Tonko Offers Bipartisan Bill to Strengthen Review of Rare Disease Treatments
/Constituent-inspired legislation brings scientists and doctors with direct expertise into FDA review process
For Immediate Release, February 16, 2021
Contact: Matt Sonneborn, 202-680-8902
WASHINGTON—U.S. Representatives Paul D. Tonko (D-NY) and David McKinley (R-WV) introduced their Helping Experts Accelerate Rare Treatments (HEART) Act today, widely-supported bipartisan legislation that strengthens the U.S. Food and Drug Administration (FDA) review process for drugs that treat rare and ultra-rare diseases by directly involving scientists and doctors with expertise throughout the review process. Rep. Tonko was inspired to introduce this legislation through his work with Melissa Goetz, constituent and Co-President of The Familial Chylomicronemia Syndrome (FCS) Foundation, whose daughter is living with FCS.
“Our bipartisan legislation opens doors of hope and promising treatment for 25 to 30 million Americans who live with a rare disease,” Rep. Tonko said. “We can make a real difference for these individuals and their families by ensuring top experts and patient perspectives are front and center in FDA's rare disease work. I urge my colleagues to join us in moving this legislation forward without delay.”
“People suffering from rare diseases should have access to cutting-edge treatments that best serve their specific needs. This bill makes practical changes to the FDA’s approval process to expand access to safe and effective treatments for rare diseases and ensure patients have a voice at the table.” said Rep. McKinley. “By bringing doctors, scientists, and patients together to participate in this process, we can better serve the needs of the rare disease community.”
The HEART Act:
Requires FDA Rare Disease Program staff to participate in reviews for drugs to treat rare diseases
Directs the inclusion of experts in rare diseases on Advisory Committee panels for rare disease drugs
Assigns the FDA to develop an annual report on the number and progress of rare disease drug applications within each division at the agency
Requires that patients be consulted regarding Risk Evaluation and Mitigation Strategy (REMS) when a rare disease drug needs patient participation
Directs 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
This legislation is supported by the FCS Foundation, Haystack Project, and a broad coalition of other leaders in patient advocacy and treatment for rare diseases.
“As a constituent of Congressman Tonko I am so grateful for his leadership on the HEART Act,” said Melissa Goetz, Co-President of The FCS Foundation. “The FCS Foundation strongly supports this bill that will benefit both the FDA and all rare disease groups. As a mother of a child with FCS, I believe this bill will provide a promise of treatment options for her within her lifetime. We hope all members of the rare disease community including patients, clinicians, researchers, industry partners and friends of those impacted by rare disease will voice their support for The HEART Act by calling their representatives in Congress.”
“In our mission to expand treatment access and reimbursement for patients living with rare and especially ultra-rare diseases, the first and most important issue is that drugs that can provide benefit are reviewed and approved,” said Jim Caro, CEO of Haystack Project. “We applaud the FDA for their many recent efforts to address the unique needs of the rare disease community. The easy-to-implement modifications in The HEART Act will represent a major advance in that process.”
Other organizations supporting the HEART Act include: Usher 1F Collaborative / Soft Bones / Alagille Syndrome Alliance / NBIA Disorders Association / Tuberous Sclerosis Alliance / Alpha-1 Foundation / The National Ataxia Foundation / American Porphyria Foundation / Cutaneous Lymphoma Foundation / International Foundation for CDKL5 Research / The Siegel Rare Neuroimmune Association / International Pemphigus and Pemphigoid Foundation / CDG Care / Usher Syndrome Coalition / Cure Cadasil / Global DARE Foundation / ICAN, International Cancer Advocacy Network / National Niemann-Pick Disease Foundation / International Fibrodysplasia Ossificans Progressiva Association / Kids With Heart National Assn for Children’s Heart Disorders / The FCS Foundation / National Lipid Association Foundation
###
Haystack Project Applauds Introduction of Tonko-McKinley “HEART” Act to Bolster Rare Expertise in FDA Review of Rare and Ultra-rare Treatments
/View Rep. Paul D. Tonko Press Release
The HEART Act, sponsored by Congressmen Paul Tonko (D-NY) and David B. McKinley (R-WV), is supported by a broad coalition of ultra-rare patient advocacy groups.
ANNAPOLIS, MD – July 9, 2020 – Haystack Project, the nation’s leading advocacy organization dedicated to supporting patient access and reimbursement for rare and ultra-rare disease patient communities, announced its support for The HEART Act 2020 (Helping Experts Accelerate Rare Treatments Act), a bill to improve the Food and Drug Administration (FDA)’s review of rare and ultra-rare treatments. Introduced today by Congressmen Paul Tonko (D-NY) and David B. McKinley (R-WV), the HEART Act provides tangible and practical solutions to support rare disease patients and accelerate efforts underway at the agency.
“In our mission to expand treatment access and reimbursement for patients living with rare and especially ultra-rare diseases, the first and most important issue is that drugs that can provide benefit are reviewed and approved,” said Jim Caro, CEO of Haystack Project. “We applaud the FDA for their many recent efforts to address the unique needs of the rare disease community. The easy-to-implement modifications in The HEART Act represent a major advance in that process.”
The HEART Act will position more rare disease experts, including patients and their clinicians, to have an active role in the review process for new drugs. The changes outlined in the HEART Act are designed to be implemented seamlessly and quickly, without increasing drug development timelines or adding new levels of bureaucracy. The HEART Act calls for these changes:
The FDA must consistently include its own Rare Disease Program staff in reviews for drugs to treat rare diseases.
The FDA must consult directly with patients about any Risk Evaluation and Mitigation Strategies (REMS) for a rare disease drug when those REMS programs call for patient participation.
Experts in rare diseases must be included in FDA Advisory Committee panels when reviewing 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, including numbers on the prevalence of those conditions.
The Government Accounting Office must review the EU process for approval of rare disease drugs and provide an assessment of how those processes might apply in the US, including their use of data from open label extension studies.
“The HAYSTACK Project shares our commitment to finding ways to improve the regulatory review process for drugs to treat rare diseases in the U.S. and we are very pleased to join with them in supporting this important bill,” said Lindsey Sutton, co-founder of The FCS Foundation and an FCS patient. “We invite all members of the rare disease community including patients, clinicians, researchers and industry partners to support the HEART Act by asking their own representatives in Congress to cosponsor this important legislation.
It is estimated that there are more than 7,000 different rare diseases, many life-threatening and most without treatments. The development of new therapies to treat rare diseases can be challenging, but patients and their families work tirelessly to bring new treatments forward and should have a place in the regulatory and reimbursement decisions.
“Rare and ultra-rare diseases present a unique challenge for drug development,” said Tonko. “Our bipartisan legislation tackles this by ensuring rare disease experts are at the table as new therapies are being reviewed by the FDA. I urge my colleagues to join us in moving this legislation forward without delay.”
“Oftentimes 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 rare disease experts have a voice at the table,” said Rep. McKinley.
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.
Media Contact:
Jim Caro
CEO
Haystack Project
Jim.caro@haystackproject.org
Saira Sultan
Policy Consultant
Haystack Project
Saira.sultan@haystackproject.org