L14 - Regulation and commercialisation

Question 1

What is the pipeline for conventional drug developmet?

Answer 1

Discovery - preclinical studies - phase 1/2/3 - authorisation
P1 - healthy volunteers
P2 - people with disease
P3 - large groups of people

Question 2

What are the main regulatory agencies?

Answer 2

European medical association (Europe)
Food and drug agency (USA)
Ministry of health, labour and welfare (Japan)

Question 3

What is an ATMP?

Answer 3

A medicinal product which is either:

• A gene therapy medicinal product
• A somatic cell medicinal product
• A tissue engineered product

Question 4

What are some concerns related to the use of cells?

Answer 4

Survival, interaction, evolving function and migration
Working with living cells - tumours
Case-by-case considerations

Question 5

What is the general set of overarching considerations?

Answer 5

1. Establishment of manufacturing process and controls
2. Preclinical safety and efficacy studies
3. Clinical trials on human participants

Question 6

What is preclinical safety and efficacy?

Answer 6

Testing in model animals
Considerations of; function and engraftment, safety and immune response
THERE IS NO DEFAULT ANIMAL MODEL - chosen based on scientific reasoning

Question 7

Exploratory trial

Answer 7

Involves small group of patients

Primary concern is safety

Question 8

Dosing

Answer 8

Start based on realistic possibility of beneficial effects

Usually a threshold dose

Question 9

Defining comparator

Answer 9

Gold standard existing treatment

Often surgery based therefore surgeon-dependent

Question 10

Randomisation/blinding

Answer 10

Not always possible with RM - surgery

Patient care and follow up needs to be standardized

Question 11

‘Valley of death’

Answer 11

Gap between discoveries in lab and therapeutic leads that enter clinical trial