Advances in clinical trial design for rare diseases

In Europe alone, an estimated 30 million people suffer from a rare disease, a disorder that affects less than 5 out of a population of 10 000. The ASTERIX project addressed the limitations of clinical trials for such diseases, aiming to speed up the implementation of novel therapies.

Currently there are more than 6 000 rare diseases known for their chronic and debilitating effects. From the multitude of new drugs in development with an orphan designation, only a small fraction has reached the market. This highlights the need to revisit methodology for the design of clinical trials for rare diseases, in light of the inevitable sample size restrictions.

Further details: Advances in clinical trial design for rare diseases