks to be one area mainstay, with little movement in this area since
where it never rains but it pours. last year’s report. One of the elders of targeted
therapy, ErbB-2 antagonists, also took a hit,
One of the subgroups of IO therapy, immune with the number of development candidates
checkpoint inhibitors, reports a nothing less actually falling. Elsewhere, the continued rise
than stupendous 59.5% growth in pipeline size. of antibody-drug conjugates produced an
This category embraces a plethora of different increase in DNA topoisomerase I-targeting drug
individual categories, including some which candidates, with TOP1 representing an attractive
appear under their own right in the top 25, such target for antibody-drug conjugate warheads.
as PD-L1 and PD-1 antagonists. Even more
dramatically, the size of the pipeline of immune
checkpoint stimulants has more than doubled.
This can be partly accounted for by the fact
that, during the course of last year, we created
a new Target Family to encompass targets in
this area, which led to a cleanup of the data and
consequently more drugs being assigned this
mechanism of action.

Two other broad IO categories involved in cell

therapy, T cell stimulants and natural killer cell
stimulants, feature, although their progress has
somewhat stalled this year. Overall, the field of
immuno-oncology is keenly represented, as it
continues to have its moment in the sun.

The development of targeted protein degraders

also saw a noticeable increase in 2023, with
ubiquitin E3 ligase being the most popular
target for these novel therapeutic agents. Keen
observers will note an extraordinary rise in the
number of ubiquitin ligase E3 stimulants in
this year’s Top 25, the explanation for which
is twofold. First, there were a huge number
of protein degraders (around 60) using this
mechanism entering development over the
past 12 months.

56 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

Immuno-oncology makes its presence similarly felt in Table 29, where we look at the Top 25
individual protein targets which today’s pipeline drugs are hitting. Here, there has been a change
at the top, with CD3e, a target heavily used by CAR-T therapeutics, returning to the pole position.
It deposes another IO-based target, PD-L1, down to number 3, while a more traditional anticancer
target, Her2, moves up to number 2, so IO is not having everything its own way. Another IO-based
target involved in CAR-T, CD19, also drops down the top five.

Table 29: Top 25 drug protein targets

POSITION
NUMBER OF DRUGS
2024 TARGET TREND
2024 (2023)
(2023)
1 (2) CD3 epsilon subunit of T-cell receptor complex 223 (207) ↔

2 (4) erb-b2 receptor tyrosine kinase 2 [Her2] 217 (187) ↔

3 (1) CD274 molecule [PD-L1] 211 (210) ↔

4 (5) epidermal growth factor receptor 205 (178) ↑

5 (3) CD19 molecule 199 (194) ↔

6 (6) programmed cell death 1 [PD-1] 194 (165) ↑

7 (8) glucagon like peptide 1 receptor 169 (126) ↑

8 (7) vascular endothelial growth factor A 168 (160) ↔

9 (9) KRAS proto-oncogene, GTPase [K-Ras] 133 (118) ↔

10 (10) 5-hydroxytryptamine receptor 2A 127 (117) ↔

11 (11) opioid receptor mu 1 104 (107) ↓

12 (12) insulin receptor 97 (87) ↔

13 (14) cannabinoid receptor 1 94 (86) ↔

14 (13) TNF receptor superfamily member 17 [BCMA] 94 (87) ↔

nuclear receptor subfamily 3 group C member 1 ↔

15 (16) 91 (84)
[glucocorticoid receptor]
16 (18) membrane spanning 4-domains A1 [CD20] 90 (82) ↔

17 (16) TNF receptor superfamily member 9 [CD137] 88 (84) ↔

18 (21) claudin 18 87 (75) ↔

19 (15) tumor necrosis factor 87 (86) ↔

20 (22) androgen receptor 76 (75) ↔

21 (23) transforming growth factor beta 1 76 (74) ↔

22 (19) opioid receptor kappa 1 75 (80) ↓

23 (24) CD47 molecule 74 (73) ↔

24 (26) kinase insert domain receptor 73 (69) ↔

25 (25) prostaglandin-endoperoxide synthase 2 72 (73) ↓

Note: NCBI names are used, except for additions in italics made by us for clarity.
Source: Pharmaprojects®, January 2024

57 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 Outside of the Top 10, the air is stiller this year, development does continue to expand, however,
with few dramatic changes, aside from the with the total for 2024 hitting 2,035, up from 1,974
continued decline of the opioid targets mu1 and the year before, with a bigger increase than that
kappa1. Conspicuous by its absence this year is seen from 2022 to 2023. We are clearly a long
the COVID-19 spike protein, which drops out of way still from there being nothing new under
the upper echelons to number 31. It might have the sun.
appeared in 2021’s chart like a bolt from the blue,
but its decline has been similarly lightning fast.

