Cell Therapies 2

Question 1

3 main obstacles to translation:Hazards and hurdles

Answer 1

-Setting up reliable, consistent and scalable manufacturing system which is key to producing cell therapy on a wider sclae
-Reduce the high costs
-Ensuring safety for patients and suitable education for healthcare professionals.

Question 2

How do you define unproven cell therapy?

Answer 2

• LOA of MOA
• No data from assays
• Inadequate data disclosed to patients
• Uncontrolled experimental procedures in humans

Question 3

What is an ATMP?

Answer 3

An ATMP is any substance or combo of substances administered to human beings which can modify physiologiucal funcions by a pharmcological effect.

It includes:
GTMP- Gene ther
SCTMP- Somatic cell ther
TEP- tissue engineered product
Combined ATMP (contains a medical device as an integral part of a viable cell whcih are liable to act upon the body with action that can be considered primary to the device element.

Question 4

Barriers to progress?

Answer 4

Time to reach market
Different regulations internationally

Question 5

Unmet clinical needs?

Answer 5

T cell immunotherapies for cancer

Question 6

Key considerations- regulation

Answer 6

Pharmacovugilance: detection, assessment, understanding and prevention of adverse effects

Good clinical practice:safety and dignity of human volunteers

Clinical trials: prospectively planned and controlled