FDA Continues Focus on Rare Disease Drug Development, Announces Evidence Principles

FDA recently released two draft guidance documents for industry related to cell and gene therapy (CGT) products as the agency seeks to advance the efficient development and review of safe and effective CGT products and fulfill related commitments set forth in the 2022 reauthorization of the Prescription Drug User Fee Act (PDUFA VII). This latest guidance may be of particular interest to CGT developers and various patient groups.

On Tuesday, September 23, 2025, FDA published guidance titled “Consideration of Enforcement Policies for In Vitro Diagnostic Tests During a Section 564 Declared Emergency.” The guidance, a draft of which was published on May 6, 2024, specifies the factors that FDA intends to assess in deciding whether to issue an enforcement discretion policy with regard to in vitro diagnostics (IVD) manufacturers offering unapproved IVDs, among other devices, during a declared emergency. These factors include:

Last week, the U.S. Food and Drug Administration (FDA) issued a proposed rule to revoke the color additive listing for Orange B, a synthetic dye historically used on the casings and surfaces of frankfurters and sausages. This action is part of the broader “Make America Healthy Again” (MAHA) initiative to phase out petroleum-based foods from the American food supply by the end of 2026 and efforts to modernize the agency’s regulations.

The White House’s Make America Healthy Again (MAHA) Commission, led by the Department of Health and Human Services (HHS) Secretary Robert F. Kennedy Jr., released its highly anticipated strategy outlining a multi-agency approach focused on addressing childhood chronic disease. The strategy builds on the Commission’s inaugural health assessment, a report which examined the rising rates of childhood chronic diseases in the country and identified four primary drivers: poor diet due to consumption of ultra-processed foods (UPFs), exposure to environmental chemicals; increased technological use; and overmedicalization. To combat these challenges, the strategy released on September 9, 2025, outlines a four-prong approach which provides further insight into the areas the administration sees as key areas of MAHA going forward: advancing research, realigning incentives, increasing public awareness and fostering private sector collaborations.

On September 3, 2025, the Food and Drug Administration (FDA) announced Rare Disease Evidence Principles (RDEP) with processes aimed at providing greater predictability and facilitating the development and review of drugs intended to treat rare diseases with very small patient populations and significant unmet medical needs driven by a known genetic defect. In conjunction with the agency’s announcement of the new processes jointly proposed by the Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER), the agency also unveiled additional information regarding eligibility and the potential for post-marketing requirements for those sponsors who participate in it.

Food dye reform has been an area of focus for the Trump administration as part of its Make America Healthy Again (MAHA) initiative, giving momentum to an effort the Food and Drug Administration (FDA) had already started by banning Red No. 3 for use in food and ingested drugs. FDA has continued to prioritize this work by taking actions to phase out the use of synthetic dyes and move to natural dyes since calling for these changes earlier this year, as discussed in our previous blog on the related announcement by the administration.

Last week, former Food and Drug Administration (FDA) Commissioner David Kessler submitted a citizen petition urging FDA to revoke the generally recognized as safe (GRAS) status of refined carbohydrates used in industrial food processing. The petition follows FDA’s and the United States Department of Agriculture’s (USDA) joint request for information (RFI) to establish a formal definition for ultra-processed foods (UPFs) and offers FDA a legal pathway to change the regulatory status of these ingredients.

On August 6, 2025, the U.S. Food and Drug Administration (FDA) became the latest governmental agency to confirm that fluoropolymers are both safe and necessary. After an independent safety review of fluoropolymers in medical devices showed no conclusive evidence of health issues, FDA concluded that fluoropolymers are “very unlikely to cause toxicity” because of molecular size and further that they are essential for medical devices to function. As a result, “FDA’s evaluation is that currently there is no reason to restrict their continued use in devices.” This announcement will be a source of relief to the device and diagnostics industries.