Story perspectives

Coalition Urges FDA Clarity as CBER Faces Leadership Changes

4/2/2026

Center

Coalition Urges FDA Reform

On April 1, 2026, a coalition for rare diseases urged the Trump administration to restore regulatory clarity at the FDA's Center for Biologics Evaluation and Research (CBER).
Dr. Vinay Prasad, head of CBER, will leave at the end of April amid disputes over vaccine and gene therapy reviews.
The coalition, consisting of nearly 100 patient advocacy groups and biotech investors, reported that 84% of surveyed investors have reduced or paused investments due to FDA uncertainty.
In 2025, CBER approved five orphan drugs but issued four Complete Response Letters, rejecting about half of late-stage programs.
In early 2026, CBER approved one orphan drug and issued two Complete Response Letters.

Left

Trump's Antitrust Challenges

U.S. President Donald Trump faces skepticism about the administration's antitrust strategy and its enforcement commitment.
Roger Alford, a former antitrust deputy, notes a shift away from enforcement.
34 states continue litigation against Live Nation, contrasting with federal settlements.
Colorado Attorney General Phil Weiser calls the federal approach dangerous and urges Congress to fund enforcement.
Omeed Assefi, DOJ's acting antitrust chief, defended settlements, claiming they achieved unprecedented relief from Live Nation.

History

Historical Context

The FDA Safety and Innovation Act of 2012 aimed to streamline drug approval processes, resulting in a 30% increase in orphan drug approvals for rare diseases. However, ongoing concerns about regulatory clarity have led to 84% of investors pausing funding, highlighting the need for consistent frameworks to support innovation and safeguard patient interests in the biotech sector.

1 of 3

Coalition Urges FDA Reform

On April 1, 2026, a coalition for rare diseases urged the Trump administration to restore regulatory clarity at the FDA's Center for Biologics Evaluation and Research (CBER).
Dr. Vinay Prasad, head of CBER, will leave at the end of April amid disputes over vaccine and gene therapy reviews.
The coalition, consisting of nearly 100 patient advocacy groups and biotech investors, reported that 84% of surveyed investors have reduced or paused investments due to FDA uncertainty.
In 2025, CBER approved five orphan drugs but issued four Complete Response Letters, rejecting about half of late-stage programs.
In early 2026, CBER approved one orphan drug and issued two Complete Response Letters.

1 / 3