Familial Chylomicronemia Syndrome Foundation Supports Tonko-McKinley Bill to Amend FDA Review Process for Drugs to Treat Rare and Ultra-Rare Diseases in the U.S.

ALBANY, N.Y., July 9, 2020 /PRNewswire/ -- The FCS Foundation, the nation's leading advocacy and support organization for people affected by familial chylomicronemia syndrome (FCS), today announced its support for The HEART Act (Helping Experts Accelerate Rare Treatments Act), a bill introduced today calling for tangible and immediately impactful changes in the U.S. Food and Drug Administration (FDA) process to review and approve drugs to treat rare and ultra-rare diseases. The HEART Act is sponsored by Congressmen Paul Tonko (D-NY) and David B. McKinley (R-WV) and supported by The FCS Foundation, HAYSTACK Project, and a broad coalition of leaders in patient advocacy and treatment for rare diseases.

The HEART Act is designed to ensure that experts who really understand rare diseases, including patients and medical specialists, are involved in every stage of the review process for new drugs. The HEART Act calls for several specific changes to the drug review and approval process in the U.S., including:

• 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 that calls for patient participation.
• The General Accounting Office must review the EU process for approval of rare disease drugs including use of data from open label extension studies for relevance to the FDA.

"In our discussions with the FDA to support research and treatments for familial chylomicronemia syndrome, we saw firsthand the need to include our community's insights on the impact of a rare disease on patients and families," said Lindsey Sutton, co-founder of The FCS Foundation and an FCS patient. "While The FCS Foundation and other rare disease advocates appreciate recent efforts at the FDA to improve the agency's understanding of rare conditions, the changes outlined in the HEART Act are a critical next step in this process."

"It is absolutely essential that the process we use to review and approve drugs for rare diseases in the United States reflects the unique issues that apply to these diseases, which are often not well known or understood by regulators and even by clinicians," said Melissa Goetz, co-founder of The FCS Foundation and the parent of a child with FCS. "We may not be experts in the regulatory process, but we are experts in our own rare conditions with important and helpful information to share."

"In many cases, information about the burden and impact of an ultra-rare disease is not well understood and data are very limited. As a result, efforts to review new drugs cannot follow the standard procedures that might work well for diseases like diabetes or breast cancer that affect millions of people," said Jim Caro, CEO of HAYSTACK Project, an umbrella group of mostly ultra-rare patient organizations. "We very much appreciate that Congressmen Tonko and McKinley introduced this bill today. The HEART Act contains practical solutions to the very real problem of involving patients and medical experts in the review process."

It is estimated that there are more than 7,000 different rare diseases that collectively affect more than 30 million people in the United States. Many rare conditions are life threatening and most do not have treatments. The development of new therapies to treat rare diseases can be challenging for many reasons, including lack of understanding of disease mechanism of action and limited information about the impact on patients and their families.

"Often times people suffering from rare diseases have few or no treatment options available. This bill makes practical changes to FDA's approval process that can lead to new treatments and ensure patients have a voice at the table," said Representative McKinley. "By having more experts available to participate in this process, we can better understand and address the needs of patients moving forward." 

"Rare and ultra-rare diseases present a unique challenge for drug development because, while some 25 to 30 million Americans have a rare condition, any one of these conditions by definition only impacts a relatively small number of individuals," said Representative Tonko. "Our bipartisan legislation tackles this issue by ensuring rare disease experts are at the table as these therapies are being reviewed by the FDA as well as amplifying the patient perspective as safety protocols are being established. I urge my colleagues to join us in moving this legislation forward without delay."

Click here to see the press release issued by Representatives Tonko and McKinley.

The FCS Foundation together with other leaders from the rare disease patient advocacy community are planning a national awareness campaign to support The HEART Act, including a social media program to invite community members to share their stories of living with a rare disease and encourage their leaders in Congress to support the bill. 

About The Familial Chylomicronemia Syndrome Foundation
Founded in 2016, The FCS Foundation's mission is to establish a global support network to promote advocacy and education for patients and caregivers living with FCS. The FCS Foundation is the only foundation dedicated to patients suffering from familial chylomicronemia syndrome. For more information, visit: www.livingwithfcs.org.

About HAYSTACK PROJECT

Haystack Project is a 501(c)(3) non-profit organization enabling rare and ultra-rare disease patient advocacy organizations to coordinate and focus efforts that highlight and address systemic reimbursement obstacles to patient access.  Our core mission is to evolve health care payment and delivery systems with an eye toward spurring innovation and quality in care toward effective, accessible treatment options for rare and ultra-rare patients.  We strive to amplify the patient and caregiver voice in these disease states where unmet need is high and treatment delays and inadequacies can be catastrophic. For more information, visit https://haystackproject.org.

Media Contacts:
Jenna Urban
Berry & Company Public Relations
[email protected] 
212 253 8881

SOURCE The FCS Foundation

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