LAB - 2 - Phases...

Question 1

A systematic investigation in human subjects for evaluating the safety and efficacy of any new drug.

Answer 1

Clinical Trial

Question 2

are a set of tests in medical research and drug development that generate safety and efficacy data for health interventions in human beings. They are conducted only if satisfactory information has been gathered on non-clinical safety and health authority/ethics committee approval is granted in the country where approval of the drug is sought.

Answer 2

Clinical trials

Question 3

Drug Review Steps:

Answer 3

Preclinical (Animal) Testing
Investigational New Drug (IND) Application
Phase 1 Studies
Phase 2 Studies
Phase 3 Studies
Submission of New Drug Application (NDA)
FDA Review
Phase 4 Studies

Question 4

Involves experiments on animal and human cells to gather data on the safety and efficacy of a new drug.

Answer 4

Preclinical (Animal) Testing

Question 5

The sponsor outlines the proposed human testing in clinical trials and submits this application to regulatory authorities.

Answer 5

Investigational New Drug (IND) Application

Question 6

The first stage of testing in human subjects to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of the drug.

Answer 6

Phase 1 Studies

Question 7

Therapeutic exploratory trials to confirm effectiveness, monitor side effects, and further evaluate safety.

Answer 7

Phase 2 Studies

Question 8

Large-scale trials to establish the efficacy of the drug against existing therapies and collect extensive safety data.

Answer 8

Phase 3 Studies

Question 9

The formal step asking the FDA to consider a drug for marketing approval.

Answer 9

Submission of New Drug Application (NDA)

Question 10

Reviewers approve the application or determine it as “approvable” or “not approvable.”

Answer 10

FDA Review

Question 11

Post-marketing surveillance to detect rare adverse reactions, drug interactions, and explore new uses for drugs.

Answer 11

Phase 4 Studies

Question 12

Involves pharmacodynamic studies in animals or in vitro preparations, absorption, distribution, and elimination studies, toxicity studies, and evaluation of the therapeutic index (safety and efficacy).

Answer 12

Preclinical Phase (Animal Studies)

Question 13

Filed after preclinical studies have indicated the safety and efficacy of a drug, seeking regulatory approval for Phase 1, Phase 2, and Phase 3 clinical evaluations.

Answer 13

IND (Investigational New Drug) Application Filing

Question 14

Study of a new drug in micro doses to derive pharmacological information in humans before undertaking Phase 1 studies.

Answer 14

Phase 0 Studies

Question 15

The aim of Phase 0 Studies is to obtain ___ with advantages such as reduced chances of adverse effects, shorter duration, fewer volunteers, and reduced cost and time of development.

Answer 15

pharmacokinetic data

Question 16

The first stage of testing in human subjects to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of the drug. Typically involves 20-25 healthy volunteers and lasts 6-12 months. The primary aim is to determine the maximum tolerated dose of the new treatment.

Answer 16

Phase 1 Studies

Question 17

Therapeutic exploratory trials involving 20-300 subjects to confirm effectiveness, monitor side effects, and further evaluate safety. The drug is given to patients with the disease it is expected to treat. The duration can range from 6 months to several years.

Answer 17

Phase 2 Studies

Question 18

Phase II studies are subdivided into:

Answer 18

Phase IIA: Designed to assess dosing requirements.
Phase IIB: Designed to study efficacy.

Question 19

Therapeutic exploratory trials on a larger scale, involving several hundred to 3000 patients. The duration can be up to 5 years. The goal is to establish the efficacy of the drug against existing therapies, determine the method of usage, and collect comprehensive safety data.

Answer 19

Phase 3 Studies

Question 20

Subtypes of Phase 3 include:

Answer 20

Phase IIIA: To gather sufficient and significant data.
Phase IIIB: Known as “label expansion” studies, allowing the drug to be marketed for additional types of patients/diseases beyond the original approval.

Question 21

A formal proposal submitted to the FDA for approval of a new drug for sale. The application must provide sufficient evidence to establish that the drug is safe and effective, that its benefits outweigh the risks, and that the proposed labeling is appropriate.

Answer 21

New Drug Application (NDA)

Question 22

Post-marketing surveillance (PMS) with no fixed duration or patient population. These studies help detect rare adverse reactions, drug interactions, and explore new uses for drugs. Periodic Safety Update Reports are to be submitted by the manufacturer every 6 months for 2 years, and annually for the next 2 years. Harmful effects discovered during this phase may result in the drug being withdrawn from the market or restricted to certain uses.

Answer 22

Phase 4 Studies