MassBio CEO & President Kendalle Burlin O’Connell originally posted this update to LinkedIn.
Policy was certainly on the minds of attendees at yesterday’s State of Possible Conference, including the current regulatory environment. FDA has been active with announcements in recent weeks, and we’re encouraged to see the agency issuing forward-looking guidance and convening stakeholders. But announcements are not operations, and we continue to hear from members that day-to-day uncertainty at FDA is creating real friction in drug development timelines. Here’s what’s on our radar this week:
FDA Schedules Public Hearing on Commissioner’s National Priority Voucher Program: On June 12, FDA will hold a public hearing on the Commissioner’s National Priority Voucher (CNPV) Pilot Program, a new incentive mechanism aimed at spurring development of high-priority treatments. The program has come under fire from some, including certain members of Congress who have questioned whether the program is within FDA’s scope. MassBio will be monitoring stakeholder input closely and engaging members as details emerge.
FDA Workshop on Pediatric Cell & Gene Therapy: On April 9, FDA is convening a scientific workshop on advancing clinical trials for pediatric cell and gene therapy, with a focus on scientific, ethical, and regulatory challenges. This reflects growing FDA attention to one of the most complex and promising frontiers in medicine, and one where Massachusetts companies are leading globally. Regulatory clarity in this space is essential to maintaining our competitive edge, and we welcome the agency’s engagement with the field.
FDA Releases Draft Guidance on Alternatives to Animal Testing: FDA released draft guidance on alternatives to animal testing in drug development, signaling continued momentum toward modernizing preclinical research and development. The draft guidance established four validation principles for New Approach Methodologies (NAMs): Context of Use; Human Biological Relevance; Technical Characterization; and Fit-For-Purpose Assurance. Comments on the guidance are due May 18.
Data Shows Stalled NIH Grant Funding: March 31 marks the midpoint of the federal fiscal year and NIH has reportedly obligated only around 15 percent of its estimated $38 billion in grants and contracts to universities and research institutions, according to data from the Association of American Medical Colleges. Though the stalled funding is due in part to the historically long government shutdown, it is a striking figure that deserves serious attention. Massachusetts institutions are among the largest recipients of NIH funding in the nation. Delays in grant obligations threaten to slow the basic research pipeline that feeds the next generation of Massachusetts biotech companies. We’ll be monitoring closely for whether funding ticks up over the coming months.
CDC Director Search Postponed: The White House indefinitely postponed its selection of a permanent CDC director this week, leaving the agency without a full-time political leader for the moment. The CDC has been without a Senate-confirmed director since August, and has now cycled through multiple acting leaders, a prolonged leadership vacuum that public health experts have flagged as deeply problematic. For Massachusetts life sciences companies developing vaccines, diagnostics, and infectious disease therapeutics, a functional and stable CDC is a core part of the regulatory and public health infrastructure our industry depends on.
Reconciliation 2.0 Taking Shape and We’re Watching Drug Pricing: House and Senate Budget Committee chairs met this week to begin planning a second reconciliation bill, with the early focus on defense and immigration funding. While drug pricing is not the stated driver of this vehicle, the broader environment, including the administration’s MFN push for codification, means we are watching closely to ensure any reconciliation package does not become a vector for drug price controls that would undermine the innovation ecosystem. MassBio will continue to engage aggressively on this front.
SBIR/STTR Update: While we’re thrilled to see House and Senate finally pass legislation to reauthorize SBIR and STTR programs, we understand the legislation has not yet been presented to the President for signature. This delay is due in part to President Trump’s unwillingness to sign any legislation until SAVE Act is passed by Congress. We remain optimistic that the President will sign the legislation once the impasse is resolved, but exact timing remains a question at the moment.