The lifeblood of the industry, though, has to be

the continued identification of novel targets for
drug development, showers of which are needed
every year to prevent the drug development
landscape rapidly becoming an arid desert.
Figure 29 records that the number of newly
identified drug targets in 2023 was very similar
to that seen in 2022, at 93. This paints the year
as another one very slightly below the par of 100
per year. The universe of targets for active drug

Figure 29: Number of new drug protein targets identified by Pharmaprojects, by year

200

179
180

160

140 139
NUMBER OF NEW TARGETS

131

120 116
113
107
100 102
100 97 97
93 94 89 92

80 77
75
72
68
67

60

40

20

0
2023 2022 2021 2020 2019 2018 2017 2016 2015 2014 2013 2012 2011 2010 2009 2008 2007 2006 2005

YEAR
Source: Pharmaprojects®, January 2024

Famously, no two snowflakes are alike. While ways to treat diseases and targets for drug development
will presumably not be similarly infinite, it seems likely that we may have barely scratched the surface
of the myriad ways in which science may yet treat the burden of disease. Let’s hope that the flurry of
new targets identified, and new drugs produced, can turn into a blizzard.

58 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 Types of Pipeline Drugs
Is the continued move into biotech
starting to meet a frosty reception?

Most experts believe that climate change is rapidly approaching an inflection point. If action
is not taken now, we might reach the point of no return, whereby we lose the ability to control
or reverse the changes happening to our world’s weather. Last year, scientists from the British
Antarctic Survey reported that ice melt in West Antarctica has accelerated and is now inevitable
for the rest of the century, no matter by how much carbon emissions are cut. If this ice sheet is lost
completely, it would raise global sea levels by five meters, leading to many coastal cities having to
be abandoned. However, it is believed that if we act now, we can at least save the East Antarctic
ice sheet, which has the potential to cause sea levels to rise by as much as 10 times that amount.

A change in pharma that also has seemed to be Figure 30 therefore makes interesting reading.
hitting a tipping point — although by no means It now looks like there is clear evidence that
a similarly negative trend — is the ongoing move the ongoing march into biotech has indeed
away from traditional small molecule drug R&D plateaued. The overall proportion of biotech
to that based on biotechnological techniques. drugs this year stands at 44.2%, up just 0.2pp
There were signs last year that this too might be on last year, and still 0.5pp below where it was
hitting an inflection, and that the disappearance in 2022. It does appear that, after many years
of large chunks of chemistry-based drug of biotechnology drugs taking an increasingly
discovery might be slowing. This is one data large slice of the pie, a set point might have
point we will address in this last section of this been reached.
year’s report, which looks at the types of
drugs under development.

Figure 30: Biological vs. non-biological drugs as a percentage of the pipeline, 1995—2024
100%

90%

80%
% OF PIPELINE

70%

60%
Non-Biotech
50%
Biotech
40%

30%

20%

10%

0%
1996

2006

2016
2000

2010

2020
1995

1998
1999

2002
2003
2004
2005

2008
2009

2012
2013
2014
2015

2018
2019

2022
2023
2024
1997

2007

2017
2001

2011

2021

YEAR
Source: Pharmaprojects®, January 2024

59 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

We can break this down further in Table 30, which gives data on the number of active drugs assigned
to each of Citeline’s individual drug type categories, referred to as origins as they indicate how the
drug was originally produced. Here is further confirmation that predictions of the demise of the small
molecule may well have been premature. Synthetic chemicals still top this chart, and in fact post
a 5.1% increase on last year, although admittedly this is a smaller percentage increase than 2023’s
7.8%. The next five categories provide some insight into the most common types of biotechnology-
based candidates. This kicks off with antibodies at number 2, posting a heftier 11.1% rise in numbers,
followed by four categories whose numbers report little change: recombinant proteins, autologous
cell therapies, virally delivered nucleic acids, and heterologous cell therapies. Note that the origin
classification is another hierarchical one, hence the category for cell therapies whose type is
unknown appearing at number 7.

