Let’s Make History for ALL Rare Disease Communities!

In 2020, the FCS Foundation joined with the HAYSTACK Project and other leaders from the Rare Disease Community to ask Congress to make some important changes in the process we use to review and approve drugs to treat rare diseases in the U.S.

The bill, called the HEART Act (Helping Experts Accelerate Rare Treatments), is designed to ensure that experts who really understand rare diseases are involved in the review process. The HEART Act calls for these simple and common-sense changes:

• The FDA must include members of its own Rare Disease Program staff in reviews for drugs to treat rare diseases.
• FDA must include experts in rare diseases on Advisory Committee panels for rare disease drugs.
• Each year the FDA must prepare a report showing how many rare disease drug applications were reviewed by each division at the Agency.
• Members of the patient community must be consulted when devising or reviewing a Risk Evaluation and Mitigation Strategy (REMS) for a rare disease drug.
• We should review the EU process for approval of rare disease drugs including the use of data from open-label extension studies.

JOIN US IN SUPPORTING THE HEART ACT!

The HEART Act will help make sure that the drug review process in the U.S. reflects the unique needs of all rare disease communities. It will allow both patients and clinicians who really understand rare diseases to participate in the review process. TO GET THIS BILL PASSED, WE NEED YOUR HELP!

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