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# Rare Disease Firms Urge Accelerated Approval Support
- URL: https://www.fdaweb.com/rare-disease-firms-urge-accelerated-approval-support/
- Published: 2021-08-04T12:00:00.000Z
- Updated: 2026-09-14T17:09:15.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5149858

The 14 life science companies that belong to the Rare Disease Company Coalition (RDCC) have written to House and Senate leaders to express their support for the FDA accelerated approval program. Their 7/29 [letter](https://www.rarecoalition.com/wp-content/uploads/Rare-Disease-Company-Coalition-Accelerated-Approval-Support-Letter.pdf?ref=fdaweb.com) also discusses concerns with policy recommendations from the Medicaid and CHIP Payment Access Commission (MACPAC) and some state Medicaid programs that they say would undermine the accelerated approval pathway intent; prevent and discourage investment, research, and development into rare disease treatments; and hinder access to treatments for patients who have serious conditions.

The companies describe accelerated approval as a “targeted and robust, science-based pathway established by Congress and FDA to speed the availability of new therapies to patients with serious conditions, especially when there are no available alternatives, while preserving FDA’s rigorous standards for safety and effectiveness.” They say accelerated approval has been and continues to be used in select and appropriate circumstances where evaluating efficacy on clinical endpoints isn’t practical, feasible, or ethical in a reasonable timeframe.

Turning to concerns about recent MACPAC and state Medicaid policy proposals, the companies say the plan to address “high-cost specialty drugs … would effectively curtail the accelerated approval pathway and directly impact the accessibility of new treatments for patients, particularly those with rare diseases.”

The letter says the MACPAC policy recommendations are not in the interests of patients and lists these specific concerns: 

- diminishes patient perspective and may ignore the long-term detrimental impact to rare disease patients;
- undermines the purpose of the pathway; and
- disincentivizes the development of rare disease treatments.

“The RDCC supports policies that will, in reality, strengthen the accelerated approval program and preserve its intent to give patients who have debilitating diseases a chance to be treated with cutting-edge science,” the letter concludes. “We urge members of Congress interested in further strengthening the accelerated approval pathway to recognize the harmful consequences of undercutting reimbursement, coverage, and patient access to accelerated approval therapies and instead focus on meaningful opportunities to optimize FDA’s governance and implementation of this pathway in a way that accounts for evolving science and data generation in the post-market setting and continues to keep patients front and center.”