Mr.

Siva Kartheek Matam

M.Pharm
Patient Safety Specialist
Mumbai
 Any research conducted on humans with ethical
values to show that a device, drug, or treatment
method is safe and effective.

 It is a process of taking a new drug molecule in to

the market through the regulatory approvals.
1937 – Elixir Sulfanilamide disaster

 105 US citizens died after treatment with

sulfanilamide elixir containing diethylene glycol
(DEG)

 The incident hastened final enactment in 1938 of

the Federal Food, Drug and Cosmetic Act
1950’s - Thalidomide tragedy

 Prescribed for controlling

nausea and vomiting in pregnant
women

 Teratogenic – children born with

deformed limbs.

 1962 – Ban of thalidomide use in

pregnant women.
 PHASE I:
 Objective:
 Objective of Phase 1 studies is to determine the maximum-
tolerated dose with well-defined potential toxicities
 Initial assessment of safety, drug tolerability, and dose range
in humans
 To study pharmacokinetic and pharmacodynamic parameters
of the drug in human beings
 Healthy volunteers
 Small subject population (20-80)
 PHASE II:
 Objective:

 Objective of Phase 2 Studies is to define the dose regimen of

the drug that elicits the desired therapeutic benefit and that
outweighs the observed clinical risks

 Initial assessment of efficacy (proof-of-concept) and

further assessment of safety

 Involve patients who have the indicated disease or

condition

 Small patient population (100-300)

 PHASE III:
 Objective:

 To provide sufficient data to convince the regulatory

authorities of the favourable benefit/risk ratio of the drug
under investigation

 Large-scale studies aimed at verifying efficacy

establishing safety, and establishing the optimum dosage

 Phase-3 studies also called as confirmatory studies

 Involve a larger number of patients (1000-3000)

 Protocol
 Investigator’s Brochure (IB)
 Form 1572 (FDA) – Statement by Investigator
 Undertaking by Investigator (CDSCO –
ScheduleY)
 Financial Disclosure form
 Ethical Committee approval
 Directorate General for Foreign Trade (DGFT)
approval
 Informed Consent Form (ICF)
 Source Documents
 Case Report Form (CRF)
 Serious Adverse Event (SAE) Reporting form
 Investigator Trial File (ITF)
 Correspondence file
 Filing
NDA
(New Drug Applications)
at
FDA
POST MARKETING SURVEILLANCE (PMS):
 Objective:
 To assess the long term effects of the drug

 To determine drug behavior in huge patient pool

 To determine new therapeutic benefits or indications

 Regulatory requirement:
 Structured Adverse Events reporting system
 Periodic Safety Update Reports (PSURs)
 The PSURs are required to be submitted to DCGI
–
 Once in every six months for the first two years of the
drug approval

 Yearly once for the next two years

 Then to be submitted annually.

 The Periodic Safety Update Report (PSUR) is
required as part of the FDA Post Marketing Drug
Risk Assessment (PMDRA) program. 

 USFDA-quarterly reports during first 3 years,

then annual reports

 EMEA - Report should be submitted every 6

months for first 2 years, annually for next three
years and then five yearly after the first renewal
 Country: India
 Authority: Central Drug Standard Control
Organization (CDSCO)
 Head: Drug controller general of India (DCGI)
 The CDSCO, headed by the DCGI, is primarily
responsible for coordinating the activities of the
State Drugs Control Organization, formulating
policies and ensuring uniform implementation of the
Drugs and Cosmetics Act of 1940 (DC Act) and the
Drugs and Cosmetics Rules of 1945 (DC Rules)
throughout India.

 Schedule Y: The legislative requirements of

pharmacovigilance in India are guided by
specifications of Schedule Y.
 United States of America
 Authority: United States Food and Drug
administration (USFDA)

 United Kingdom
 Authority: Medicines and Healthcare products
Regulatory Agency (MHRA)
 European countries
 Authority: Evaluation agency for European
medicines (or) European medicines evaluation
agency (EMEA)

 Australia
 Authority: Therapeutic Goods Administration
(TGA)
 Country: Canada
 Authority: Health Canada
 Medical Pharma

Therapeutics
Statistician
Guidelines
And
Regulations
Other
Life Statistician
Sciences
 JOB
 Clinical Research Coordinator (CRC) or Clinical Trial
PROFILES
Assistant (CTA)
 Clinical Research Associate (CRA)
 Pharmacovigilance Specialist
 Clinical Data Associate
 Medical coder
 Medical Writer
 Regulatory Affairs or Medical Affairs Associate
 Literature Expert
 Medical Adviser or Medical Monitor (Medical
background)
 Biostatistician
Questions???
Thank
You