MassBio CEO & President Kendalle Burlin O’Connell first posted this update on LinkedIn.
Congress is still out, but we had big news come out of the Administration this week with the President naming a nominee to lead FDA, the White House releasing a science and technology strategy that further solidifies the Administration’s treatment of biotechnology as national security infrastructure, and FDA opening the comment window on the PDUFA VIII commitment letter. Here’s what we’re watching:
Heidi Overton Nominated to Lead FDA: President Trump announced on August 19 that he is nominating Dr. Heidi Overton as Commissioner of FDA. Overton is currently deputy director of the White House Domestic Policy Council, where she has led health policy since the start of the term. She is a physician, trained at the University of New Mexico with a general surgery residency at Johns Hopkins, and served as a White House fellow in the first Trump administration before running the health policy center at the America First Policy Institute. She would succeed Marty Makary, who resigned in May. The last eighteen months produced real progress at FDA in some circumstances but were overshadowed by concerns over staff turnover, leadership shakeups, and delays. We hope this nomination signals continued commitment to gold standard science and predictable, science-based review, and we will be listening for exactly that in her confirmation testimony. The nomination now goes to Senate HELP, where Chairman Cassidy (R-LA) has repeatedly broken with the administration. Cassidy held up the CDC director nomination earlier this month before ultimately supporting Dr. Erica Schwartz. Overton stood behind the President at the August 10 signing of new vaccine framework and publicly urged states to adopt the new framework, which will draw questions from Cassidy and others. Committee Democrats, including Sen. Patty Murray (D-WA), announced opposition within hours of the announcement.
FDA Opens the PDUFA VIII Comment Window: FDA published the draft PDUFA VIII commitment letter and notice of public meeting on August 14, covering FY 2028 through FY 2032. Current authority expires September 30, 2027. According to the Federal Register notice, PDUFA VIII would include incentives designed to encourage U.S.-based drug development. These include a proposed 50% reduction in the premarket application fee for submissions that include data from a U.S.-based Phase 1 trial initiated after October 1, 2027, as well as restricting the small-business fee waiver to U.S. companies. The public meeting is September 16 and written comments are due October 16. MassBio is reviewing the commitment letter with members and will engage ahead of the October deadline.
White House Strategy Treats Biotechnology as National Security Infrastructure: The Administration released its National Security Science and Technology Strategy this week. Biotechnology is featured heavily throughout, including being included on the updated critical and emerging technologies list, with named subfields including synthetic biology, genome and epigenome engineering, protein design and engineering, novel therapeutic design and manufacturing, biomanufacturing and bioprocessing, and neurotechnologies. It is one of only three technologies, alongside AI and quantum, identified specifically as potentially transformative areas where the United States intends to lead outright. The strategy also commits to implementing and strengthening the Outbound Investment Security Program under the Comprehensive Outbound Investment National Security Act of 2025, and states plainly that the administration will expand such restrictions to cover additional areas implicated by China’s military-civil fusion strategy.
MassBio Comments on NIH’s Scientific Impact RFI: NIH’s Request for Information on Measuring and Rewarding Scientific Impact closed August 19, and MassBio filed comments. The RFI asked how the agency should measure success across different metrics. We focused on three: entrepreneurship and translation, foundational scientific exploration, and public impact. Our central argument is that the gap between what NIH measures and what NIH’s mission requires is widest in translation. An investigator whose work produces an issued patent, an executed license, a company, an IND, and a first-in-human trial has generated precisely the public value this RFI describes.
Two Comment Deadlines We’re Watching: Two FDA draft guidances with near-term deadlines. MassBio is monitoring both and welcomes perspectives from member companies. The guidances include Master Protocols for Drug and Biological Product Development, with comments due August 24. It revises the December 2023 draft and adds substantial material on basket trials alongside umbrella and platform trials, with new recommendations on randomization, choice of control, blinding, informed consent, and regulatory submission. The other is Leveraging Prior Knowledge in the Development of Human Gene Therapy Products Incorporating Genome Editing, with comments due September 1. The proposal clarifies when sponsors of ex vivo and in vivo somatic genome editing products may rely on existing CMC, nonclinical, and clinical knowledge to reduce or eliminate the need to generate new data.
