Robert M Califf, MD, MACC, on Urgency of Funding and Equity in Neuromuscular Research and Care
The former FDA commissioner discussed the necessity of policy and funding efforts for neuromuscular disorders, the stress on the clinical care system, and the need to acknowledge healthcare inequities.
This video originally appeared on our sister site, NeurologyLive®.
"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."
Healthcare organizations, such as the National Institutes of Health (NIH) and the FDA, have been significantly affected by recent federal funding cuts. The NIH announced in February the capping of indirect cost reimbursements at 15%, a drop from the previous average of 27%.1 Because funds allocated for administrative expenses and facility maintenance are ultimately impacted by this change, academic medical centers have become concerned about research programs' sustainability. Furthermore, funding for various research grants, including those related to HIV studies and climate change’s health effects, has been terminated by the NIH.2
The funding cuts threaten ongoing health research and could set biomedical innovation and public health advancements back, according to concerns expressed by several leaders of academic institutions. At the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific conference, held March 16-19, in Dallas, Texas, keynote speaker Robert Califf, MD, MACC, a cardiologist and former FDA commissioner, spoke about the therapeutic era for neuromuscular diseases, and the importance of ongoing funding. The conference, which is the largest medical meeting in the United States entirely dedicated to neuromuscular disease, 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 CGTLive®'s sister site 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.
