"THE STUDY ON DRUG DISCOVERY, [CPCSEA] GUIDELINES OF LABORATORY ANIMALS AND TYPES OF

ANIMAL MODELS USED IN PRECLINICAL RESEARCH”

Report submitted to
Faculty of Pharmacy
PALAMURU UNIVERSITY
MAHABUBNAGAR – 509 001

Submitted by:
[Link] BEGUM - 353122881017

2.A ANUSHA - 353122881019

3.K. RAJA NARSIMHA REDDY - 353122881034
[Link]. RAHMAN - 353122881049
5.V. SANDHYA - 353122881061
Under the guidance of
Mrs. [Link] [Link].,

Assistant Professor
DHANVANTHRI COLLEGE OF PHARMACEUTICAL SCIENCES
THIRUMALA HILLS , MAHABUBNAGAR – 509001 DECEMBER-2025
 DRUG DISCOVERY AND
DRUG DEVELOPMENTAL
PHASES
CONTENT :
• Drug Discovery
• Characterization of Investigational drugs
• Formulation of Investigational drugs
• Pharmacokinetics aspects and Drug
Disposition
• Preclinical Toxicity studies and Investigational
New Drug Application
• Bioanalytical Testing and Clinical Trials
 DRUG DISCOVERY :
• Drug discovery is the scientific process of identifiying new candidate of medicines and developing them
into safe and effective therapeutic agents. It integrates biology, chemistry, pharmacology, biotechnology,
computational science and clinical trials .
1. HISTORICAL BACKGROUND :
• Traditionally, drugs were discovered from natural sources ( plants , microbes , marine organisms).
• Example : Penicillin (from penicillium fungus) , Aspirin (from willow bark).
2. MAJOR STEPS IN DRUG DISCOVERY PROCESS :
• Step -1 : Targets identification and validation
Technique used :
1. Gemonic & Proteomics
2. RNA interference
3. CRISPR gene editing
Examples : HIV Treatment and Reverse Transcriptase
 • Step -2 : Hit Identification
• Step -3 : Hit - to - Lead Development
• Step -4 : Lead Optimization
• Step -5 : Preclinical studies
• Step -6 : Clinical Trials

3. MODERN APPROACHES IN DRUG DISCOVERY :

• Advancement in science and technology
have transformed drug discovery
• Rational drug design
• Artificial intelligence
4. CHALLENGES IN DRUG DISCOVERY :
• High cost
• Long timeline
• High failure rate
• Safety and ethical concern in clinical testing.
CHARACTERIZATION OF INVESTIGATIONAL DRUGS :
• This is a critical step between discovery and clinical trials ensure the new molecule is safe, stable
and well understood before it is given to human.
1. PHYSICOCHEMICAL CHARACTERIZATION :
• Define the Intrinsic, chemical and physical nature of the investigational drugs like
formulation, stability, solubility, bioavailability, molecular purity and partition coefficient.

2. PHARMACOLOGICAL CHARACTERIZATION :
• This evaluate the therapeutic potential of the investigational drugs.
1. MOA
2. Potency and efficacy
3. Dose response relationship
3. PRE- FORMULATION STUDIES
• It focus on Excipients compatibility, solubility enhancement and stability studies in formula.
4. ADME STUDIES :
• Absorption, Distribution, Metabolism in phase – I, II, III , Excretion.
5. TOXICOLOGY CHARACTERS :
• Acute, sub – acute, chronic and sub- chronic
• Genotoxicity and carcinogenicity.
6. IMMUNOLOGICAL CHARACTERIZATION:
• Immunological characterization include:
1. Immunogenicity testing
2. Innate immune response
3. Adaptive immune response
7. BIOPHARMACEUTICAL CHARACTERIZATION :
• Biopharmaceutical properties describes how the drug behaves in biological
systems
1. Dissolution and Disintegration studies
2. Permeability testing
3. Excipients interaction studies
8. REGULATORY AND QUALITY CHARACTERIZATION :
• Must comply with ICH , FDA, EMA guidelines
• Requires GMP and GLP
FORMULATION OF INVESTIGATIONAL DRUGS :
• Formulation of an investigational drug refers to the process of designing and preparing a suitable dosage form of a new
drug candidate (API – Active Pharmaceutical Ingredient) so that it can be safely and effectively administered during
preclinical and clinical studies.
1. Objectives of Formulation
2. Factors Affecting Formulation
Dose consideration
Route of administration
3. Formulation Development Stages
(a) Preclinical Formulation Development
(b) First-in-Human (Phase I) Formulation
(c) Phase II & III Formulation
(d) Final Market Formulation
4. Common Formulation Approaches
1. For poorly soluble drugs:
2. For unstable drugs:
3. For peptides/biologics:
4. For oral drugs:
5. For parenteral drugs:
5. Packaging and Labeling of Investigational Drugs
6. Regulatory Considerations
PHARMACOKINETICS ASPECTS AND DRUG DISPOSITION:
1. Introduction:
• Pharmacokinetics (PK) is the quantitative study of how the body affects a drug after
administration. It describes the time course of (ADME) of drugs. These processes determine the
concentration of a drug in plasma and tissues,
2. Pharmacokinetic Phases
• Pharmacokinetics is broadly divided into four phases:
a) Absorption
b) Distribution
c) Metabolism (Biotransformation):
Phase I (Functionalization reactions): oxidation, reduction, hydrolysis.
Phase II (Conjugation reactions): glucuronidation, sulfation, acetylation, methylation
d) Excretion
DRUG DISPOSITION :
1. Definition Drug disposition refers to the fate of a drug in the body after
absorption, mainly involving distribution, metabolism, and excretion.
2. Components of Drug Disposition
a) Distribution
b) Metabolism (Biotransformation)
c) Excretion
3. Pharmacokinetic Parameters in Drug Disposition
4. Clinical Importance
 PRECLINICAL TOXICITY STUDIES AND
INVESTIGATIONAL NEW DRUG [IND] APPLICATION :
PRECLINICAL TOXICITY STUDIES :
• Before a drug can be tested in humans , it must undergo preclinical studies to assess it’s safety profile.

