Pharmacology

Lab

Drug Discovery and Development

Introduction:
Drug discovery is the process by which drugs are discovered and designed. It is a
process which aims to create a compound therapeutically useful in curing & treating
disease. The development of new drug is a product of cooperation between public and
private organizations to translate basic researches into medicine.

Steps involved in Drug Discovery and Development:

1) Discovery
2) Preclinical Testing
3) Clinical Trials
4) Approval for Manufacture
1) Discovery:
The discovery process includes all early researches to identify a new drug candidate
and testing its usefulness. Discovery involves the following arrangements:
 Target Identification: Understanding the disease mechanisms.
 Target Validation: Choose a molecule to target the disease; that is believed to be
connected to pathogenesis.
 Leads Discovery: Find a promising lead compound that could become a drug and
confirm its role in the disease.
 Early Safety tests: Perform initial safety tests on the lead compound.
 Lead optimization: Alter the structure of the lead compounds to improve properties.

2) Preclinical Testing:
The pre-clinical development includes the following:
 Animal safety studies; carcinogenicity tests;
 Drug delivery; elimination and metabolism studies;
 Drug formulation experiments; dose-ranging studies in animals.
Wide ranging dosages of the compounds are introduced to the cell line or animal in
order to obtain preliminary efficacy and pharmacokinetic information.

3) Clinical Trials: This phase is the longest one in drug development ranging from 2-
10 years. This is carried out by many methods e.g.:
Placebo controlled trials
Double blinded studies

Clinical trials consist of 4 phases:

Phase 1 - A small group of healthy volunteers (20-80) are selected to assess the safety,
tolerability, pharmacokinetics, & pharmacodynamics of a therapy.
Phase 2- Performed on larger groups of patients (20-300) & are designed to assess the
activity of the therapy, & continue phase1 safety assessments.
Phase 3 -Randomized controlled trials on large patient groups (hundreds to thousands)
aimed at being the definitive assessment of the efficacy of the new therapy. Side
effects are also monitored. Once a drug has proven acceptable is submitted to the FDA
for review.
Note: Successful drugs must be: absorbed into the bloodstream, distributed to the
proper site of action in the body, metabolized efficiently and effectively, successfully
excreted from the body and demonstrated to be not toxic.
Phase 4 - Post-launch safety monitoring & ongoing technical support of a drug may be
initiated by the pharmaceutical company designed to detect rare or long term adverse
effects over a large patient population & timescale than was possible during clinical
trials.

GOOD LUCK