Developing Medicines

Question 1

What are the key stages of drug development?

Answer 1

Discovery research

Phase 1 = 50 healthy volunteers, pharmacokinetics, safety

Phase 2 = 200-400 with target disease, determines dose

Phase 3 = 1000-3000, efficacy against placebo, long-term safety data

Regulatory review = review of efficacy and safety

Phase 4 = <10,000, new indications or formulations

Question 2

Outline the basic scientific principles of early stage studies

Answer 2

Idea/concept

Feasibility

Target validation

Identification of potential compounds

Selection of candidate drug

Question 3

What are the 4 principles of ethics?

Answer 3

Justice – being fair

Beneficence – doing good

Non-maleficence – not doing harm

Autonomy – allowing individual to determine what happens to him/her

Question 4

Outline the basic regulatory environment in which drugs are developed

Answer 4

Regulatory review = MHRA, based on phase 3 data, issues product licence

Critical appraisal

Question 5

What questions are asked during a critical appraisal?

Answer 5

To what sort of pt will the results apply?

What question is being asked in the trial?

What treatments were being compared?

How was randomisation achieved?

Was the study single/double blind?

What outcome measures were used?

If the trial gives a positive result, could this have arisen by chance or by bias?

If the result is negative, could the trial have been too small to detect a clinically important effect?

Question 6

What is the average cost of developing a new medicine?

Answer 6

~800 million