Unlocking Lifesaving Treatments for Rare Diseases

Specific Rare Disease Accelerated Approval Language in FDASIA:

“(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.”

“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.”

Background on ULTRA/FAST:
If you have any additional questions, please don't hesitate to contact us at 415-884-0223 or patientadvocate@kakkis.org.
Watch testimonials on ULTRA from our parent advocates: