Trish Brown, Senior Vice President, Payer Relations and Reimbursement at GeneDx, shared a post on LinkedIn:
“I had the pleasure today to attend the FDA announcement at HHS with Linda Genen on the new Plausible Mechanism framework; this reflects a meaningful shift in how we approach ultra‑rare disease innovation. Thank you to the U.S. Department of Health and Human Services (HHS), FDA, and their visionary leaders for this important work.
By acknowledging that traditional randomized trials are often infeasible for ultra‑rare conditions, regulators are creating space for individualized therapies to reach patients. But regulatory progress alone isn’t enough.
Coverage and reimbursement frameworks must evolve alongside approval pathways—aligning evidence expectations with scientific reality, leveraging real‑world data, and reducing administrative delays.
You can’t treat what you can’t diagnose. Individualized therapies depend on identifying the precise genetic drivers of disease, yet too many patients still lack access to high‑quality genomic testing due to coverage and policy barriers.
Expanding access to timely, appropriate testing is foundational-not only for clinical impact, but for making these therapies economically viable.
Encouraging regulatory leadership is an important step. Ensuring that diagnostic and access infrastructure keeps pace with innovation is the work ahead. You can help by joining us in supporting the The Genomic Answers for Children’s Health Act (H.R. 7118) to ensure that children with undiagnosed rare disease get access to genetic testing and the answers they deserve.”
Other articles featuring Trish Boh on OncoDaily.