July 29, 2020

On July 9, 2020 the Helping Experts Accelerate Rare Treatments (HEART) Act, a proposed bill to strengthen the review of rare disease treatments, was introduced by Representatives Paul Tonko of New York and David McKinley of West Virginia and supported by the FCS Foundation.

The need for this bill was sparked partly by the FDA review process of Waylivra (volanesorsen) and the disappointing outcome for the FCS patient community – which the Foundation of the NLA saw firsthand at the drug’s Open Public Hearing in May 2018.

The HEART Act will:

1. Require FDA Rare Disease Program staff to participate in reviews for drugs to treat rare diseases
2. Direct the inclusion of experts in rare diseases on Advisory Committee panels for rare disease drugs
3. Assign the FDA to develop an annual report on the number and progress of rare disease drug applications within each division at the agency
4. Require that patients be consulted regarding Risk Evaluation and Mitigation Strategy (REMS) when a rare disease drug needs patient participation
5. and direct the General Accounting Office to review best practices used in the European Union process for rare disease drug approval including data from open label extension studies

We support the patients we serve and the FCS Foundation in their efforts to see that the U.S. Food and Drug Administration therapy review process is improved to better reflect the unique needs of the rare and ultra-rare disease communities.

“We see every day in clinical practice that there are gaps in the treatments available for severe inherited disorders such as FCS. There are still life-threatening lipid disorders that lack adequate options for effective treatment”, says Foundation of the NLA Treasurer Ann Liebeskind, MD.

Today the Foundation of the NLA sent a letter to Representative John Rutherford urging him to join us in supporting the HEART Act (Bill H.R. 7567) on behalf of his constituents in Florida’s 4^th congressional district.