RARE Project would like you to consider taking action in support of the new language (FAST Act) related to the Prescription Drug User Fee Act (PDUFA) in the following ways.  The more support there is for the FAST Act, the better positioned the rare community will be, supporting the inclusion of rare disease language in PDUFA:

1)      Please add your organization’s name to the FAST support sign-on letter  (attached) by emailing Emily Roberts at BIO at eroberts@bio.org by Wednesday March 21st

2)      Click here to email your Member of Congress and ask him/her to become a Co-Sponsor of FAST

The community would not have reached this huge milestone without the support and hard work of the many rare disease disease stakeholders that were involved in and supportive of this bill.  The more than 140 patient organizations and the 2700 advocates who took action to support ULTRA are the real champions of this achievement.

Below is the language from ULTRA/FAST that was incorporated into PDUFA:
Under Subtitle D – Accelerated Approval,  Page 186, line 16:

“(taking into account the severity or rarity of the disease or condition and the availability of alternative treatments) that the product has an effect on—
(A) a surrogate endpoint that is reasonably likely to predict clinical benefit; or
(B) a clinical endpoint, including an endpoint that can be measured earlier than irreversible morbidity or mortality, that is reasonably likely to predict an effect on irreversible morbidity or mortality or other clinical benefit.
The evidence to support that an endpoint is reasonably likely to predict clinical benefit may include epidemiological, pathophysiologic, pharmacologic, therapeutic or other evidence developed using, for example, biomarkers, or other scientific methods or tools.”
Page 191 line 3: “Considerations. – In developing the guidance . . . . the Secretary shall consider . . . . for drugs designated for a rare disease or condition under section 526 of the Federal, Food, Drug, and Cosmetic Act; and
(2)how to incorporate novel approaches to the review of surrogate endpoints based on pathophysiologic and pharmacologic evidence in such guidance, especially in instances where the low prevalence of a disease renders the existence or collection of other types of data unlikely or impractical.”

RARE Project’s strategic partner ‘The EveryLife Foundation’ believes the above language will empower the FDA to consider achievable scientific evidence to qualify a surrogate endpoint for use in a clinical trial, giving very rare diseases that have never been treated before an opportunity to use the Accelerated Approval process.  Most importantly, the inclusion of the language is a message from Congress to the FDA, that they should allow rare diseases as well as other life-threatening diseases access to the Accelerated Approval pathway.  Additionally, the guidance that the FDA is required to publish within one year of passing PDUFA will lay out a predictable and achievable regulatory process that will encourage investors and industry to fund the development of treatments very rare diseases.

More Information:

Inside Health Policy – Draft Energy & Commerce Bill Reveals FDA Reform Priorities For User Fee Package

Emil’s Blog – Guest blogger Julia Jenkins explains how ULTRA became FAST and what happens next