FDA's Orphan Drug Office Sees Leadership Change Amid Policy Shifts

Retzky's Departure Marks Latest in Series of FDA Exits

Sandra Retzky, the director of the FDA's Office of Orphan Products Development, has left her position, marking another significant change in the agency's leadership. While an unnamed source confirmed to Endpoints News that Retzky has not been fired, her new role within the FDA remains unclear. This move comes at a crucial time for orphan drug policy and regulation.

Retzky, who had been leading the orphan drug department since August 2021, oversaw the office's efforts to promote and support the development of medical products for rare diseases. Her departure follows a string of high-profile exits from the FDA and other health agencies, including Nicole Verdun from the Office of Therapeutic Products and Peter Marks from the Center for Biologics Evaluation and Research.

Recent Policy Developments in Orphan Drug Regulation

The leadership change coincides with significant policy shifts affecting orphan drugs. The Centers for Medicare and Medicaid Services (CMS) recently finalized guidelines for the Inflation Reduction Act, expanding exemptions for orphan drugs. Under the new rules, the countdown for qualifying for drug price negotiations will only begin after a drug receives approval for a non-orphan indication.

Additionally, the House Committee on Energy and Commerce has advanced legislation that could revive the priority review voucher program for rare pediatric disease drugs. This program, which lapsed in September 2024, was designed to incentivize pharmaceutical companies to invest in treatments for rare childhood diseases.

Broader HHS Staffing Shakeup Raises Concerns

The changes at the FDA are part of a larger reorganization within the Department of Health and Human Services (HHS) since Secretary Robert F. Kennedy Jr. assumed leadership in February. This restructuring has not been without controversy, as evidenced by the recent dismissals of CDC Director Susan Monarez and NIAID head Jeanne Marrazzo.

Monarez alleges her firing was due to her refusal to pre-approve certain vaccine advisory recommendations, while Marrazzo claims her removal was retaliation for a whistleblower report criticizing the HHS leadership's stance on vaccines. These incidents have raised concerns about the direction of health policy under the current administration and its potential impact on public health initiatives and pharmaceutical regulation.