Drug Development and Clinical Trials

There are many types of EB research being undertaken around the world to develop new treatments for EB. Drug development is complex and takes time. 1% of research at drug discovery phase might yield 1 successful new treatment. The good news with EB research is that many potential treatments are now in clinical trial phase and successful EB treatments in the near future look promising.

Understanding drug development and clinical trials

Drug Discovery

1000’s compounds – 3-5 Years

Basic research to understand the process behind a disease

Target for new treatments identified

Search for a drug to act on target

No testing on people

Pre-clinical

100’s compounds – 1-2 years

Assess if potential drug is safe and has an effect

This is done using computerised models, cells and animals

Engage with regulatory authority

Clinical trial application

No testing on people

Clinical Trials Phases I – III

A few compounds – 6-7 years

Phase 1

Safety and pharmacology of the potential drug

Testing on a very small group of healthy volunteers

Small doses

Phase 2

Efficacy for the compound

Tested on fewest number of patients that have the condition to provide statistical significance

Determine the most effective dose and method of delivery, doing intervals and safety

Phase 3

Confirm findings from Phase 2

Investigate the best dosage regime

Tested on a much larger population

Generate data to determine risk-benefit

Marketing Authorisation

1 new drug – 1 – 2 years

Submission for marketing authorisation to national/ European regulatory authority

Licence granted

Individual national cost –benefit assessment

Clinical Trials Phase IV

Phase 4

Data from real-life use

Safety monitoring

Very large numbers of people

Could lead to drug optimisation and further development

For more information on understanding clinical trials and to see what EB trials are recruiting and active for EB see

Clinical Trials FAQ