Legislative History of Drug Regulation Derived from the Dutch word meaning to boast (quacken), “quack” is the word Americans have commonly used to describe charlatans in medicine. Quacks peddled adulterated and mislabeled Medicines throughout the United States without penalty until 1906, when Congress passed the Food and Drugs Act, one section of which outlawed the practice. Over the next half-century, Congress passed two major pieces of legislation expanding FDA Authority. It passed the Federal Food, Drug, and Cosmetic Act (FFDCA) in 1938, requiring that Drugs be proven safe before they could be sold in interstate commerce. Then, in 1962, in the wake of deaths and birth defects from the tranquilizer thalidomide marketed in Europe, Congress passed the Kefauver-Harris Drug Amendments to the FFDCA,3 increasing safety provisions and Requiring that drugs be proven effective as well. Congress has amended the FFDCA many times, leading to FDA’s current mission of assuring Americans that the medicines they use do no harm and actually work—that they are, in other words, safe and effective. In recent decades Congress has passed additional laws to boost Pharmaceutical research and development and to speed the approval of new medicines. FDA also regulates products other than drugs—for example, biological products, medical devices, Dietary supplements, foods, cosmetics, animal drugs, and tobacco products. Sometimes the Agency addresses issues that straddle two or more product types that the law treats differently. How FDA Approves New Drugs To market a prescription drug in the United States, a manufacturer needs FDA approval. to get that approval, the manufacturer must demonstrate the drug’s safety and effectiveness according to criteria specified in law and agency regulations, ensure that its manufacturing plant passes FDA inspection and obtain FDA approval for the drug’s labeling—a term that includes all written material about the drug, including, for example, packaging, prescribing information for physicians, and patient brochures. The approval process begins before the law requires FDA involvement. Figure 1 illustrates a product’s timeline both before and during FDA involvement. The research and development process for a finished drug usually begins in the laboratory. Basic research is often conducted or funded by the federal government.7 when basic research yields an idea that someone identifies as a possible drug component, government or private research groups focus attention on a prototype design. At some point, private industry (either a large, established company or a newer, smaller, start-up company) continues to develop the idea, eventually testing the drug in animals. When the drug is ready for testing in humans, the FDA must get involved. The Standard Process of Drug Approval The four FDA steps leading to the agency’s approval of a new drug for marketing in the United States are described below.

1. TO STUDY NEW DRUG
REGISTRATION PROCESS IN U.S
MANOJ DAGWAR
GULBERGA UNIVERSITY
MBA SEM IV

2. Objectives
Learn the processes involved in drug discovery and
development
Define the phases involved in FDA drug approval
Explain the role of the Food and Drug Administration
(FDA) in the drug development and review process in
the United States

3. Reasons for Medication Use
 Prevention
 Vaccines
 Antimalarial agents
 Antibiotic prophylaxis
 Curative
 Antibacterial
 Oncology drugs
 Antifungal
 Control of Disease
Process
 Antihypertensive
 Ant diabetics
 Thyroid agents
 Palliation
 Analgesics
 Oncology drugs

4. Research and Development Process (R&D)
 Development of new drugs is a
complex and costly process
 It takes an average of 12 years
and about $800 million to get a
new drug from the laboratory to
the pharmacy shelf
 R&D involves discovery
(preclinical studies) and
development (clinical studies)
 Only one in 1000 compounds
which begin laboratory testing
will make it to human testing

5. Research and Development Process (R&D)
It takes 12-15 year to discover and develop the new
medicine
Researcher can screen up to 1,000,000 compound,
but only five make it to clinical trial, of those five
only one is approved for patient use
More than 50,000 people involve in drug research
and development process in US.
Clinical trial just 1 new medicine involve more than
5000 patient.

6. Food and Drug Administration
Drug Approval Process In USA
In 1820, the new era of USA drug regulation was
started with establishment of U.S pharmacopoeia
In 1906 passed Food and Drug Act
1937 sulphanilamide tragedy
Drug and cosmetic act

7. Role of FDA
The Food and Drug Administration (FDA) is
required to review and approve all new drugs in the
United States
The FDA reviews and evaluates new drugs based on
the evidence presented from the clinical research
studies performed by the drug sponsor-typically a
pharmaceutical company

8. Discovery/Pre-Clinical Testing
Laboratory & animal studies
Assess safety, biological
activity (if any) MOA and formulations
5,000 compounds evaluated
6.5 years

9. Preclinical Studies
Synthesis and purification of the new drug
Pharmacology of the new drug:
 Pharmacokinetics: absorption, distribution, metabolism, excretion,
half-life
 Pharmacodynamics: mechanism of action and estimates of
therapeutic effects
 Toxicology including carcinogenicity, mutagenicity, and
teratogenicity
Efficacy studies on animals

10. IND
 Investigational New Drug (IND): Application for
permission to administer a new drug to humans
 Outlines the proposal to use the new drug for
human testing in clinical trials
 Studies in humans can only begin after IND is
reviewed and approved by the FDA and an
institutional review board (IRB)

11. Clinical Studies
 Phase 1: Efficacy studies on healthy volunteers
 Phase 2: Clinical studies on a limited scale
 Phase 3: Comparative studies on large number of patients
 New Drug Application (NDA): Regulatory review
 Phase 4: Continued comparative studies. Registration and
market introduction

12. Phase 1
Typically involves 20-80 healthy
Emphasis is on drug safety
Goal is to identify major side effects, metabolism and
routes of excretion
About 70% of drugs will pass this phase

13. Phase 2
Typically involves 100-300 individuals who have the
target disease
Emphasis is on effectiveness
Patients receiving the drug are compared to similar
patients receiving a placebo or another drug
About 33% of drugs will pass this phase
Evaluate side effects & effectiveness
Pharmacodynamics
Determine dose response curve

14. Phase 3
Typically involves 1000-3000 patients
Emphasis is on safety and effectiveness
Investigates through well-controlled studies different
populations and different dosages as well as uses
new drug in combination with other drugs
25-30% of drugs will pass this phase
 Confirm effectiveness monitor adverse
 reactions from long-term use

15. NDA
Pre-NDA period: FDA and drug sponsors meet
Submission of NDA: Formal step asking the FDA to
consider approving a drug for marketing
FDA has 60 days to decide whether it will file it for
approval consideration
If filed, a review team is assigned to evaluate the new
drug

16. FDA Role
 The review team evaluates the research on the safety of
the drug and its effectiveness
 The FDA reviews the information to go on the drug label
 It inspects the facilities where the drug will be
manufactured
 The application will be classified as “approvable” or “not
approvable”

17. FDA Role
If approvable, the FDA requests additional
information from the sponsor
The NDA is again reviewed
Following drug approval, sponsors of the drug will be
required to continually assess the safety of the drug

18. Phase 4
Post-market surveillance of the drug to continually
assess the safety of the drug
May include incidence and severity of rare adverse
reactions, cost-effectiveness analyses, comparative
trials, and quality of life studies
Additional testing as required by the FDA
Continuous monitoring of drug in public use

19. Resources
http://www.fda.gov/Drugs/DevelopmentApprovalProce

20. THANK YOU