ARPKD/CHF Alliance Suports Faster Access to Specialized Treatments Act
Faster Access to Specialized Treatments (FAST) Act
ARPKD/CHF Alliance Supports H.R. 4132 FAST
We are excited to share that rare disease Accelerated Approval language made it into the House draft PDUFA bill. This is a huge accomplishment for the rare disease community.
H.R. 4132, the Faster Access to Specialized Treatments (FAST) Act will modernize and expand the FDA’s Accelerated Approval pathway to encompass a broader range of diseases. This reform will speed the approval of much-needed therapies for rare diseases that remain untreated.
In many cases patients are using a therapy that is older and may not work as effectively and safely or have no available treatment.
Language important to arpkd and chf includes:
(A) a surrogate endpoint that is reasonably likely to predict clinical benefit
(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 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.”
This language will empower the FDA to consider achievable scientific evidence to qualify a surrogate endpoint for use in a clinical trial, for rare diseases that have never been treated an opportunity to use the Accelerated Approval process.
Most importantly, passing PDUFA lays out an achievable regulatory process that encourages investors and industry to fund the development of treatments for rare diseases.
