Drug Approval Process

Question 1

Pure Food and Drug Act of 1906

Answer 1

• required accurate labeling of the ingredients in drug products
• sought to prevent the adulteration of products
• NO REQUIREMENT of proven safety or efficacy to market drugs

Question 2

Why did the Pure Food and Drug Act of 1906 come about?

Answer 2

-sale of patent medicines that often contained toxic, habit-forming or other non-beneficial ingredients

Question 3

Food, Drug and Cosmetic Act of 1938

Answer 3

• required any NEW drug to be evaluated for safety for the labeled use by its manufacturer before marketing
• created the FDA

Question 4

Why did the Food, Drug and Cosmetic Act of 1938 come about?

Answer 4

-sulfanilamide elixir that contained ethylene glycol caused 100 deaths

Question 5

Durham-Humphrey Amendment of 1952

Answer 5

-created legal distinction between prescription and NONprescription drugs

Question 6

Kefauver-Harris Amendments of 1962

Answer 6

-pharmaceutical companies required to prove efficacy and safety for the labeled indication before FDA approval for marketing

Question 7

Why did the Kefauver-Harris Amendments of 1962 come about?

Answer 7

-thalidomide malformations (was used to treat morning sickness in pregnancy –> kids born with missing or malformed limbs)

Question 8

Orphan Drug Amendments 1983

Answer 8

-provided tax benefits and other incentives for manufacturers to test and produce drugs to treat rare diseases

Question 9

Drug Price Competition and Patent Restoration Act of 1984

Answer 9

• extended patent life of drug products by the amount of time required for regulatory review of a NDA
• accelerated the approval of generic drugs by allowing submission of an abbreviated NDA

Question 10

Accelerated Drug Approval 1992

Answer 10

-authorized accelerated drug approval for new drugs to treat life-threatening conditions (eg AIDS, cancer, Ebola)

Question 11

List the steps in drug development in the US (7).

Answer 11

1. discovery and characterization of new drug
2. pre-clinical studies
3. IND (investigational new drug) application
4. clinical studies
5. submission of NDA (new drug app)
6. approval of NDA
7. postmarketing surveillance

Question 12

Step 1: Discovery and Characterization of a New Drug

Answer 12

• isolate or synthesize a new drug

- determine chemical/pharmaceutical properties of new drug

Question 13

Step 2: Pre-clinical Studies

Answer 13

• determine pharmacokinetic and pharmacodynamic properties of drug
• animal testing for toxicity, teratogenesis, mutagenesis and carcinogenesis

Question 14

Step 3: IND Application

Answer 14

• outline properties of the drug
• report results of study to date
• propose clinical studies (protocols, data analysis methods, etc)

Question 15

Step 4: Clinical Studies

Answer 15

• phase I: drug safety and pharmacokinetics in healthy volunteers
• phase II: efficacy, safety, proper dose in small group of patients
• phase III: statistical evidence of drug safety and efficacy (compare to a placebo or a current drug for the same disease)

Question 16

Step 5: Submission of NDA

Answer 16

• outline properties of the drug
• report results of all experimental and clinical studies
• propose labeling of drug and clinical indications for use=

Question 17

Step 6: Approval of NDA

Answer 17

-marketing of drug

Question 18

Step 7: Post-marketing Surveillance

Answer 18

-gather and analyze voluntary reports of adverse effects