Table 30: Top 25 origins of pipeline drugs

POSITION
NO. OF DRUGS IN PIPE-
2024 ORIGIN TREND
LINE 2024 (2023)
(2023)
1 (1) Chemical, synthetic 10,831 (10,307) ↑

2 (2) Biological, protein, antibody 3,038 (2,734) ↑

3 (3) Biological, protein, recombinant 957 (932) ↔

4 (4) Biological, cellular, autologous 722 (758) ↓

5 (6) Biological, nucleic acid, viral vector 716 (677) ↔

6 (5) Biological, cellular, heterologous 695 (687) ↔

7 (7) Biological, cellular 688 (583) ↑↑

8 (8) Chemical, synthetic, nucleic acid 612 (536) ↑

9 (10) Biological, protein 558 (523) ↔

10 (9) Biological, virus particles 535 (534) ↔

11 (11) Biological, nucleic acid 493 (477) ↔

12 (12) Chemical, synthetic, peptide 491 (461) ↔

13 (13) Biological, other 392 (358) ↔

14 (14) Biological, bacterial cells 339 (330) ↔

15 (15) Biological, peptide 272 (254) ↔

16 (17) Biological, nucleic acid, non-viral vector 266 (185) ↑

17 (19) Biological 251 (164) ↑

18 (16) Natural product, plant 218 (215) ↔

19 (18) Biological, peptide, recombinant 176 (171) ↔

20 (21) Natural product, fungal 58 (51) ↔

21 (20) Chemical, semisynthetic 53 (57) ↔

22 (22) Natural product, bacterial 46 (50) ↔

23 (23) Natural product 38 (38) ↔

24 (25) Chemical, synthetic, isomeric 21 (21) ↔

25 (24) Natural product, animal 20 (22) ↔

Source: Pharmaprojects®, January 2024

60 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

In a table that, as usual, doesn’t exhibit much structural change, there are a couple of increases
for individual categories that are worth calling out. Nucleic acids produced by synthetic chemistry
rise by 14.2% at number 8, while biologically produced nucleic acids delivered via non-viral means
are up by an impressive 43.8% at number 16. It’s also interesting to note how little natural product
development contributes to the R&D pipeline now, with just 1.7% of current candidates being sourced
this way.

Despite the stasis of this table, there is a bit of movement in the related metrics on drug delivery
route, as evidenced in Figure 31. The proportion of drugs administered by injection is up a bit this
year, with a concomitant fall in those delivered orally. Taken together, these delivery types cover 90%
of all pipeline drugs, with topical and inhaled preparations constituting 3% each, and other routes
even smaller percentages.

