Obama signing a bill

President Signs Rare Disease Legislation into Law


FDA User Fee Bill Promises Real Hope for Rare Disease Patients

Major Wins for the Grassroots Patient Community

July 9, 2012, Washington, DC —– Rare Disease Legislative Advocates (RDLA) hails the President’s singing of The Food and Drug Administration Safety and Innovation Act (FDASIA), S. 3187, landmark legislation that will encourage the development of new treatments for the 30 million Americans suffering from rare diseases. There are currently fewer than 400 treatments approved by the U.S. Food and Drug Administration (FDA) for the nearly 7000 rare diseases which have been identified.

FDASIA includes the reauthorization of the Prescription Drug User Fee Act (PDUFA), the culmination of more than a year of negotiations between industry and the FDA and advocacy by the patient community to ensure the reauthorization will encourage the development of new treatments.  The legislation passed the House and Senate with broad bipartisan support.

“The inclusion of so many rare disease provisions in FDASIA is a testament of the power of the grassroots patient community” said John Crowley, parent advocate and CEO of Amicus Therapeutics.  More than 300 patient organizations voiced their support for various provisions over the months of Congressional discussions and negotiations.  However, “it was the united voices of the patient community that ensured the ultimate success of the rare disease provisions,” said Crowley.

RDLA worked with patient organizations to help educate the patient community about the different legislative initiatives and to build grassroots support for rare disease legislation.  More than 10,000 emails were sent from constituents to Congress from RDLA’s Congressional action center to support the legislation.  In February, RDLA hosted its first lobby day, bringing 70 patient advocates to Washington, D.C. to advocate for the Creating Hope Act and the Unlocking Lifesaving Treatments for Rare Diseases Act (ULTRA), which was later included into the Faster Access to Specialized Therapies (FAST) Act. Both provisions were included in FDASIA.

The Creating Hope Act, sponsored by Kids v Cancer, creates additional incentives for industry to develop treatments for rare pediatric diseases and cancers by granting a voucher for priority review status that the sponsor can use to expedite review of another product.  ULTRA/FAST, spearheaded by the EveryLife Foundation for Rare Diseases seeks to improve access to the accelerated approval process for rare disease treatments, significantly decreasing the time and cost of FDA review while maintaining high safety and efficacy standards.

During the negotiations to reconcile the House and Senate bills, 121 organizations signed onto a letter circulated by Parent Project Muscular DystrophyGenetic Alliance and the EveryLife Foundation championing the inclusion of the strongest rare disease provisions in the final bill.  All five provisions supported in the letter were included in the final bill.

These provisions include the EXPERRT Act, championed by the Cystic Fibrosis Foundation, that will allow the FDA to consult with rare disease experts; the Breakthrough Therapies Act, backed by Friends of Cancer Research, that requires the FDA to take actions to expedite the development and review of a “breakthrough” drug; and the Patient Participation in Medical Product Discussions provision, championed by the Parent Project Muscular Dystrophy, that provides for a path forward to deepen patient engagement in reviewing medical products to expand and strengthen the patient voice in FDA discussions.

Also included in FDASIA was language to encourage greater use of the existing Humanitarian Use Device (HUD) program by expanding the scope to include adults and allowing companies to make a profit, and the reauthorization of and improvements to the FDA’s Orphan Drug grant program.

Finally, during the user fee negotiations, FDA Commissioner Margaret Hamburg, M.D. signed a commitment letter agreeing to a Rare Disease Initiative, which includes:

  • Increased staffing of the Center for Drug Evaluation and Research (CDER)/Center for Biologics Evaluation and Research (CBER) Rare Disease Programs (RDP) which provides expertise in orphan drug development to the product review divisions;
  • Increased FDA efforts to ensure that product reviewers, industry, and patients are working together;
  • Broadening of  research and programming in the areas of non-traditional clinical trial design, endpoints, and statistical analysis associated with orphan drug development;
  • Enhanced staff training for reviewers with specific regard to approval of drugs for rare diseases;
  • Better integration of RDP staff into review teams.

RDLA will host a “Rare Voice” Awards Gala on November 14th in Washington, D.C. to honor and thank Members of Congress, Congressional staff, patient organizations and grassroots advocates for their efforts to pass this essential legislation and also other “rare disease voices” that speak up for the community and its needs.

Rare Disease Legislative Advocates (RDLA) is a collaborative organization designed to support the advocacy of all rare disease groups.  RDLA works to empower the individual to become an advocate by providing informational meetings, legislative resources, advocacy tools, and special events that support organizations and advocates working to promote rare disease legislation.  RDLA’s goals are to bring the rare disease community together, grow the patient advocacy community and work collectively to ensure that the many voices of patients with rare diseases have an opportunity to be heard on Capitol Hill.