House FDA User Fee Bill Will Unlock Lifesaving Treatments for Rare Diseases
Rare Disease Community Applauds House Energy & Commerce Committee for User Fee Reauthorization Legislation that Spurs the Development of Treatments

Thursday, May 10, 2012, WASHINGTON, DC—– Today the House Energy & Commerce Committee finished mark-up of HR 5651, the “Food and Drug Administration Reform Act of 2012” that includes several very important provisions to improve access to the Accelerated Approval pathway for rare diseases and spur the development of lifesaving treatments.

The EveryLife Foundation for Rare Diseases applauds HR 5651, the House version of the user fee bill for including language that will fix a “catch-22” that prevents very rare diseases from accessing the Accelerated Approval pathway.  “We are very grateful to Chairman Fred Upton (R-MI) and Ranking Member Henry Waxman (D-CA) for including this essential provision that will unlock lifesaving treatments that have been stalled early in the development process,” said Foundation President Emil Kakkis, M.D., Ph.D.

Subpart D of the House bill will improve the Accelerated Approval pathway for rare and other serious, life threatening diseases.  This would allow for a surrogate endpoint or biomarker (such as a measurement from a blood or urine test) to be used in clinical trials to determine whether the drug is working, which significantly decreases the time and cost to develop a treatment.  Accelerated Approval has been extremely successful in getting treatments approved for cancer and AIDS patients.

Currently, the FDA requires prior clinical information in order to use a surrogate endpoint in a clinical trial.  However for very rare diseases, that prior information does not exist because it has never been treated before.  The language in Section 842 of the House bill could allow the FDA to use all available science to decide if a surrogate endpoint is qualified for use in a clinical study.  “Investment and interest in development will surge for these diseases with very low prevalence if there is an opportunity to access to the Accelerated Approval pathway” says Kakkis.

The revised House bill, which is now hailed to be bi-partisan, includes additional provisions that improve the FDA’s review and approval process that were originally included in the Senate draft.  The EXPERRT Act that will allow the FDA to consult with rare disease experts and the Breakthrough Therapies Act that requires the FDA to take actions to expedite the development and review of a “breakthrough” drug are now both in the House version.  Language from the Creating Hope Act to create additional incentives for industry to develop treatments for rare pediatric diseases and cancers through a priority review voucher was also included in the House version; however it is not in the Senate bill.

“With less than 400 drugs approved to treat 7000 rare diseases affecting 30 million Americans, all of the rare disease provisions in HR 5651 are important to improve the development of lifesaving treatments” said Representative Cliff Stearns (R-FL) who championed the rare disease Accelerated Approval language with Representative Ed Towns (D-NY) that first appeared in the ULTRA Act and later was reintroduced in the FAST Act.

Early this week during the Health Subcommittee mark-up, Rep. Towns submitted for the record, more than 50 letters from patient organizations supporting the inclusion of the rare disease Accelerated Approval language in FAST into PDUFA.  Among these were a press release from the National Organization of Rare Diseases (NORD) and a letter from the Biotechnology Industry Organization (BIO).  All the letters support “the guidance language that I hope that we maintain as we continue to move through the legislative process. … I feel that all of these materials are important to include as part of the record,” wrote Towns in his opening statement.

The bill is a major win for the rare diseases community.  “We were very pleased that so many Members of Congress spoke about the need to help rare disease patients during mark-up,” said Kakkis.  “The House’s version of the bill is more compelling to improve the use of a surrogate to determine if a treatment is effective, which is essential for diseases with very small patient populations.”  The Foundation is optimistic that the House version of the Accelerated Approval language, which is preferred by rare disease groups, will be retained during Conference Committee to ensure what is best for the patients.

Now that the bill has passed out of the Energy and Commerce Committee it must be voted on by the full House.  According to Chairman Upton, the bill will be on the House Floor for a vote before the end of the month.  “That will give us time to work with our good friends in the Senate to have this bill to the President before the 4th of July,” said Upton during mark-up.  The Senate and the House will pass separate bills; both bills are expected to pass without much difficulty.  The differences between bills will be negotiated during Conference Committee in June before it is sent to the President for his signature.

The EveryLife Foundation for Rare Diseases is dedicated to accelerating biotech innovation for rare disease treatments through science-driven public policy.  We can do more with the science we already have and bring life saving treatments to millions of people suffering from rare diseases. 

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Contact: Julia Jenkins, Director of Public & Government Relations
Email:  jjenkins@EveryLifeFoundation.org    Phone:  415-884-0223

1 thought on “HR 5651, the Food and Drug Administration Reform Act of 2012”

  1. Julie,

    This is a great step for the rare community. Your work is greatly appreciated.
    Marianne Vennitti
    Cryoglobluminemia Vasculitis Organization
    CVO-Assistant Director

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