In a July 9 press release, the Familial Chylomicronemia Syndrome (FCS) Foundation announced its support for a bill introduced by Congressman Paul Tonko (D-NY) and David B. McKinley (R-WV). The bill, also known as the HEART Act, seeks to alter the FDA approval process for therapeutics designed to treat rare diseases. As the leading advocacy group for patients with FCS, the FCS Foundation is uniquely poised to enact change in this realm.
Ultimately, the Act would encourage the inclusion of patient voices into the drug review experience. Although many drug developers work to improve patient outcomes, understanding the intricacies and complexities of rare disease serves to create more targeted and personalized research goals.
The HEART Act stands for Helping Experts Accelerate Rare Treatments. Supported by the FCS Foundation, patient advocacy organizations, and the HAYSTACK Project alike, the HEART Act would encourage new review and approval procedures for rare disease therapeutics. To begin, patients, specialists, and other experts would play a larger role in the drug review process. Additionally, members of the FDA’s Rare Disease Program must partake in the review process.
Next, the HEART Act sets forth a series of provisions, such as:
- Patients will act as consultants for Risk Evaluation and Mitigation Strategy (REMS) plans.
- The FDA must release a yearly report on how many applications they reviewed for rare disease NDAs.
- The Advisory Committee for rare disease therapies must involve rare disease experts (patients, specialists, advocates).
This offers a unique opportunity for the medical field to enhance and uplift patient voices. Additionally, learning directly from patient experiences helps develop effective and topical safety protocols. Admittedly, patients may not have regulatory experience. However, they live with their conditions daily, making them experts in their fields. By using this knowledge, patients feel more in-tune with their medical experience, while researchers better understand rare diseases.
To learn more about the activities of the Familial Chylomicronemia Syndrome Foundation, click here.
To learn more about familial chylomicronemia syndrome, click here.