Unlocking Lifesaving Treatments for Rare Diseases

(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.”

• Blog about how ULTRA became FAST 
• Inside Health Policy: Draft Energy & Commerce Bill Reveals FDA Reform Priorities for User Fee Package 
• BIO Century:  How Fast is FAST 
• Foundation President, Emil Kakkis' blog about why we decided to seek legislation
• Rep Stearns' Press Release on the introduction of the ULTRA Act
• FDA Law Blog: ULTRA Bill Introduced in the House; Legislation Seeks to Permit Broader Use of Scientific Data to Support Surrogate Endpoints for “Ultra Orphan” Drug Approvals
• Dec. 16, 2011, InsideHealthPolicy.com's FDA Week reports Rep Stearns to Introduce the ULTRA Act
• Nov. 11, 2011 FDA Week: Patient Advocates Seek To Accelerate Approval of Ultra Orphan Drugs
• Feb. 8, 2012 Congress Member Towns speaks about ULTRA on the House Floor 
• Oct. 14, 2011 Rep Stearns letter to FDA regarding ultra rare diseases