Lecture 2

• Any substance other than food that when inhaled, injected, smoked, consumed, absorbed via a patch on the skin or dissolved causes a physiological change

• Any substance other than food that when inhaled, injected, smoked, consumed, absorbed via a patch on the skin or dissolved causes a physiological change

What is a drug?

• Substance used to treat, cure, prevent, diagnose a disease or promote well-being

What is a medicine?

• Drug substance which is responsible for primary medicinal effect

What is the DS?

• Active pharmaceutical ingredient

What is API?

• Final market ready formula which contains the API (active pharma ingredient)

What is the DP?

• Excipients

What is the term used for any non-API components?

1. Drug discovery

2. Preclinical development

3. Clinical development – phase I-III

4. Phase IV – post market surveillance

What are the 4 stages of drug development?

Any person/organisation that has legally delegated or authority to perform a designated function

What is a competent authority?

• Sponsor – responsibility for writing marketing approval application

• Investigators – conducting clinical trials

Who are the two key stakeholders in pharma reg affairs?

• Law/ legal basis – written statutes

• Regulations – standards and rules

• Guidance – documentation

What are the three regulatory instruments?

• IB – investigators brochure

What document is used in early development?

IMPD – investigational medicinal product dossier

What documents are required in clinical development?

• CTD – common technical document

What doc is needed for licensing approval?

• ICH – International Council on harmonisation

Who sets the format for the CTD?

• Overview and summaries of modules 3-5

What is in module 2 of the ICH CTD?

Quality

What is in module 3 of the ICH CTD?

• Safety – pre-clinical investigations

What is in module 4 of the ICH CTD?

Efficacy – clinical investigations

What is in module 5 of the ICH CTD?

1. Good quality

2. Safe

3. Effective

What three essential criteria must medicine meet?

Health products regulatory authority

What does the HPRA stand for?

• European medicines agency – decentralised EU agency (not regulatory)

What is the EMA?

Eudra-net

What is the name of the IT network that links all parties?

7

How many scientific committees are in the EMA?

• Licensing drugs

• Evaluation of all medicines in EU

• Controlling, advertising medicines

• Research/development of medicines

• Price and reimbursement

• Clinical trial approval

• Med devices

• Healthcare policies

What is the EMA not responsible for?

• Committee for Human Medicinal Products for (CHMP)

• Committee for Pharmacovigilance Risk Assessment (PRAC)

• Committee for Medicinal Products for Veterinary Use (CVMP)

• Committee for Orphan Medicinal Products (COMP)

• Committee on Herbal Medicinal Products (HMPC)

• Paediatric Committee (PDCO)

• Committee for Advanced Therapies (CAT)

What are the 7 scientific committees of the EMA?

Committee for Human Medicinal Products

What does CHMP stand for?

• Centralised is reserved – restricted to innovative products in priority areas

What is the difference between centralised and decentralised procedure?

• FDA CDER (Centre for drug evaluation and research)

• FDA CBER – centre for biologics evaluation and research

• FDA CDRH – centre for devices and radiological health

What are the three FDA centres?

• Investigational new drug (IND) application

What gets filed after a successful pre-clinical trial in the US?

• Generic drugs which do not require full clinical development program

What is the abbreviated new drug application (ANDA) for?

• Harmonize technical requirements for registration or marketing approval

• Develop and register pharma in efficient and cost effective way

• Promote public health

• Prevent unnecessary duplication of clinical trials on humans

• Minimise animal testing

What are the main objectives of the ICH?

1. Build scientific consensus

2. Agreeing on draft text

3. Consulting with regional reg agencies – comment period

4. Adopting harmonized guidelines

5. Implementing guidelines in ICH regions

What are the 5 steps to achieving harmonisation?

• Quality

• Safety

• Efficacy

• Multidisciplinary

What are the 4 types of guidelines in the ICH?

• Quality of biotechnological products

What is the 5th quality guideline in the ICH?