Rare Disease Advocates Seek Regulatory Clarity as FDA Balances Rigor and Urgency

Coalition of patient groups and biotech executives call on new FDA leadership to provide more predictable guidance for rare disease therapies.

Apr. 2, 2026 at 1:38pm

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Rare disease drug developers are facing a disconnect between the FDA's stated intent to accelerate therapies and the agency's actual regulatory decisions, as CBER leadership changes have created policy ambiguity. A coalition of advocates and executives have penned a letter to the Trump administration imploring it to 'restore regulatory clarity' as the FDA Center for Biologics Evaluation and Research (CBER) transitions to new leadership.

Why it matters

The FDA is walking a tightrope between scientific rigor and meeting unmet needs for rare disease patients. This balancing act has created strain between the agency and the patient groups and biotech companies working to advance rare disease therapies, with concerns over a lack of regulatory flexibility and predictability.

The details

Over the past 11 months, CBER director Vinay Prasad has led the division in a more stringent and unpredictable manner, as guidance previously given by the agency is seemingly reversed and therapies tested following that advice are rejected. In 2025, CBER greenlit just 5 orphan drugs and issued 4 complete response letters, meaning 44% of decisions resulted in a rejection - compared to the typical 10% rejection rate in prior years. Meanwhile, CDER has approved 4 rare disease drugs in 2026.

  • On March 6, news broke that Vinay Prasad will depart the FDA's CBER division at the end of April.
  • In February, the FDA pivoted from a requirement of two pivotal trials to one for new drug applications, representing a 'redefinition of rigor'.

The players

Vinay Prasad

The controversial and embattled head of the FDA's CBER division since last spring, who will depart the agency at the end of April.

Peter Marks

The longtime and well-respected former regulator who previously led the CBER division before his surprising departure, which sent the XBI biotech stock index plummeting.

Rare Disease Advocacy, Biotechnology, and Investor Coalition (RDBI)

A coalition of patient groups and biotech executives that penned a letter to the Trump administration calling for regulatory clarity as the FDA CBER transitions to new leadership.

Stacey Frisk

The executive director of the Rare Disease Company Coalition (RDCC), who provided data on CBER's recent approval record.

Marty Makary

The FDA Commissioner who noted the agency's ability to exercise regulatory flexibility while also complying with the legal requirement to approve drugs based on 'substantial evidence' of effectiveness.

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What they’re saying

“I know that institutional transitions create policy ambiguity, even when the intent is aligned. But the challenge right now is translating that FDA daily direction into actual, predictable and operational guidance for sponsors.”

— Rahul Gupta, President, GATC Health

“The FDA is capable of doing two things: one, exercising regulatory flexibility; and two, complying with our obligation under the law to approve drugs based on 'substantial evidence' of effectiveness.”

— Marty Makary, FDA Commissioner

“In rare disease we have different challenges. We need to have the understanding that the expectation for a single perfect trial is going to be too high of a bar for many rare diseases.”

— Stacey Frisk, Executive Director, Rare Disease Company Coalition

What’s next

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The takeaway

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