By EveryLife Foundation for Rare Diseases

NOVATO, CALIF., JUNE 25, 2013 — /PRNewswire-USNewswire/ — The EveryLife Foundation for Rare Diseases assembled a working group drawn from industry and patient organizations to develop a white paper to address the critical scientific issues that would govern a successful Guidance for Accelerated Approval in rare disease treatment development.  The Food and Drug Administration Safety and Innovation Act (FDASIA), signed by the President in July 2012, includes language to encourage the appropriate use of Accelerated Approval and requires FDA to finalize a Guidance for Industry on Accelerated Approval in rare diseases by July 2014.

The Foundation, with the support of the rare disease community, advocated for the ability to incorporate the value of all the science available for qualifying biomarker endpoints for use in clinical trials for rare diseases, so more patients have the opportunity to access lifesaving treatments.  Accelerated Approval using biomarkers has been used frequently for cancer and AIDS, but is rarely used for rare disease treatments.  There are currently fewer than 300 approved treatments for nearly 7,000 rare diseases.  Investment and interest in drug development will surge for rare diseases if there is better access to Accelerated Approval.

In order to ensure the timely implementation of the Guidance required by FDASIA, the Foundation began preparing a white paper to provide a scientific framework for the use of biomarker endpoints to measure whether a drug is effective in clinical trials.  The first draft of the paper titled, “Accelerated Approval for Rare Diseases: Recommendations on Guidance for Industry for Qualifying Biomarkers as Primary Endpoints in Pivotal Clinical Studies,” was reviewed by stakeholders and FDA at the Foundation’s May 15, 2013 Workshop.  The second draft, released today, incorporates the feedback received from the Workshop and addresses concerns raised by FDA.  The draft is open for public comment through July 9, 2013.

To view the draft paper or see information from the workshop go to

The Foundation seeks to finalize the white paper by mid-July.

The EveryLife Foundation for Rare Diseases is dedicated to accelerating biotech innovation for rare disease treatments through science-driven public policy.  We can do more with the science we already have and bring lifesaving treatments to millions of people suffering from rare diseases. 


Julia Jenkins, Director of Public & Government Relations 


Phone:  415-884-0223


SOURCE EveryLife Foundation for Rare Diseases