This document discusses the process of drug discovery and development. It begins with an introduction and then defines the key differences between drug discovery and invention. It notes that drug discovery involves identifying candidate medications through screening and assays, while development involves preclinical and clinical studies for approval. The document then outlines the major steps in drug discovery, from target identification to clinical trials. It notes that the average time and cost to develop a new drug is 12-15 years and $800 million. The need for new drugs to treat diseases and combat resistance is also discussed.

1. Guided by
Dr. A.D. Kulkarni
HOD of PQA
Prepared by
Mr. Vinayak R. Bodhankar
F.Y.M.PHARM (PQA)
Roll No. : 01
Sanjivani College of Pharmaceutical Education & Research,
Kopargaon

2.  Introduction to Drug discovery
 Discovery verses Invention
 Cost of Drug discovery and development
 Need of new drugs
 Steps in Drug discovery
 Clinical Research
 References

3. Discovery vs Invention

4.  Drug discovery is a process by which new
candidate medications are discovered.
 The process of drug discovery involves
identification of candidate, synthesis,
characterization, screening & assay for
therapeutic efficacy.
 Once a compound shows its value in these test,
the process of Drug development starts which
involves preclinical & clinical studies including
regulatory approval for commercialization of
New Chemical Entity

5. Synthesis of New Chemical Entity
IND to conduct Clinical trials
Completion of Phase – III trials
NDA to grant permission
Scrutiny of NDA
Launch the Drug in the Market

6.  The average time required to bring a New drug to the
market range from 12-15 years at an average cost of
$800 million.
 The innovator company synthesizes near about 5000-
7000 compounds out of which approximately 3-5
compounds reach to clinical level & out of which only
one comes to the market.
 Success rate is very low and it is estimated that only 3
out of 10 newly introduced drugs are able to recover
the R & D investment and can generate profit from it.

7. Fig. Approximate stepwise duration for Drug discovery and
development

8.  To combat the drug resistance
 For the improvement in the treatments of
existing diseases
 For the treatment of Newly identified
diseases
 Production of safer drugs by removal of
side effects

9.  Choose a disease
 Choose a Drug target
 Bioassay
 Find a Lead compound
 Isolate & purify lead compound
 Structural determination of lead compound
 Structural Activity Relationship
 Indentify Pharmacophore
 Improve target interaction

10.  Pharmacokinetic properties
 Design manufacturing process
 Clinical trials
 Patent the drug
 Launch the drug in a market

11. - Enzymes
- Receptors
- Nucleic acids, etc.

12.  It is an analytical method used to determine
concentration or potency of drug substance by it’s
effect on living cells or tissues.
 It is used to detect biological hazards.

13.  Compound that possess desired pharmacological
activity is called lead compound.

14. A) Natural sources :
Plants : Digitalis, morphine, etc.
Animals : Insulin, shark liver oil, etc.
Microorganisms : Penicillin, tetracycline, etc.
B) Synthetic banks
C) Existing drugs : It mainly includes “Me too drugs”
e.g of “Me too drugs”
Captopril Enalpril

15. D) Drug can also acts a lead compound based on its side
effects.
e.g Sulfanilamide --------- Tolbutamide
(Antibacterial) (Hypoglycemic agents)
Side effect : Hypoglycemia
E) Serendipity
 It is a chanced or accidental discovery.
 e.g Anti – impotence drug Vigra was discovered by chance
from a project aimed at developing a New heart drug.

16.  Purpose of isolation and purification of lead
compound is to separate the active constituent
from mixture and to get the active constituent
in 100% pure form.

17.  This can be done by using various analytical
techniques such as UV, IR, NMR, Mass
spectroscopy, etc.

18.  Objective of SAR is to determine part of drug
molecules which are important for biological
activity and which are not.
 X – ray crystallography and NMR can be used to
study and identify the interaction between drug
and active sites.
 In this way it is possible to find out which
groups are essential for biological activity and
which are not.

19.  It refers to study of location of functional
groups with respect to biological activity

23.  Manufactured drug need to fulfill the criteria of
clinical trials.
 This is necessary for safety purposes.

25.  Clinical research is a branch of healthcare science that
determines the safety and effectiveness of medications,
devices, diagnostic products and treatment regimen
intended for human use.
 Clinical research process includes :
 Preclinical testing
 IND
 Clinical studies : Phase – I, Phase – II, Phase – III
 Licensing and approval
 Post marketing studies

26.  It is study used to determine the pharmacological
profile of lead molecule using suitable animals.
 Many preclinical studies uses Review committee to
determine if the use of animals is warranted or not.
 Preclinical evaluation mainly consist of :
1) Toxicity study
2) Pharmacodynamic study
3) Pharmacokinetic study

27.  Upon satisfactory completion of preclinical studies, the
new compound is subjected to clinical studies through
proper application known as Investigational New Drug
Application to respective regulatory authorities
 In US : FDA
 In INDIA : DCGI
 For any IND protocol IRB approval is mandatory

28.  It consist of for different phases with specific objectives
 It Involves : Phase – I
Phase – II
Phase – III

29. Number of
participants
Length of
study
Drugs move
to the Next
phase
Objectives
Phase - I 20 - 100 06 – 09
month
70 % Determinatio
n of safe and
tolerable
dose
Phase – II 200 – 300 06 month to
2years
33% Determine
safety and
efficacy of
the drug
Phase - III 300 - 3000 1 to 4 years 25 – 30 % Monitor
adverse drug
reactions

30.  It is started once drug is marketed
 Also called as Post marketing surveillance
 It do not have specific period of time.
 It mainly focused on identification of rare side
effects, previously unknown side effects,
therapeutic indications, etc.

31.  https://www.fda.gov/forpatients/approvals/
drugs/ucm405382.htm
 http://www.drugdiscoverytoday.com
 Basic principles of Drug discovery and
Development by Benjamin E. Blass