Ways and methods about Drug discovery and development

2. Introduction
 In the past most drugs have been discovered either by
identifying the active ingredient from traditional
remedies or by serendipitous discovery.
 But now we know diseases are controlled at molecular
and physiological level.
 Also shape of an molecule at atomic level is well
understood.
 Information of Human Genome

4. History of Drug Discovery :
Pre 1919
•Herbal Drugs
•Serendiptious discoveries
1920s, 30s
•Vitamins
•Vaccines
1940s
•Antibiotic Era
•R&D Boost due to WW2
1950s
•New technology,
•Discovery of DNA
1960s
•Breakthrough in Etiology
1970s
•Rise of Biotechnology
•Use of IT
1980s
•Commercialization of Drug
Discovery
•Combinatorial Chemistry
1990s
•Robotics
•Automation

5. Drug Discovery methods
 Drugs Discovery methods:
 Random Screening
 Molecular Manipulation
 Molecular Designing
 Drug Metabolites
 Serendipity

6. Steps Of Drug Discovery
Target Selection
Lead
Discovery
Medicinal
Chemistry
In Vitro
Studies
In Vivo
Studies
Clinical
Trials
Cellular &
Genetic Targets
Genomics
Proteomics
Bioinformatics
Synthesis and
Isolation
Combinatorial
Chemistry
Assay
Development
High
Throughput
Screening
Library
Development
SAR Studies
In Silico
Screening
Chemical
Synthesis
Drug Affinity
and Selectivity
Cell Disease
Models
MOA
Lead Candidate
Refinement
Animal models
of Disease States
Behavioural
Studies
Functional
Imaging
Ex-Vivo
Studies
Phase-I
Phase-II
Phase-III
Phase-IV

7. Gene Therapy
 Technique for correcting defective
genes.
 It is the process of inserting genes
into cells to treat diseases.
 Gene therapy is used to correct a
deficient phenotype.

8. Recent Developments
 Nanotechnology + gene therapy yielded treatment to
torpedo cancer
 Results of world's first gene therapy for inherited
blindness show sight improvement
 New Method of Gene Therapy Alters Immune Cells
for Treatment of Advanced Melanoma
 Dual Gene Therapy Suppresses Lung Cancer in
Preclinical Test

9. Orphan Drugs:
 An orphan drug is a pharmaceutical agent that has been
developed specifically to treat a rare medical condition,
the condition itself being referred to as an orphan disease.
 National Organization for Rare
Disorders
 European Organization for Rare
Diseases

10. Advantages:
 Tax incentives.
 Enhanced patent protection and marketing rights.
 Clinical research financial subsidization.
 Rise in research and developmen.
 Crown Corporation.

11. Orphan Drugs Act:
 4th January 1983
 6000 Orphan Diseases
 Unprofitable Drug Development
 Affecting < 2,00,000 Persons
 Orphan Drug Status to 1,090
Drugs
 1985 Amendment- Marketing
Exclusivity
Tourette
Syndrome
An Orphan
Disease