The Heart Act

THE HEART ACT – Helping Experts Accelerate Rare Treatments


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.


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!

Please fill out your information below, and our tool will help you identify your representatives in the House and Senate and populate an email for you. Then you can edit the email and send it out with a simple click!

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Everything is pre-populated in the tool below 




Here are some other ways that you can support the HEART Act:

  • Send us a quotation of support from your ED or other member of your organization that we can use in press releases and other efforts to promote the HEART Act.
  • Contact your members of Congress and ask them to support the HEART Act and ask your stakeholders to do the same. Please go to or and use the tools provided to send your stories to Congress members to support the HEART act. 
  • The FCS Foundation (FCSF) and other advocacy groups will be conducting a social media campaign to support The Heart Act – please like and share our content and develop your own – and please use the hashtag #HEARTACT2020
  • FCSF can also provide you with a brief article about The HEART Act for your in-house newsletter or LinkedIn page.
  • Reach out to your industry partners to ask them to support this effort.

The next step is to get The HEART Act filed for review in Congress. We are working with many supportive legislators to move this forward. Please make your voice heard on behalf of your constituents and all people affected by rare diseases in the U.S. We look forward to updating you about our progress.