MassBio CEO & President Kendalle Burlin O’Connell originally shared this update on LinkedIn.
This week was a reminder that federal policy moves slowly until suddenly it doesn’t. SBIR reauthorization may finally be days away, MFN pricing is back in the national spotlight, and FDA is signaling real openness to modernizing how we develop therapies for the rarest diseases. Here’s where things stand on the issues we’re tracking most closely.
- SBIR Reauthorization Moves Closer to the Finish Line: After a months-long stalemate, Senate leaders are moving toward final action on an SBIR/STTR reauthorization that would extend the programs through Sept. 30, 2031. The House is expected to take up the legislation shortly after Senate passage, meaning SBIR could be turned on as soon as early next week. MassBio has been actively involved in conversations with offices for months, and we’re encouraged to finally see a path forward. Reauthorization is essential, but so is the restart. We’ll be watching closely for how quickly agencies can re-open and will be tracking proposed updates to make sure reforms strengthen, rather than slow, research for early-stage biotech companies.
- Trump Puts MFN Codification Front-and-Center in the State of the Union: During the State of the Union earlier this week, President Trump again urged Congress to codify “Most Favored Nation” (MFN) drug pricing into law. MassBio remains deeply concerned about this prospect and continues to share perspectives on how MFN-style international reference pricing would inject sweeping uncertainty into the U.S. market and undercut the investment model that funds high-risk R&D. We’ll continue engaging lawmakers on the real-world consequences for innovation, patients, and U.S. competitiveness.
- MassBio Submits GLOBE/GUARD Comments: Earlier this week, we submitted comment to CMS in response to their proposed GLOBE (letter) and GUARD (letter) models, which we believe would significantly undermine incentives for innovation in the United States and put patient access to the latest innovations at risk, without meaningfully increasing patient access or reducing patient costs.
- Senate Aging Committee Focuses on Rare Disease & FDA Predictability: This morning, the Senate Special Committee on Aging is holding a hearing focused on how FDA processes and evolving standards can unintentionally delay access, particularly for rare disease patients, while also exploring practical ways to improve predictability and regulatory clarity. The Hearing is timely as it comes during Rare Disease Week, and we appreciate seeing Massachusetts represented with Dr. Jeremy Schmahmann, MD, Director of Massachusetts General Hospital Ataxia Center, testify as at witness. MassBio welcome serious, solutions-oriented oversight in this space and look forward to working with Committee leaders and stakeholders on reforms that help patients and preserve rigorous science.
- FDA Advances an “Plausible Mechanism” Framework for Ultra-Rare Diseases: One promising development: FDA issued draft guidance on Feb. 23, 2026, describing a framework to accelerate development of individualized therapies when randomized trials aren’t feasible and where there is a plausible expectation that the treatment would work, such as understanding a specific genetic defect causing the disorder and how the treatment would address it, especially relevant for ultra-rare genetic diseases. We’re pleased to see efforts to modernize clinical trials like this, and we’ll be watching closely to ensure the framework is implemented in a clear, consistent, and workable way for developers.