Eye on FDA

On February 23, 2026, the Food and Drug Administration (FDA) released a draft guidance entitled “Considerations for the Use of the Plausible Mechanism Framework to Develop Individualized Therapies that Target Specific Genetic Conditions with Known Biological Cause.” This guidance is designed for targeted, individualized therapies for which randomized controlled trials are not practical due to a small patient population, and was previewed by the Commissioner in several public forums.

On February 3, 2026, the Consolidated Appropriations Act, 2026 (P.L. 119-75) became law and marked an important milestone in the bipartisan congressional effort to reauthorize the rare pediatric disease priority review voucher program. Section 6604 of this law extends the Food and Drug Administration’s (FDA) authority to issue priority review vouchers to encourage treatments for rare pediatric diseases through September 30, 2029.

In case you missed it, Department of Health and Human Services Secretary Robert F. Kennedy Jr. and former Food and Drug Administration (FDA) Commissioner David Kessler appeared on 60 Minutes this past Saturday, February 15, in a segment titled “Generally Recognized as Safe.” As the title suggests, the conversation centered on FDA’s Generally Recognized as Safe (GRAS) regulatory pathway (for background, see our prior post discussing in more detail the GRAS process), as well as Commissioner Kessler’s August 2025 citizen petition urging FDA to revoke the GRAS status of refined carbohydrates used in industrial food processing (discussed in more detail in our earlier coverage of the petition).

On December 18, 2025, the Food and Drug Administration (FDA) released a final guidance entitled “Processes and Practices Applicable to Bioresearch Monitoring Inspections.” The guidance was issued to comply with the Food and Drug Omnibus Reform Act of 2022, which directs FDA to issue guidance describing the processes and practices applicable to inspections of sites and facilities inspected under FDA’s Bioresearch Monitoring (BIMO) inspection program, to the extent that is not covered in already available FDA guides and manuals. The BIMO program was established to assess and monitor the conduct and reporting of FDA-regulated research as well as postmarketing activities through on-site inspections, investigations and Remote Regulatory Assessments.

On December 5, 2025, FDA announced its Technology-Enabled Meaningful Patient Outcomes (TEMPO) for Digital Health Devices Pilot, a new voluntary pilot that seeks to accelerate innovation and expand access to digital health devices for people living with chronic conditions. Under TEMPO, FDA will evaluate a new, risk-based enforcement approach that supports digital health devices intended for use to improve patient outcomes in cardio-kidney-metabolic, musculoskeletal and behavioral health conditions. Under the pilot, participating manufacturers may request that the agency exercise enforcement discretion for certain requirements, such as premarket authorization and investigational device requirements, while manufacturers collect and share real-world data demonstrating the device’s performance.

Last week, FDA released draft guidance titled “Scientific Considerations in Demonstrating Biosimilarity to a Reference Product: Updated Recommendations for Assessing the Need for Comparative Efficacy Studies.” This draft guidance reflects an evolution in FDA’s approach to determining whether a comparative clinical study with efficacy endpoints (a comparative efficacy study or CES) is necessary to support a demonstration of biosimilarity. Specifically, the agency notes that a comparative analytical assessment (CAA) is generally more sensitive when it comes to detecting differences between products than a CES.

On October 23, 2025, FDA released its final guidance regarding Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments. The guidance is the third guidance in a four-part series of FDA guidance focused on patient-focused drug development (PFDD) that describe how stakeholders, such as patients, caregivers, researchers, medical product developers and others can submit patient experience data and other relevant information that can be used for medical product development and regulatory decision making.

On October 16, 2025, the Food and Drug Administration (FDA) unveiled the first group of nine voucher recipients under the Commissioner’s National Priority Voucher (CNPV) pilot program, a program announced by the agency earlier this year that provides a path for accelerated drug review for companies supporting national interests as determined by the Commissioner. The identified areas of priority by the agency include meeting large unmet medical needs, bolstering domestic manufacturing and increasing the affordability of medicines for American patients. As previously noted, the new program, which is not defined in statute or regulations, aims to significantly speed up FDA’s standard 10-12 month review timeline to just 1-2 months after filing an application for a drug or biologic. The agency has touted the  benefit of recipients of the vouchers receiving enhanced access with FDA review staff and a “team-based review” model.

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.