Drug Development

Question 1

What are the major stages in drug development?

Answer 1

Discovery research,

phase 1: pharmaco-kinetics and safety,

phase 2: pharmacology in disease,

phase 3: efficacy, regulatory review,

phase 4: >10,000 for new indications

Question 2

Discuss early basic research

Answer 2

Idea phase: potential molecular targets,

feasibility phase: model devel/hypotheses,

target validation: screening,

identification of potential compounds: Pk/potency/metabolism, selection of candidate drug

Question 3

What is the role of clinical trials

Answer 3

Each stage has diff aims:

1 = pharmaco-kinetics and safety in healthy patients.

2 = therapeutic action and dose, patients with disease.

3 = efficacy, patients with disease.

4: large scale >10,000 long term safety

Question 4

What are the regulatory processes

Answer 4

Submit to national body (MHRA) for product licence required to market drug

Question 5

What is a critical appraisal

Answer 5

Key questions asked: trial question, patients recruited, treatments compared, randomisation, single/double blind, outcomes measures, chance/bias, missed clinical effects?

Question 6

What are the 4 principles of ethics?

Answer 6

Justice (being fair),
beneficence (doing good),
non-maleficence (not doing harm),
autonomy (choice)

Question 7

Why do the pharmacokinetics need to be research properly?

Answer 7

To find the therapeutic window [drug plasma]