| | | | |  | | By David Lim , Ben Leonard , Carmen Paun , Grace Scullion and Ruth Reader | POLITICO is partnering this week with the Milken Institute to bring a special edition of Future Pulse to the Milken Institute Future of Health Summit. We are taking readers inside one of the most influential gatherings of global health industry leaders and innovators as they tackle today's pressing health challenges.
| | | |  FDA Principal Deputy Commissioner Janet Woodcock | Getty Images | Only about 5 percent of Covid-19 clinical trials aimed at identifying or developing treatments were rigorous enough to produce quality evidence, FDA Principal Deputy Commissioner Janet Woodcock said during a Milken Summit panel discussion on improving the clinical trial enterprise. "We hardly got any evidence … because it was every person for themselves setting up these little, as I call them, small crappy trials," Woodcock said. She added that researchers struggled because they were competing for patients. Though clinical trials for mRNA vaccines and therapeutics like Paxlovid passed FDA muster and resulted in groundbreaking treatments, many others didn't. The longtime FDA regulator said efforts to make clinical trials more affordable, expand the use of remote access to trials and increase the use of platform trials, in which researchers compare multiple intervention groups with one control group, are needed. "I've been preaching the doctrine of the platform trials for … 12 years now in every venue I could," Woodcock said. "But now it's real, and people are doing it and the results are clear."
| | | | GO INSIDE THE MILKEN INSTITUTE FUTURE OF HEALTH SUMMIT: POLITICO is featuring a special edition of our Future Pulse newsletter at the 2022 Milken Institute Future of Health Summit from Dec. 6 to 8. The newsletter takes readers inside one of the most influential gatherings of health industry leaders and innovators solving the biggest global health issues to ensure a healthier, more resilient future for all. The special edition will be the only Future Pulse newsletter sent on December 6-8. As a subscriber to Future Pulse, you will receive the special edition version for those two days. Learn more about the Milken Institute's Future of Health Summit . | | | | | | | | | 
White Mountain National Forest, New Hampshire | Shawn Zeller | This is where we explore the ideas and innovators shaping health care. Who's to blame for the increase in obesity and diabetes in America? The tech giants are, according to George Yancopoulos, the president and chief scientific officer of Regeneron Pharmaceuticals. "Their whole goal is to addict us to behaviors that promote sedentary behavior that lead to neuropsychiatric disorders and are becoming the leading cause of obesity and diabetes," Yancopoulos said. Do you agree? Share news, tips and feedback with Ben Leonard at bleonard@politico.com, Ruth Reader at rreader@politico.com, Carmen Paun at cpaun@politico.com or Grace Scullion at gscullion@politico.com. Send tips securely through SecureDrop, Signal, Telegram or WhatsApp. Today in our Pulse Check podcast, Ben talks with Steve Posnack, HHS' deputy national coordinator for health information technology, about what he thinks the biggest barriers are to people getting their health data. Plus, Jack Resneck, the president of the American Medical Association, talks about the nearly 8.5 percent Medicare payment cut for doctors that will take effect in January unless Congress acts.
| | | | | | |  Medical records are going digital and providers are now required to give patients access to theirs. | AP | It's getting easier for patients to access their health data since a new federal regulation took effect this fall that bars providers from withholding them, but it's still a significant challenge. Data is often siloed in different systems, and sometimes patients need to lean on antiquated technology like CDs to get their records. Milken Summit panelists had plenty of gripes about it:
- "It's insane to me that it isn't more fluid," said Anna McCollister, an independent consultant. "It's because people haven't chosen to make it a priority. It's not the people in government; it's people in the private sector."
- Harlan Krumholz, a professor of medicine at Yale and director of the Center for Outcomes, Research and Evaluation, said he's frustrated that the medical industry often waits for mandates instead of "saying this is the right thing to do" and making it easier to access data.
- Payment incentives are misaligned with increased data sharing, said moderator Deven McGraw, lead of data stewardship and sharing at biotech firm Invitae.
- "We're not paying physicians to do that in clinical care," said Elise Berliner, global senior principal of real world evidence strategy at Cerner Enviza.
Why it matters: Congress ordered the Department of Health and Human Services to bar providers from hoarding patient data in a 2016 law, and HHS mandated they comply starting last October. If providers do, it should help patients as they shop around for medical care. Panelists said they had hope for the future: Steve Posnack, deputy national coordinator at HHS's Office of the National Coordinator for Health IT, told Future Pulse he's optimistic federal regulations will help patients obtain their data. And Peter Levin, the CEO of data firm Amida, said that, with the rules and the technology now in place, it's up to patients to push providers to make it happen.
