Rare Daily Staff

The Rare Disease Company Coalition, an industry group focused on policy issues, sent a letter to Congressional leaders calling on them to preserve the U.S. Food and Drug Administration’s Accelerated Approval following its widely criticized use to approve an expensive Alzheimer’s therapy that lacked significant evidence of providing benefit to patients.

The group shared concerns with recent policy recommendations advanced by the Medicaid and CHIP Payment and Access Commission (MACPAC), certain state Medicaid programs, and others that the coalition said would “undermine the intent of the accelerated approval pathway; prevent and discourage investment, research, and development into rare disease treatments; and hinder access to treatments for patients who have serious conditions.”

The FDA’s Accelerated Approval program is a mechanism to expedite the availability of safe and effective therapies for patients who have serious conditions with an unmet need. The accelerated approval pathway has been credited with significant advances in the treatment of life-threatening diseases where patients have limited or no treatment options. Historically, this pathway has been used primarily for oncology drugs with 151 oncology accelerated approvals through January 1, 2021. The pathway can provide a critical route for therapies for rare diseases as well.

A drug is considered for the accelerated approval pathway if it treats a serious condition and generally provides a meaningful advantage over available therapies and demonstrates an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit on a clinical endpoint that can be measured prior irreversible morbidity or mortality.

The FDA has maintained that prescription drugs and biologics approved under expedited approval pathways must meet clinically meaningful endpoints and that the benefits of the treatment must outweigh the risks of treatment.

MACPAC wants Congress to increase the minimum rebate percentage and the additional inflationary rebate in the Medicaid program for drugs approved via the Accelerated Approval program tied to the completion of post-marketing confirmatory trials, claiming this approach strikes a balance between reducing Medicaid costs while still maintaining access.

The Rare Disease Company Coalition said it “strongly disagrees.”

“Like recent state proposals requesting the authority to implement closed formularies for their Medicaid programs that would exclude coverage of accelerated approval products, MACPAC’s recommendations would compound access issues already faced by patients with rare diseases,” the group wrote.

The group said that MACPAC’s policy recommendations are not in the interests of patients and does not align with the intent of the Accelerated Approval program to expedite access to rare disease treatments for patients with serious conditions, especially for diseases that have limited or no treatment options.

It argued the policy change would effectively disincentivize and curtail research and development for the treatment of certain rare diseases. It said the change would diminish patient perspective, undermine the purpose of the accelerated approval pathway, disincentivizes the development of rare disease treatments.

“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 approved 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,” the group wrote. “Any future policy reforms to the Accelerated Approval program should build upon FDA’s recent steps to reinforce its oversight of accelerated approval products, leverage and enhance its existing authorities, and protect the integrity of this critical pathway.”

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