1. OBJECTIVES :
• To identify potential toxic effects of the investigational drugs.
• To identify target organs of Toxicity
2. TYPES OF PRECLINICAL TOXICITY STUDIES :
• Acute Toxicity studies
• Sub acute Toxicity studies
• Chronic Toxicity studies
• Genotoxicity studies
• Carcinogenicity studies
• Reproductive and developmental Toxicity
3. TYPICAL TEST SYSTEMS :
• In vitro (cell cultures, genetic assays )
• In vivo (at least two animals species :one rodent, one non – rodent e.g. Rat and dog)
INVESTIGATIONAL NEW DRUG (IND) APPLICATION :
• The IND application is a formal request to regulatory authorities ( e.g. FDA in the US, CDSCO in India ) to begin clinical trials in humans.
• MAIN COMPARTMENTS OF IND :
1. PRECLINICAL DATA :
• Pharmacology
• Toxicology
2. CHEMISTRY, MANUFACTURING AND CONTROLS :
• Information on drug composition, purity, stability, formulation and manufacturing.
3. CLINICAL PROTOCALS AND INVESTIGATOR INFORMATION :
• Qualification of clinical investigators
• Safety monitoring plans
4. ADMINISTRATIVE INFORMATION :
• Cover sheet , Sponsor information ,Commitments to follow regulations .
• PURPOSE OF IND :
• Protect the rights and safety of human subjects.
• Ensure that risks are minimized
• Allow regulatory. Review before exposing humans to the drugs.
 BIOANALYTICAL TESTING AND CLINICAL TRIALS :
• BIOANALYTICAL TESTING :
• Bioanalytical testing refers to a set of laboratory methods used to quantify drugs , metabolites or biomarkers in biological samples
(blood, plasma, urine tissues, saliva etc. ) using validated analytical techniques.
1. OBJECTIVE :
• To determine drug concentrations in biological fluids.
• To support pharmacokinetics and pharmacodynamics studies.
2. TECHNIQUES USED :
• Chromatographic methods
• Immunological methods
• Molecular methods
3. KEY PARAMETERS :
• Specificity & selectivity ( ability to measure analyte without interference)
• Sensitivity ( LOD/LOQ) ( lowest detectable / quantifiable concentration)
4. APPLICATION S :
• Preclinical studies
• Clinical studies
CLINICAL TRIALS :
• Clinical trials are systematic investigations in humans to assess safety, efficacy, dosing and outcomes of investigational
vaccines or medical devices.
PHASES OF CLINICAL TRIALS :

CONCLUSION :
• Bioanalytical testing and clinical trials are interdependent components of drug development. Bioanalytical ensures
accurate measurement of drug and biomarkers levels while clinical trials evaluate the therapeutic outcomes of humans.
Together they provide the scientific and regulatory foundation for bringing safe and effective drug to market .
REFERENCE :
• PHARMACOLOGY TEXT BOOK BY M. M.
DALE AND P. K . MOORE…
… THANK YOU …