Figure 31: Pipeline by delivery route, 2023 and 2024

1% 0%
0%
0%
Vaginal
3%

Transdermal

Topical
2023 29%

61% Rectal

Otic
Injectable
2%
3% Implant
Oral
1% Inhaled

Ophthalmological
Ophthalmological
Oral

0%
0%
0% 1% Otic
Vaginal
Inhaled
3%
Rectal

Topical
Transdermal
Implant
Transdermal

Vaginal Topical
Injectable
26%
2024
Rectal
64%

2%
Otic
3%

1%
Oral

Ophthalmological
Source: Pharmaprojects®, January 2024

Inhaled

61 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 What’s the Forecast for Pharma?
Plenty of rays of sunshine should hold off any storm
clouds on the horizon
Since I began compiling this report at 2023 was the “hottest” year for pharma
the beginning of January 2024, it’s been R&D in terms of the number of drugs in
officially confirmed that 2023 was the development too, and I think we can make
hottest year on record for planet Earth. a similar prediction that 2024 is extremely
The average land and ocean surface likely to be even hotter still. The trend here,
temperature during the year was 1.18°C like the temperature, is heading inexorably
(2.12°F) above the mean for the 20th upwards. But, just as climate change might
century, making it the highest global mean a nice warm summer and a mild
temperature among all years in the US winter for some, but wildfires and floods for
National Oceanographic and Atmospheric others, bigger drug pipelines are similarly
Administration’s (NOAA’s) climate records, a mixed blessing. They are signs that the
which began in 1850. In doing so, it industry is continuing to grow and that
beat the next warmest year, 2016. More there is money available for investment,
alarmingly, the 10 warmest years since but as the vast majority of drugs will still
1850 have all occurred in the past decade. fail, it could be said that ever-increasing
The NOAA estimates that the chances of amounts of capital are being spent for no
2024 being in the top five warmest years return. While the last few years have seen
ever are 99%. some very encouraging signs in terms of
the numbers of new drugs making it onto
the market, 2023’s data on this are still
pending at the time of writing, and whether
there is an overall upward trend is at this
point moot. Certainly, the fact that a lower
percentage of drugs in Phase II seem to be
making it into Phase III must be a worrying
statistic. Drug development will always be
an inherently risky undertaking, but the
chances of success ought to be getting
better, not worse.

62 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 on further, it has been somewhat eclipsed by
the ghastly events of the Israel-Gaza conflict
that erupted in early October. At the time of
writing, no resolution is in sight, with both sides
entrenched in a whirlwind of horror. Thankfully,
as yet, this hasn’t expanded into a hurricane
of regional conflict, although recent events in
Yemen and across the Iran-Pakistan border
are a chilling reminder that this remains a
possibility. With the Middle East a major source
of the world’s oil, there is the potential that
rising oil prices could again put the kibosh on
a recovering economic picture. Maybe climate
change isn’t the only reason why we should be
weaning ourselves off fossil fuels.

And as we go into another US election year,

we can expect more instability in the political
atmosphere, which is likely to be exacerbated
I reflected last year on how well the industry had by a bruising increase in the temperature of
done to not only just continue doing what it does the rhetoric of the culture wars. The result of
during the COVID-19 pandemic, but also how it November’s poll could seriously affect the
significantly contributed to society moving past direction of travel of the US in multiple fields, not
it, and to use it as a springboard to supercharge least in respect of its relationship with China. We
advances. The use of mRNA vaccine technology have seen in this report just how much China has
comes to mind particularly here. To mix a range emerged as a global player in pharma R&D, and
of weather-based idioms, the industry had been it’s not done yet. However, “business in China is
cooking up a storm of innovation for some time, going through a period of uncertainty,” pointed
and when the clouds of COVID gathered, and out Helen Chen, Greater China managing
we were suddenly all snowed in, this definitely partner and Asia healthcare sector head at
enabled us to see the sunshine after the rain L.E.K. Consulting, in an article of our sister
sooner. Many countries around the world are publication Scrip. “Distractions are many,” she
now in the post-match analysis phase, holding continued, “a relatively low 5.5% GDP growth,
scientists, administrators, and politicians alike political tensions with the US, pricing pressures
to account for what we can learn so that we and the hospital anti-corruption campaigns.”
can handle the (inevitable) next pandemic Meanwhile, there is still the potential that out
better, as many parts of the world were woefully to sea, a storm could brew over control of the
underprepared. We should all agree that pharma disputed territory of Taiwan.
was a rare ray of sunshine here. It also emerged
with its reputation enhanced, even if it is still not
considered to be as pure as the driven snow.

Forecasting the fortunes of pharma can be as

tricky as predicting next week’s weather, as a
COVID-style tempest can always wind up out
of nowhere. However, we can certainly look at
how external factors have the potential to blow
things off course. I always make note of the
global geopolitical outlook in my conclusion, as,
just like how El Niño affects weather thousands
of miles away, the pharma industry is always
buffeted by world events, no matter how well it
is insulated from their worst effects, like donning
a duffel coat on a blustery day. Unfortunately,
despite the sun peeping out as the clouds from
COVID disperse, the outlook became grimmer
during 2023. While the war in Ukraine grinds

