Intro to case

Question 1

outline the drug discovery process

Answer 1

1. target dentification
2. target validation
3. lead discovery
4. lead optimisation
5. pharmacological and toxicological profiling
6. formulation development
7. marketing authorisation

Question 2

what is remdesivir used for

Answer 2

1. programme to find drugs against RNA based viruses
2. shown to be affective against Ebola virus
3. good safety profile

Question 3

what is elixir sulphanilamide used for

Answer 3

1. antibacterial drug
2. demand for liquid formulation
3. mixture tested for flavour, appearance and fragrance but not for toxicity
   - more than 100 people died of kidney failure
   - company prosecuted for mislabeling a product

Question 4

what was thalidomide used for

Answer 4

1. first marketed as a sedative and hypnotic
2. used for morning sickness in pregnant women
   - but caused birth defects

Question 5

what are the expectations of medicines

Answer 5

1. fitness for purpose
2. safety
3. quality
4. efficacy

Question 6

what is the medicines act 1968

Answer 6

1. a response partly due to thalidomide tragedy
2. a consumer protection act, designed to protect the public from faulty products and unscrupulous vendors
3. introduced a licensing system for medicinal products and their manufacture
4. evidence for safety, quality and efficacy of product
5. provided marketing authorisation for medicinal products

Question 7

what is the human medicines regulations 2012

Answer 7

1. replaced the medicines act 1968
2. consolidating legislation
3. ensured compliance with EU directives

Question 8

describe the role of the medicines and healthcare products regulatory agency

Answer 8

1. ensure medicines, medical devices and blood components for transfusion meet applicable standards of safety, quality and efficacy
2. ensure the supply chain is safe and secure
3. promoting international standardisation and harmonisation to assure effectiveness and safety of biological medicines
4. help educate public and healthcare professionals about risks and benefits of medicines

Question 9

what are the key questions for the MHRA

Answer 9

1. do the advantages outweigh the disadvantages of taking the medicine
2. does the medicine do the most good for the least harm for most people who will be taking it
3. are the side effects acceptable

Question 10

who is responsible for monitoring the safety of medicines

Answer 10

the commission on human medicines in 2005

Question 11

describe the involvement of the EU

Answer 11

1. issues directives
2. allows for marketing authorisation across EU
   - centralised procedure through EMA
   - mutual recognition procedure via member states
   - centrally authorised products

Question 12

who is the Uks medicines and medical devices regulator

Answer 12

MHRA

Question 13

describe how Brexit affects licensing of medicines

Answer 13

1. all existing EU MAs for centrally authorised products will automatically be converted into UK MAs
2. EU MA holders of centrally authorised products can opt out of this process if they wish

Question 14

what is the consumer protection legislation made up of

Answer 14

• quality
• safety
• efficacy