Commentary
Video
Author(s):
The former FDA commissioner spoke on the need for policy and funding efforts for neuromuscular disorders, the stress on the clinical care system, and the need to acknowledge healthcare inequities. [WATCH TIME: 4 minutes]
WATCH TIME: 4 minutes
"Translational research is equally critical—to take these insights and develop technologies, from gene editing to devices, that can lead to effective interventions. But it’s not just about the science. Our healthcare system is under tremendous financial strain, and often, the patients who need care the most are not the ones who generate profit."
Recent federal funding cuts have significantly impacted healthcare organizations, notably the National Institutes of Health (NIH) and the Food and Drug Administration (FDA). In February, the NIH announced a cap on indirect cost reimbursements at 15%, down from the previous average of 27%.1 This change ultimately affects funds allocated for administrative expenses and facility maintenance, leading to concerns among academic medical centers about the sustainability of research programs. In addition, the NIH has terminated funding for various research grants, including those related to HIV studies and climate change’s health impacts.2
Several leaders from academic institutions have expressed that the funding cuts jeopardize ongoing health research, and may setback biomedical innovation and public health advancements. Robert Califf, MD, MACC, a cardiologist and former FDA commissioner, recently presented the keynote speech at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific conference, held March 16-19, in Dallas, Texas, discussing the therapeutic era for neuromuscular diseases, and the importance of continued funding. As the largest U.S. conference solely dedicated to neuromuscular diseases, the meeting highlighted the latest advancements in research, clinical care, and treatment development.
Califf, who currently serves as an instructor in the department of medicine at Duke University, sat down with NeurologyLive® at the meeting to discuss the critical areas in neuromuscular disorders where policy and funding efforts should be directed. Responding to questions about advancing care, he emphasized the ongoing need for investment in basic science, particularly in understanding gene interactions in human models, and translational research to develop technologies like gene editing and devices that enhance patient care. In addition, he discussed the stress on the clinical care system, exacerbated by financial priorities in health systems and the erosion of Medicaid funding, which disproportionately affects disabled and underserved populations.
Click here for more MDA 2025 coverage.
FDA Approves Diazoxide Choline Extended-Release Tablets for Hyperphagia in Prader-Willi Syndrome