Robert Califf, MD, MACC, a cardiologist and 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.
Robert Califf, MD, MACC
Recent federal funding cuts have significantly affected healthcare organizations, such as the National Institutes of Health (NIH) and the FDA. Concerns have been raised by several leaders of academic institutions that such funding cuts endanger ongoing health research and could hold back innovation in the field.
CGTLive®'s sister site NeurologyLive® sat down with keynote speaker Robert Califf, MD, MACC, a cardiologist and former FDA commissioner, at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific conference, held March 16-19, in Dallas, Texas, to discuss the role of funding in neuromuscular disease research. Califf gave his view on where funding is most urgently needed in this field and spoke about the stress on the clinical care system, which has been intensified by financial incentives and scaling back of Medicaid funding.
Robert Califf, MD: That's a very interesting question to be asking right now because you can pretty much take what's happening and reverse engineer it and go where I think we need to go. At the very basic level, we still need fundamental basic science, so the NIH needs to keep its funding, which is very much being threatened right now. I think particularly for the kind of diseases we're talking about at the MDA, I think we need to really be studying how genes interact and how they interact in the human condition, whether it's in human tissue, models of human tissue (which are evolving rapidly), or in the intact human being. That basic research still is very important.
We also need translational research. How do you take those ideas and develop technologies which can eventually lead to effective interventions of various types, whether it's anything from gene-editing to taking gene function and enhancing it, or knocking it down?—Or even in many of these patients, devices are very important. We didn't talk much about devices today in my part of the meeting, but if you look at people who have diminished muscular function, the device world is so absolutely critical to them, and there's a lot of work to be done there. As such, engineering is a very important part of this. And of course, bringing it all together these days is AI, which is the ultimate synthetic method of bringing multiple streams of information and data together, so we have to be funding all of that.
But then on the other side, the clinical care system is very much under stress right now, as our health systems and insurance companies have increasingly fine-tuned their effort to make a financial margin. Often the patients who need help the most are not the ones on which you make money and so we're seeing real concerns about things like Medicaid, which are essential to disabled people—such a high proportion are dependent on Medicaid funding—and that funding is diminished.
So you asked about the funding, I pretty much laid out where I think it needs to go. The opposite is sort of happening right now.
I'd also add to it the issue of diversity. That word is not even allowed now in a federal grant and yet we know that if we just look at our human population, there are groups of people who do much worse than others. For example, rural people do much worse than urban people now. Those without a college degree do 8 years shorter life expectancy compared to those with one. So if we don't acknowledge that there are real differences in expected outcomes, how are we going to fix them? I think we've got to restore funding to acknowledge the issues we have with health outcomes and then have plans to do something about it.
This transcript has been edited for clarity.
Read more coverage of the 2025 MDA Conference here.
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