welcome to DuchenneXchange

- a positively charged Duchenne muscular dystrophy community.
  • join today!

Rare diseases require unique approaches in clinical trial design

There are 7,000 known rare diseases and disorders, and collectively they affect nearly 30 million people in the USA. The vast majority of rare diseases currently have no approved treatments available for patients, despite the fact that research related to rare diseases has expanded significantly in the past 20 years. A key reason may be the many unique challenges researchers may face when developing drugs to treat rare diseases, including difficulties designing and executing effective and appropriate clinical trials.

The example of DMD offers us an important road map indicating how clinical research and regulatory review in rare diseases can be improved in the years ahead. In recognition of the challenges in developing therapies for rare diseases, the FDA introduced the Safety and Innovation Act (FDASIA) of 2012 and the 21st Century Cures Act of 2016, both of which include provisions that recognize the need for new levels of flexibility in the regulatory review process for many of these diseases. These acts also highlight the importance of hearing from patients and caregivers about their experiences and using those insights to assess treatment benefits.


Rare Diseases Require Unique Approaches In Clinical Trial Design