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| The health care industry needs to think big — and move urgently — to overhaul a research system that's broken for lack of diversity, Donna Cryer, the president and CEO of the Global Liver Institute, an international patient advocacy organization, said during a summit session on improving health equity in rare disease research. But clinical research isn't conducted in communities of color, even though they have the settings, such as federally qualified health centers or churches, where they could be established. "How can we create a clinical research enterprise that people want to participate in, that they feel reflects them?" Cryer asked. Giving up on the stereotype that minority patients don't want to participate in clinical trials will also help improve diversity, said Tamar Thompson, head of global corporate affairs at Alexion, an AstraZeneca subsidiary. "Often, they're not asked," she said. Thompson acknowledged the abuses against Black people in medical research, like in the cases of Henrietta Lacks and the syphilis study at Tuskegee , but said there are people of color willing to help study new treatments. "Some of those historical stereotypes are just that," Thompson said.
| | | |  Retailing giant Walmart is getting into the clinical trials business. | Getty Images | Walmart hopes to be part of the solution when it comes to clinical trials, its chief medical officer, John Wigneswaran, said. Since the company announced the launch of its Healthcare Research Institute in October, with the goal of increasing participation of older adults, rural residents, women and minority populations in studies on treatments for chronic conditions, Wigneswaran said 5 million people have told the company they want information about participating. Touting the retailers' reach across the country — a Walmart within 10 miles of 90 percent of the population — Wigneswaran said this should help solve the diversity gaps in clinical trials. In 2020, 75 percent of clinical trial participants were white, 11 percent Hispanic, 8 percent Black and 6 percent Asian, Walmart said in October, citing Food and Drug Administration data. "So one of the things that we're trying to hold ourselves to is to try to think about 'great, you recruited someone, but what is the long-lasting impact in the community after,'" Wigneswaran said.
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Califf | Drew Angerer/Getty Images | | | "Even today, in 2022, we have 47 million Americans using tobacco products; we're gonna lose almost 500,000 Americans this year to tobacco-related disease." – Robert Califf, FDA Commissioner | | Decreasing tobacco use represents the greatest opportunity, aside from Covid-19 vaccination and treatment, to quickly reduce the number of people dying in the U.S. from preventable disease, FDA Commissioner Robert Califf told Milken Summit attendees. To put it in perspective, the half-million tobacco-related deaths Califf predicts would be nearly double this year's toll from Covid. Overall tobacco use was declining before the pandemic, according to the Substance Abuse and Mental Health Services Administration. But the FDA and CDC reported in November that more than 3 million middle and high school students had used a tobacco product in the past month, with flavored e-cigarettes the biggest draw. The agencies said differences in survey methodology related to the pandemic make it impossible to fully compare those results to those in the 2021 National Youth Tobacco Survey. The 2021 survey found that 2.55 million children reported using tobacco products in the past 30 days. Last spring, using authority Congress granted in the 2009 Family Smoking Prevention and Tobacco Control Act, the FDA proposed banning menthol cigarettes, the last flavored variety the agency allows. In June, using a different authority in the 2009 law, the FDA ordered all products made by the once-popular e-cigarette maker Juul from the market. It rescinded that order a month later, saying it needed to conduct a scientific review.
| | | | POLITICO AT CES 2023 : We are bringing a special edition of our Digital Future Daily newsletter to Las Vegas to cover CES 2023. The newsletter will take you inside the largest and most influential technology event on the planet, featuring every major and emerging industry in the technology ecosystem gathered in one place. The newsletter runs from Jan. 5-7 and will focus on the public policy related aspects of the event. Sign up today to receive exclusive coverage of CES 2023. | | | | | | | | |  Remdesivir, originally approved to treat hepatitis C, was found to be effective against Covid-19. | AP | We're in a golden age of drug repurposing, said Joni Rutter, director of the National Center for Advancing Translational Sciences at the National Institutes of Health, during a Milken Summit panel on the subject. "Over the last three years, I think we have experienced the most intensive time of drug repurposing that we've ever seen," she said. Many Covid-19 treatments were initially developed to treat other conditions, like malaria, influenza and parasitic infections, and the flurry of research on their efficacy represents a bright spot amid the pandemic, added David Fajgenbaum, who directed the CORONA insights project to find Covid treatments. "If we can recreate what we did for Covid for a lot of other diseases, that would be an incredible outcome of this horrible pandemic," he said. Fajgenbaum has since founded Every Cure, a data repository designed to accelerate the identification of existing drugs to treat rare diseases. Why it matters: While drug repurposing research and development flourished for Covid during the pandemic, rare diseases don't receive that same attention. And though those diseases may be rare, their impact is sweeping: One in 10 Americans has a rare disease, and 95 percent of rare diseases have no treatment, according to the National Organization for Rare Disorders. "Cures sit on the shelves for decades because of systemic barriers," said Fajgenbaum. The main barrier? Money. Repurposing a drug costs an average of $40 million to $80 million, and it can still take upwards of a decade for such a drug to reach the market, according to Pharmacy Times. For rare diseases especially, pharmaceutical companies hesitate to invest millions in small patient populations. "These are business decisions; they are not medical decisions," said Mike Rea, CEO and co-founder of IDEA Pharma. Milken panelists offered potential solutions: - Peter Stein, director of the Office of New Drugs at the FDA, said his office can lower the regulatory requirements for drugs treating rare diseases by accepting persuasive evidence in lieu of costly clinical trials.
- Fajgenbaum said leveraging nonprofit foundation dollars can help get repurposed drugs to the market when private investment isn't there.
- Rea advocated for a "biotech social compact" for pharmaceutical companies that would enforce deadlines for brand-name drugs to go generic.
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