The document summarizes the key steps in the drug discovery and development process. It begins with target identification and validation to find potential drug targets. High throughput screening is then used to identify initial hit compounds. Hits undergo further testing including assays and screening to identify lead compounds. Lead optimization is used to improve potency and safety. If leads are promising, they undergo pre-clinical animal testing before human clinical trials. Clinical trials involve 4 phases to test safety, efficacy, and dosing in humans. If successful, the drug sponsor submits a New Drug Application to the FDA for review and potential approval.

1. Drug
Discovery

2.  Target Identification And Validation
 Target identification finds a gene or protein (therapeutic agent) that plays
a significant role in disease
 To validate targets, researchers use modern tools and techniques such as
disease association, bioactive molecules, cell-based models, protein
interactions, signaling pathways analysis, functional analysis of genes, in
vitro genetic manipulation, antibodies, and chemical genomics

3. High Throughput Screening
The initial point can be found by screening a large number of molecules that can interact with
a prospective target after it has been identified and validated.

4. Hit Identification and Discovery
The hit is defined as a molecule that must interact with the target to result in a desired therapeutic effect
High Content Screening, phenotypic screening, fragment-based screening, structure-based screening, and virtual
screening
Assay Development and Screening
High Content Screening (HCS) is used for secondary substance screening, lead optimization, substance profiling,
identification and validation of effective targets, investigations in the area of ADME (absorption, distribution,
metabolism, and excretion), and toxicity studies
Hit-To-Lead (H2L)
In the early stages of drug discovery, the Hit-To-Lead (H2L) stage is one of the most crucial. Finding the right leads to
advance along the pathway to a final clinically active medicine is the primary goal of the H2L.

5. Lead Optimization
In the lead optimization (LO) process, the lead compounds discovered
are synthesized and modified to improve potency and reduce side
effects
Active Pharmaceutical Ingredients
Active pharmaceutical ingredients (APIs) are biologically active
ingredients in a drug that produce desired effects

6. Drug Development
Process
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8. In vivo
In vitro
Pre Clinical
• In vitro is another Latin term meaning “in the
glass.
• Organic cells are transplanted into a testing
vessel that acts as an artificial testing
environment
• Focuses on how a compound interacts at
the cellular level
• In vivo is a Latin term meaning “in the whole
body,”
• It requires active testing on live test subjects.
Today, murine (mice or related rodent) models are
the most common test subjects for early-phase
pharmaceutical and biomedical research.
• Primarily focuses on the pharmacokinetics of a
compound in a living organism with a focus on
drug metabolism, safety, and efficacy

9. PHARMACODYNAMICS
1. Pharmacodynamics is the study of a drug's molecular, biochemical,
and physiologic effects, action, and how it affects the organism
"pharmakon" means "drug" and "dynamikos" means "power."
2. All drugs produce their effects by interacting with biological
structures or targets at the molecular level.
3. It determines a drug’s effect during drug discovery and development.
Describes the effect of the drug in relation to its concentration in
a body fluid
4. The relationships between drug concentration and its effect on
the body.
5. It dedicates to determining the dose, and duration of the effect in
clinical use.
6. Animal model is built to conduct research on drug discovery and
development

10. PHARMACOKINETICS
1) Pharmacokinetics is described as what the body does to a drug, that is
movement of drug into, through, and out of the body— absorption,
bioavailability, distribution, metabolism, and excretion.
2) Determining the mechanisms of a drug's absorption, biodistribution, when
it is eliminated from the body, and what it becomes.
3) pK studies provide the required and useful information that indicates
human equivalent doses (HED), no effect levels (NOEL), and Conducting PK
studies also allows the determination of PK parameters such as AUC,
clearance, and half-life, the volume of distribution, Cmax, and Cmin.
4) Pharmacokinetics of a drug depends on patient-related factors as well as on
the drug’s chemical properties.
5) Other factors are related to individual physiology.

11. ● It is a laboratory test of a new drug or a new medical device, usually done on animal
subjects, to see if the treatment really works and if it is safe to test on humans.
● The drugs are tested using animal models and human cells grown in the laboratory
● The main goals of toxicology is to ensure the safety of the drug/vaccine, determine a
starting, and assess the potential toxicity of the product
● Adverse effects are seen
● Range: 1-3 years, Average: 18 months
Toxicology
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12. Investigational New Drug (IND)
● Investigational New Drug (IND) program by which a pharmaceutical company obtains
permission to start human clinical trials and to ship an experimental drug across state lines (usually
to clinical investigators) before a marketing application for the drug has been approved.
● FDA Time 30 Days for Safety review (US)
● Central Drugs Standard Control Organization (CDSCO) (INDIA)
● Health Canada (HC)
● European Medicines Agency (EMA)
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13. What details should be provided to
IND?
1. The data shows that it is reasonable to begin tests
of a new drug on humans.
2. How, Where and by Whom the new studies will be
conducted
3. Chemical structure of the compound
4. How the compound is manufactured.
5. Toxic effects in animal studies.
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14. Clinical Trials
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Range : 2-10 years , Average : 5 years

15. Informed Consent Form
● The Patient is provided with a consent form
● A consent form is a signed document that
outlines an individual’s informed consent for a
medical study, clinical trial, or activity.
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16. Phase 0(Zero)
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● In recent years, a new trial phase term has emerged – the so-called
phase 0 (zero) or micro-dosing trials.
● It’s an interim step between pre-clinical research and phase I studies
● Investigators use a very small dose of medication to make sure it isn’t
harmful to humans before they start using it in higher doses for later phases

17. ✔The main goal of this trial is to check the safety of the drug and its
Immunogenicity.
✔It takes up to 1-2 years
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Phase I
2-12 Volunteers

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Phase II
● High possibility of drug Rejection or Drug failure in this
process only 33% of medications move on to the phase III
20-100 volunteers
✔ Check the safety
✔ Optimal dose/ doses
✔ Time period

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Phase III
● In this trial the new drug can be compared with the same drug available on the market.
● Long process and Expensive
1000-3000 volunteers
Safety
Efficacy
Shelf life

20. Random Selection
Existing medication
/Placebo
New Drug
Randomized / Double-blind study
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21. ●This involves randomly choosing some participants to receive the new
medication and others to receive an existing medication/ placebo
●A double-blind study is one in which neither the participants nor the
experimenters know who is receiving a particular treatment.
●A placebo is an inactive substance that looks like a drug(Sugar pills)
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New Drug Application
1. A New Drug Application (NDA) tells the full story about a drug from preclinical
data to Phase 3 trial data in an NDA and must include reports on all studies, data,
and analyses.
2. The purpose of an NDA is to provide the FDA reviewer with adequate data to
ensure the safety and efficacy of the drug, labeling, and manufacturing process
3. Along with clinical results, developers must include:
 Proposed labeling
 Safety updates
 Drug abuse information
 Patent information
 Institutional review board compliance information
 Directions for use

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Phase IV
● Done after the drug has been marketed
● Post Marketing Surveillance (PMS).
● No fixed duration / patient population
● Studies continue to collect data about effects on various
populations & side effects of long-term use.
● These are primarily observational or non-experimental in
nature.
● Harmful effects discovered may result in a drug being no
longer sold, or restricted to certain uses.

24. For NDA
1. https://www.fda.gov/drugs/types-applications/new-drug-application-nda