Eye on FDA

On June 24, 2026, the Food and Drug Administration (FDA) published a notice of availability in the Federal Register requesting information and public comment on an Expedited Investigational New Drug (IND) pilot program. The proposed pilot program is part of the Trump administration’s focus on taking steps to strengthen America’s leadership in clinical research, accelerate the development of lifesaving treatments and ensure that patients have access to the most innovative therapies in the world.

On May 12, 2026, the U.S. Food and Drug Administration (FDA) announced a major milestone in its approach to post-market oversight of chemicals in the food supply, finalizing a new proactive food chemical safety post-market assessment program and releasing two foundational papers: the “Enhanced Systematic Process for Post-Market Assessment of Chemicals in Food” and the “Tool for the Prioritization of Food Chemicals for Post-Market Assessment.”

In December 2016, the bipartisan 21st Century Cures Act (P.L. 114-255) was enacted, marking a pivotal milestone in advancing a patient-focused drug development (PFDD) paradigm. This law directed the Food and Drug Administration (FDA) to develop and implement strategies to solicit view of patients during the medical product development process and consider the perspectives of patients during regulatory discussions. Over the past decade, FDA has steadily taken steps to implement the 21st Century Cures Act patient-focused drug development provisions, including through meetings by which patients, caregivers, family members and patient advocates, among others, have been able to provide information about patients’ experiences with a disease or condition. The opportunity to provide such feedback through these patient-focused meetings is a key pillar of the agency’s patient-focused drug development engagement.

On April 28, 2026, the Food and Drug Administration (FDA) unveiled two major announcements as part of its initiative to “advance the implementation of real-time clinical trials.” In conjunction with a request for information (RFI) for a proposed pilot program, the agency announced the successful initiation of two proof-of-concept real-time clinical trials (RTCTs). Taken together, the agency’s stated goal is to enable continuous trials and reduce the “hiatuses” between the phases of clinical development, ultimately improving the drug development process.

On March 20, 2026, the Food and Drug Administration (FDA) announced the agency will hold a public hearing on the Commissioner’s National Priority Voucher (CNPV) Pilot Program, with a Federal Register notice soliciting public comment and scheduling a public hearing for June 12, 2026. As previously noted here and here, the program was announced in June 2025 for the purpose of providing select sponsors with a nontransferable voucher for enhanced engagement with FDA and expedited review for drugs and biologics supporting one or more critical national health priorities, while maintaining adherence to the law’s rigorous safety and effectiveness standards. The national health priorities include public health crisis response, innovative breakthrough therapies, large unmet medical needs, onshoring and supply chain resilience initiatives, and affordability improvements. To date, FDA has issued 18 Commissioner’s National Priority vouchers and approved four products to receive these vouchers, including two oncology drugs 44 and 55 days after filing.

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.

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.