Image courtesy of Robert Califf; design by Cure.
Overview
Former FDA Commissioner Robert Califf, MD, outlines the biggest roadblocks facing biotech today. From trust and primary care gaps to regulatory instability—and where innovation is most needed.
Few people have a clearer, more longitudinal view of the biomedical landscape than Robert Califf, MD. A longtime Duke professor and one of the most cited researchers in clinical science, Califf led the FDA under both the Obama and Biden administrations. This afforded him a front-row seat to the forces now reshaping U.S. healthcare, from chronic-disease burdens to regulatory instability to a deepening crisis in public trust.
At the recent X Factor Healthcare Innovation Summit at Cure, he joined Cure CEO Seema Kumar for a candid conversation about the biggest obstacles facing biotech founders today and what it will take for the United States to maintain global leadership in biomedical innovation.
The excerpts below have been adapted from the panel discussion and edited for length and clarity.
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When you look big-picture at global healthcare needs, what do you see as the most significant challenges—and where could innovation make the biggest difference?
If you look at the U.S. population, our biggest health problem is wealth disparity. The trends are unmistakable—we’re in last place among high-income countries in longevity and functional status. That’s because of the growing difference in access to basic things like the ability to exercise or eat healthy food. A lot of Americans simply can’t afford healthy food right now.
And if you look at what’s causing death and disability now, it’s chronic diseases. That’s here in the U.S. and increasingly in low-income countries, as infectious diseases decline. We already have effective treatments for most of them, but they’re not fully effective and there’s still a long way to go.
You might ask, “What does that have to do with innovation?” Well, this is a wide-open space for digital technology. Technology has to facilitate human interaction—we’re not going to fix this with just more people, not with the numbers we’re facing: 320 million people, an aging population, and an explosion of diagnosable conditions thanks to advances in genomics. Leaders of our big academic centers need to focus on primary care right now.
Speaking of primary care, there is a public trust problem with the healthcare system. Why is trust so low, and what needs to change?
If you ask, “Why shouldn’t people trust the American healthcare system?” my answer would be: they shouldn’t. No one can get an appointment. Even families of prominent faculty at academic institutions can’t get care unless they pay for concierge primary care. Meanwhile, systems advertise that they’re “patient-centric.” I can’t find a disabled person who believes the healthcare system they’re in is actually patient-centric.
If you say one thing but people experience the opposite, mistrust is the logical result. If you want to build trust, you need to act trustworthy.
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For innovators facing regulatory hurdles, reimbursement uncertainty, or the valley of death, how do they keep going? What practical advice do you have?
One of the most important things: When you have a target, go ahead and write what the package insert should look like. Who is this product for? What’s the clinical need? What will the conditions of use be? One of the big lessons from the patient-focused drug development movement is that innovators are often thinking from the perspective of what their interest is and not about what the patients need. And there's a mismatch there. Keep your focus on what the clinical need is, and you’re more likely to stay on target and make it.
And most important: Be tenacious. If you think about a surgeon, once they’re in the operating room, you do not want the surgeon to be thinking, Should I really do this operation? I think it's a very similar thing with innovators. Once that decision is made to do it, you have to believe in what you're doing, and you can't be shy about it. You have to go out there and sell what you're doing. Don't worry that you're overstepping your boundaries, because there are people like me to keep you from doing things you shouldn't do. But you're talking to investors. They're investing other people's money. You’ve got to really convince them that what you have to offer is going to make a difference.
What about the explosion of wellness products, supplements, and biologic injections being marketed online—often unregulated?
If we look at the dietary supplement area, the FDA can’t, by law, intervene in the free market, so you have all kinds of crap being sold on the internet. When a 74-year-old man sees something that says “Good for prostate health,” he naively assumes that somebody competent looked at the product and concluded that the benefits outweighed the risks. But that is not the case. We have no idea.
This is an enormous growing industry. In Los Angeles, you can pay $10,000 to get a protein injection. That's a biologic—completely untested—but since it's not advertised as a drug, it's not regulated. We need to push things down the avenue of evaluating risk and benefit through adequate studies.
How should the U.S. be thinking about global health, especially with recent cuts to global health aid?
The United States' role matters enormously. And I can say bluntly: We’re killing a lot of people. Not actively with our hands, but by withdrawing so precipitously, people are dying at alarming rates.
I agree that other countries need to step up. We shouldn’t create permanent dependence on the U.S. But I think it's unconscionable the way this was done. This is a disaster.
There’s anxiety about the U.S. losing its leadership position in biomedical innovation. Are we at risk?
Every pillar of the innovation ecosystem in the U.S. is under attack now.
We don’t know how the NIH is going to turn out—it’s going to be a battle between the Senate and the president as to how it comes out.
We’ve had instability at the FDA. Political appointees are stepping in at the last minute and changing decisions on individual products after ten years of development. That simply has not happened in any other administration.
The bright side is, we all knew before this that there were a lot of issues that were not being addressed because things had gotten fairly ossified. Now it's been shaken up. Humpty Dumpty is the right analogy. We haven’t hit the floor quite yet, but it could happen this winter if we get a severe triple-demic. With our low vaccination rates, places like New York could end up with numbers like during the pandemic, when there's no place to put the bodies.
And then it's going to be the job of everybody to figure out what a better system would be. Not the old one, and not the direction things are going now, but something that works better.
