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.

Getting patients more involved in the process of developing new drugs is a goal easy to set and hard to achieve, analysts said during a panel on patient engagement at the Milken Institute’s Future of Health Summit on Tuesday.

On the plus side, it’s a unifying goal with Congress’s backing. Lawmakers tasked the medical industry with doing more to incorporate patient feedback in the 21st Century Cures Act of 2016 and the FDA Reauthorization Act of 2017.

Panelists at the summit said patients are becoming more engaged, but that product developers are still struggling to diversify clinical trials. Because drugs and devices sometimes don’t work as well on all people, that puts a limit on the quality of new medical products.

Panelists offered a number of ideas for further improvement:

• Rachele Berria, vice president of U.S. medical, biopharmaceuticals, at drugmaker AstraZeneca, called for more use of wearables and telehealth in trials, as well as embedding trials in the community and having experts from diverse backgrounds design trials. 

• Theresa Mullin, associate director for strategic initiatives at the FDA's Center for Drug Evaluation and Research, said standards for digital health technologies need to be harmonized globally.

• Yvette Venable, vice president of patient engagement at the Institute for Clinical and Economic Review, a nonprofit that evaluates the cost effectiveness of medical products, said better data needs to be collected. 

The pandemic-induced adoption of virtual formats showed product makers how ease-of-access can help boost the diversity of patients taking part in trials, Mullin said.

But clinical trials still struggle to replicate real-life conditions, said Mary Dwight, senior vice president of policy and advocacy at the Cystic Fibrosis Foundation.

She urged colleagues to focus on innovation, asking them to keep thinking about: “How do you marry the data we need and collect very robustly for clinical trials and...bring it on into the real world?”

This is where we explore the ideas and innovators shaping health care. 

The Milken Institute really brought the goods with its avocado toast buffet. We’re glad to see the millennial favorite has finally made its way to the health care conference circuit. How do you like yours? With eggs? Bacon? Goat cheese?

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Today in our Pulse Check podcast, Ben talks with POLITICO's Erin Banco about her investigation into why the FDA team overseeing licensed vaccines is overwhelmed by high turnover and a backlog of inspections. Plus, Future Pulse editor Shawn Zeller previews the Milken Summit.

Gene therapy could be a game changer for patients who suffer from diseases like sickle cell, but it’s unclear whether Medicaid will cover it once it’s FDA-approved.

That was one of the dilemmas debated at a Milken Summit panel on value-based payment models.

The gene therapy treatment is likely to cost around $1 million. About 100,000 Americans have sickle cell. Is providing them what could be a functional cure for the disease worth the steep price?

Maia Z. Laing, vice president of health equity at Optum, which provides data, analytics and other services to health care providers, said it’s a clear yes.

“It is abusive for the sickle cell community to have to go through the level of waivers and step therapies for them to have to get to medications that will best address their needs,” she said.

What’s at stake: Sickle cell disease causes anemia and extreme pain. It afflicts Black people at disproportionate rates. Roughly 40 percent of people with the disease qualify for Medicaid coverage.

Studies of gene therapy show promising results: The therapy can heal blood cells that carry the disorder and relieve symptoms.

Cost quandary: Jack Rollins, director of federal policy at the National Association of Medicaid Directors, said that in the past, the Centers for Medicare and Medicaid Services has effectively had to wait for prices to drop before broadly covering expensive treatments, but gene therapy could stay expensive.

To fix the problem, he said, CMS needs to get creative about financing to avoid that enormous upfront cost. “Can some of that cost be amortized?” he asked.

What’s next: CMS is thinking about it. The agency is undergoing a strategy refresh that includes changing how it thinks about data and measuring patient outcomes. The goal is to evaluate health care services to ensure better quality care for patients and potentially more informed decision-making on coverage. But that requires data on patient experience.

The FDA’s Accelerated Approval Program has brought life-saving medicines to market years before they would typically have cleared clinical trials.

But the agency has also approved drugs that didn’t fulfill their promise, giving patients false hope and adding expense to the health care system.

Milken Summit panelists argued about how to balance those competing outcomes.

“The FDA is not holding companies’ feet to the fire as much as I think they ought to be,” said Holly Fernandez Lynch, an assistant professor of medical ethics at the University of Pennsylvania.

Fernandez Lynch argued that drug companies should include confirmatory trial designs and criteria in their applications — the companies must complete those trials after they win approval for their products — and she backed automatic expiration dates for confirmatory trials.

But Frank Sasinowski, a director at law firm Hyman, Phelps & McNamara and a former FDA deputy director of health policy, disagreed, advocating for more flexibility in the confirmatory trial process, especially for rare and slow-progressing diseases.

“I don’t know that we can put in the hard, fast law, you know, that you can’t have accelerated approval without having the [confirmatory] studies already underway because sometimes the FDA responses are going to need to negotiate what the right design is,” he said.

How it works: Accelerated approval, which the FDA launched in the 1990s as a response to the HIV/AIDS epidemic, gets drugs that treat serious diseases to market quickly by relying on “surrogate endpoints,” or markers from imaging or laboratory measurements instead of from clinical trials. Drug makers must conduct confirmatory trials to demonstrate the efficacy of their product once it’s on the market.

The trade-off is early patient access to potentially life-changing drugs, usually for cancer or rare diseases — 88 cancer treatments have been approved and shown clinical benefit since the program's inception — in exchange for less certainty about whether a drug will work in the real world.

Recent developments: POLITICO’s Lauren Gardner reported on an FDA panel’s recommendation to pull Makena, a drug approved via accelerated approval in 2011 to reduce risk of preterm birth, from the market because of its failure to show clinical benefit. The decision put a spotlight on how long a drug can continue to be sold without evidence that it works.

Congress is also considering adding language to a fiscal 2023 appropriations bill this month to tighten the rules around accelerated approval.