Drug development and regulation

Question 1

What is the process of rational drug design?

Answer 1

• Drug target identified and properties characterised by computer
• Crystallographic techniques and computer-aided prediction construct binding site structure
• Modelled interaction
• Combined with chemical libraries
• Rate-limiting factor shift to identification of suitable targets

Question 2

What has changed in approach to drug therapy?

Answer 2

• Productivity of pharmaceutical industry declined over 10-15 years
• Traditional approach of targeting membrane bound receptors and enzymes with relatively small molecules has been exploited
• Modern targets (second messenger systems, nucleic acids, cellular networks)

Question 3

What are novel therapeutic agents?

Answer 3

siRNA, gene therapy, stem cells, antibodies

Lack of development science to guide the translation of molecule into useful medicine

Question 4

What are the phases of drug development?

Answer 4

• 12-15 years
• £200-800 million (coast of failed discovery effects= £1 billion)
• Preclinical phase, 4 clinical phases
• Clinical development= early learning phase and large scale confirming stage

Question 5

What are patents?

Answer 5

Granted for 20 years from point of filing early in drug development (less than 10 years market exclusivity for product)
Recoup costs of research and development
Patent expires, price drops considerably due to competition from generic/ non-branded versions

Question 6

What is protecting market share when patents expire?

Answer 6

Withdrawing the original brand in favour of a new formulation before a generic is available mandating a switch to another branded medicine
Additional patent protection may be offered to stimulate developments in some areas in need

Question 7

How are drugs regulated?

Answer 7

Safety/ Quality/ Efficacy
Licensing= drugs/herbal remedies/ healthcare products
Pharmacovigilance= reviewing new data on ADRs, yellow card scheme
Clinical trials= regulating

Question 8

What is the British National Formulary?

Answer 8

Drugs granted a marketing authorisation by MHRA join products available for prescribing in the NHS list
Standard reference resource used by most prescribers in UK to guide decisions

Question 9

How is access to medicines managed?

Answer 9

Decisions are made as to which of the many medicines that are licensed for use are chosen by individual prescribers
National= NICE, SMC (Scottish Medicines Consortium)
Local= committees

Question 10

What is the role of a formulary?

Answer 10

Agreed limited list of drugs or Formulary with which all local prescribers and other professionals can be familiar, selected on the basis of efficacy and safety, cost-effectiveness, and local preference

Question 11

What is the role of the Drug and Therapeutics Committee?

Answer 11

• Prescribing policies and guidelines
• Formulary
• Non-approved drug requests
• New drug submissions
• Primary care interface
• Patient group direction
• Shared care protocols
• Medication safety incidents
• Reviewing external guidance
• Education