63 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

All the above feeds into a funding environment widely reported, actual launches are less so,
for pharma that remains problematic. While this so our team undertakes major research to
affects big pharma to a lesser degree, it could present you with a cast-iron list of products that
be a major source of disquiet for smaller biotech genuinely received their actual market debut
or start-up companies, which, as we have during the year). Looking at the figures it looks
seen, are legion. “The funding environment for like 2023 has surpassed 2022’s number. But
biopharma start-ups in 2024 is anticipated to be check back in April to grab a copy of this
generally challenging,” noted Koji Tamada, CEO report to be sure and to incorporate this
of Japanese start-up Noile-Immune Biotech, missing piece of the jigsaw.
also in Scrip. Michael Salako, investment director
at UK life science venture capital company Start So, the pharmaceutical industry has entered
Codon, concurred: “Signs are that the funding 2024 snowed under by a plethora of new
environment for 2024 is generally going to be a drug candidates but seems to continue to
difficult one,” he said. “If the US Federal Reserve blow hot and cold during the clinical stages
starts to reduce interest rates, this would enable of development. Thus, it can’t be said to be
capital to be moved out of risk-free money on cloud nine just yet. A continuing rising tide
market funds and into potentially higher- of new drug delivery to the market is vital if
yielding, riskier asset classes such as venture occasional sunny periods are to give way to
capital. However, this kind of change takes time there being not a cloud in the sky. As with
to trickle down to those seeking funding. I’d climate change, whether it is pleasant tomorrow
expect any benefit from a change like this to is largely immaterial — it’s the longer-term
only be felt by companies towards the end of trends that matter, and will be something we
2024 or early in 2025,” he stated. will continue to analyze. Come rain or shine,
the Citeline Pharma R&D Review will be back
What about the environment for deals? Another next year to take the industry’s temperature
of our sister publications, Biomedtracker, once again.
recorded 2,218 pharma deals in 2023, including
1,237 financing deals. These numbers were
down on their 2022 equivalents of 2,600 and
1,340, respectively, and continued an ongoing
downward trend. But here, the mood from
industry commentators was notably more
optimistic. “I’m convinced that 2024 is the
turning point for the biotech sector,” said Cary
Claiborne, CEO of Adial Pharmaceuticals, in
Scrip. “Low valuations may initiate a flurry of
acquisition activity, becoming an opportunity
during the harsh conditions.” Issei Tsukamoto,
senior vice president, head of business
development at Astellas Pharma, concurred,
noting, “While many faced a challenging year in
deal-making, there is good cause for optimism
in 2024. M&A and financing activities in biotech
sector are returning, and we can hope for a
more ‘normal’ if not quite a ‘boom’ in activity.”

One way to get a better sense of which way the “The funding environment
wind is blowing is to look at industry output in for biopharma start-ups
terms of new drug launches. This is a topic we’ll
be diving into in more detail in the forthcoming in 2024 is anticipated to
companion volume to this report, the NAS
Supplement, which reports on the full list of new
be generally challenging…”
active substances that made it onto the sunlit
uplands of commercialization during the year Koji Tamada,
just past. At the time of writing, mid-January CEO of Japanese start-up
Noile-Immune Biotech
2024, there is still some work to do to finalize
2023’s list (while drug approvals are usually

64 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline

 About
the Author
Ian Lloyd
Senior Director, Pharmaprojects
& Data Integration

Ian Lloyd is the Senior Director of Pharmaprojects

and Data Integration, overseeing the content and
analyst services for our drug development solution.
He supports clients in their drug pipeline data
requirements and inquiries, providing insight into
the best search strategies to answer their drug-
related business questions and also identifying
and analyzing trends in pharma R&D. For the past
32 years, he has authored the “Pharma Annual
R&D Review” and its new active substances (NAS)
launches supplement. This has become a must-
have industry report for those seeking to identify
the changing fortunes of drug R&D. Ian joined
Pharmaprojects in 1987, when it was part of PJB
Publications. It was acquired by Informa in 2003,
and spun out to form Citeline, now part of the
Norstella group, in 2022. He previously worked in
molecular biology as a research assistant at the
University of Bristol.

This reports also contains contributions from the

following analysts in the Pharmaprojects team:
Jonathan Stephens, Sydney Enokawa, Tollan Bell,
Abdirazak Mohamed, JT Toebbe, and Arbesa Bela.

65 Pharma R&D Annual Review 2024 Copyright © 2024 Citeline