Unlocking Lifesaving Treatments for Rare Diseases

Background:

U.S. Representatives Cliff Stearns (R-FL) and Ed Towns (D-NY), two senior members of the House Energy and Commerce Committee, introduced the Unlocking Lifesaving Treatments for Rare diseases Act of 2012, or ULTRA, H.R. 3737, to improve access to the FDA's Accelerated Approval process for very rare diseases by requiring the FDA to use the best science available, ensuring treatments are safe and effective and reach patients sooner.  More than 140 patient organizations signed on to support ULTRA and nearly 3000 people wrote their Member of Congress to support the legislation.   
 
Because of the overwhelming support of the patient community, the goals of the ULTRA Act were then incorporated into H.R. 4132, the Faster Access to Specialized Treatments (FAST) Act, a larger bill that improves the Accelerated Approval process for all serious and life-threatening diseases.  FAST has been included entirely into the House's draft Prescription Drug User Fee Act (PDUFA V).    
 
The Senate's PDUFA draft includes the Accelerated Approval language from S. 2113, the Transforming the Regulatory Environment to Accelerate Access to Treatments (TREAT) Act, which similar language to FAST, however it is missing one of the rare disease components.  More than 50 patient organizations sent letters to the Senate HELP Committee asking for the language to be included in the next draft.  We are currently working with the Senate HELP Committee as well as other stakeholders to ask for inclusion of our language in the Senate's next draft, which is expected to be published by the end of next week.  We need the language to be identical in both the House and the Senate versions or the language will be subject to conference committee and will be negotiated without input from stakeholders.
 
 
The more support we have for the FAST Act, the better positioned we will be to keep our language in PDUFA.  Please click here and take two minutes to send your Member of Congress an email asking him/her to co-sponsor the FAST Act.
 

Specific Rare Disease Accelerated Approval Language in FAST:

 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.”

More Information on FAST & PDUFA:
 
More about ULTRA:
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: