Clinical Trials: Life Cycle & Application

Question 1

Examples of drugs given for a different indication than approved for

Answer 1

‘Off-label use’

• Aspirin (technically for inflammation, but also analgesia)
• Domperidone (used for inhibiting gastroenteritis, happens to induce prolactin → mothers produce more breast milk)

Question 2

Drugs already approved to treat other problems that have been repurposed

Answer 2

Viagra & Rogaine - originally a drug for BP

Pentamidine (anti-microbial medication) and metformin (type 2 diabetes treatment) - repurposed for cancer treatments

Question 3

An example of a drug used in individualised patient care

Answer 3

BoTox

Question 4

What does the preclinical phase involve?

Answer 4

It starts with a good idea in the lab!

Question 5

What is a sponsor responsible for in a clinical trial?

Answer 5

An individual, institute, or company which is responsible for managing and financing trials

Question 6

Examples of unethical case studies

Answer 6

• Tuskegee Syphilis Experiment (black people with undiagnosed syphilis studied by white scientists without their knowledge) 1932-1972
• STI Study in Guatemala (1940s)
• India Rotavirus (2010s) - parallel RCT for oral Rotavirus vaccine, didn’t offer vaccine at end to all participants once they knew it worked

Question 7

Why is blinding not always best?

Answer 7

As seen in PD GDNF studies in Bristol..

• High cost assoc. with producing placebo drug as well as drug of interest
• Sham surgery thought to be unethical
• People thought double-blinding was wrong & criteria were too strict in this scenario

Question 8

Phase 4 of clinical trials is important for?

Answer 8

Pharmacovigilance & Post Marketing surveillance..

Observing the drug on the market, in the wider population and of less common side effects