Drug development, licensing & regulation

What are the main learning aims of drug development and regulation? (5 points)

1. Understand the stages of pharmaceutical development

2. Know why the industry is regulated

3. Be aware of ICH guidelines

4. Know types of regulatory applications

5. Understand the MHRA-s role in medicine safety and quality

What challenges are associated with developing a new medicine? (5 points)

1. Takes 12-14 years

2. Costs over -1 billion

3. High failure rate (~1 in 5000 succeeds)

4. Significant clinical trial costs

5. Only 3 out of 10 marketed medicines recoup R&D costs

Q: What are the two parallel processes in drug development? (2 points)

1. Development - converting drug into safe, effective dosage form

2. Clinical Research - assessing safety and efficacy

What stages are included in drug discovery and development? (7 points)

. Discovery (2-10 years)

2. Pre-clinical testing (animals/lab)

3. Phase I: 20-80 healthy volunteers for safety

4. Phase II: 100-300 patients for efficacy/safety

5. Phase III: 1000-5000 volunteers for long-term safety

6. Regulatory review and approval

7. Post-marketing surveillance

Q: What happens during pre-clinical testing? (4 points)

1. In vitro testing on animal tissue

2. ADME studies on animals

3. Toxicology (acute, chronic, carcinogenicity)

4. Formulation stability assessment

What are the characteristics of Phase I clinical trials? (4 points)

1. 20-80 healthy volunteers

2. Establish safety and dosage

3. High attrition rate (80-90% not progressed)

4. Stability studies in near-final packaging

What are the characteristics of Phase II clinical trials? (4 points)

1. 100-300 patient volunteers

2. Assess efficacy, side effects, dose response

3. Divided into IIa (proof of concept) and IIb (rigorous studies)

4. Finalize dosage form and conduct scale-up

What occurs during Phase III clinical trials? (4 points)

1. 1000-5000 volunteers

2. Multi-centre studies

3. Monitor adverse reactions to long-term use

4. Form main part of regulatory submission

Why is pharmaceutical regulation important? (3 points)

1. Ensures quality, safety, and efficacy of medicines

2. Protects public from unsafe or ineffective products

3. Regulatory agencies validate manufacturer claims

What defines pharmaceutical quality? (6 points)

. Correct product, strength, and container

2. Properly labelled and sealed

3. Free from contamination

4. Not degraded

5. Effective and safe for intended use

6. Manufactured consistently

What is ICH and its purpose? (4 points)

1. International Council for Harmonisation

2. Unites regulators and industry to set global standards

3. Promotes quality, safety, and efficacy in drug development

4. Produces harmonised guidelines

Q: What are the three types of regulatory applications? (3 points)

1. Clinical Trial Authorisation

2. Marketing Authorisation

3. Post-marketing Application

What does a Clinical Trial Authorisation application include? (5 points)

1. Details on sponsor, investigator, trial sites

2. Drug descriptions including comparators and placebos

3. Toxicology and pharmacology data

4. Trial design and subject criteria

5. Data handling and GCP adherence

What are the principles of Good Clinical Practice (GCP)? (6 points)

1. Trial benefits must outweigh risks

2. Subjects must give informed consent

3. Protocol must be scientifically sound

4. Qualified physician responsible for care

5. Data stored for interpretation

6. IMP manufactured under GMP

What are components of a Marketing Authorisation application? (5 points)

1. Details of drug substance/product

2. Quality, safety, and efficacy data

3. Prescribing information

4. Packaging and labeling samples

5. Patient information leaflet (PIL)

\Q: What are the 3 pillars of Marketing Authorisation evaluation? (3 points)

. Efficacy: clinical trial results

2. Safety: trial and toxicity data

3. Quality: reproducibility, stability, specifications

What is a Post-Marketing Application and why is it needed? (4 points)

What is a Post-Marketing Application and why is it needed? (4 points)

What is the MHRA-s role in UK medicine regulation? (6 points)

1. Publishes standards (British Pharmacopoeia)

2. Inspects medicine manufacture and storage

3. Analyzes medicine samples

4. Manages defect reports (e.g. Yellow Card Scheme)

5. Enforces advertising and labeling laws

6. Prosecutes illegal sales of counterfeit medicines

How are counterfeit medicines identified and mitigated? (5 points)

1. May lack active ingredients or contain wrong ones

2. Often mimic packaging of real drugs

3. Use false batch numbers and no traceability

4. Falsified Medicines Directive requires serialization

5. Unique 2D codes track each unit through supply chain