9 januari: lifecycle of a drug

Question 1

What do you need to prove to obtain market authorisation application (3)

Answer 1

Safe
efficacious
clean and reproducible manufacturing

Question 2

What makes drug development difficult (4)

Answer 2

1. Complex (laws, multidisciplinary, technical)
2. expensive
3. full of risks
4. short return of investment

Question 3

What is the difference between medical device CTs and IP CTs? Provide reasons as to why it might be easier ánd more difficult.

Answer 3

Differences:
MDs are cheaper to develop and have greater success percentage, but endpoints are harder to measure, there is a smaller market, and standardization and blinding is more difficult.

Question 4

Which phase?: A double-blind, dose-finding, randomised, placebo-controlled, multicentre study with drug A vs drug B in patients with moderate to severe disease

Answer 4

Phase II

Question 5

An open label, randomised, cross-over, single-centre study to compare pharmacokinetics of drug A vs drug B in healthy volunteers

Answer 5

Phase I

Question 6

an open label, randomised, parallel group, multicentre study to compare the eficacy and safety of Drug A vs drug B in patients with moderate to severe disease. This is a trial to investigate a new way of administration of 2 already known and marketed ashtma treatments (drug B) given together in one combination (drug a)

Answer 6

Phase III

Question 7

Describe pre-clinical (processes, subjects, objectives)

Answer 7

Synthesis and purification. Within in silico, in-vivo, in-vitro. Objective: test safety and bio activity (e.g. toxicity, fertility, mutagenicity, carcinogenicity).

Question 8

Describe phase I (objectives, subjects, design)

Answer 8

In-humano, w/ healthy volunteers, to test safety and tolerability. Open, single blinded, placebo controlled. Max Tolerable Dose is determined.

Question 9

Describe Phase II (objectives, subjects, design)

Answer 9

Dose determination, check for efficacy and short term safety. Design: selected subjects, based on diseases. Open, double-blinded, placebo-controlled. Multiple sites.

Question 10

Describe Phase IIIa en IIIb (objectives, subjects, design)

Answer 10

III = comparison to golden standard
IIIa: safety, efficacy, benefit analysis. Prior to submission. Use GS of CTs. n >
IIIb: Cost-effectiveness. Acquire data for MAA & publication. n»

Question 11

Describe Phase IV (objectives, subjects, design)

Answer 11

(not mandatory). After MAA, check for long term safety and efficacy, cost effectiveness and market position.

Question 12

What type of clinical trial is post marketing survellaince?

Answer 12

Observational