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The Rare Disease Company Coalition Urges Congress to Preserve and Reinforce Accelerated Approval Program to Enable Delivery of Therapies for Challenging Rare Diseases

WASHINGTON – July 29, 2021 – The Rare Disease Company Coalition, a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, today sent a letter to the leadership of the U.S. Senate Health, Education, Labor & Pensions (HELP) Committee and the U.S. House of Representatives Energy & Commerce Committee. In this letter, the Coalition expresses support for the Food and Drug Administration’s (FDA) Accelerated Approval Program while citing concerns with recent policy recommendations put forth by the Medicaid and CHIP Payment and Access Commission (MACPAC).

“The accelerated approval pathway is a critical mechanism that enables expedited availability of innovative treatments for patients with serious and often life-threatening diseases, including rare diseases that may be too challenging to study using a traditional pathway. In these cases, accelerated approval provides a promising roadmap to make possible the delivery of safe and effective rare disease therapies without compromising FDA’s stringent, science-based approval standards,” said Taylor Mason, Executive Director of the Rare Disease Company Coalition. “As a coalition of companies dedicated to rare diseases, we support policies that preserve the intent of the accelerated approval pathway and give all patients who have debilitating diseases a chance to be treated with cutting edge science. The Coalition urges members of Congress to recognize the harmful consequences of undercutting reimbursement, coverage and patient access to therapies developed under the Accelerated Approval Program.”

Throughout the letter, the Rare Disease Company Coalition highlights how the accelerated approval pathway has enabled the timely delivery of innovative treatments, particularly oncology drugs, and provides a proven regulatory framework that can appropriately enable access to therapies for challenging rare disorders, many of which might not be developed otherwise. The Coalition cites ill-informed policy proposals, including the recent recommendations put forth by MACPAC that selectively target accelerated approval drugs as a threat to patient access and the development of rare disease treatments. 

The full text of the letter may be found here. A recent op-ed on the importance of the Accelerated Approval Program for rare disease treatment written by Dr. Emil D. Kakkis, founder of Ultragenyx Pharmaceutical and a company member of the Rare Disease Company Coalition, can be found here. 


The Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes in order for critical innovation to continue. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit

Media Contact:

Anna Stallmann