By David Lim and Katherine Ellen Foley

Presented by 340B Health

MEDIOCRE METRICS: In the past seven years, the FDA has increased the number of neurological disease drugs approved under its accelerated approval pathway. Some of the drugs approved were the first treatments of their kind. But some experts fear this pathway — intended to get patients without options earlier access to new drugs — may not be helping patients.

Accelerated approval relies on proxy, or alternative, metrics in early-stage clinical trials. If drugmakers can show that a new treatment improves a proxy metric that suggests potential patient benefits, the FDA can conditionally approve it to get it to patients sooner. For conditions like HIV or cancer, proxy metrics have been good predictors of a drug’s merit.

But proxy metrics for neurological conditions are murkier. No causal relationships have been clearly established between some of the metrics and measurable patient improvement down the line.

“You don’t have to have a Ph.D. in biostatistics to understand that just because ‘A’ and ‘B’ co-occur, it doesn’t mean A causes B,” said Dr. Caleb Alexander, a professor of epidemiology at Johns Hopkins Bloomberg School of Public Health.

Drugmakers are required to conduct confirmatory trials to prove accelerated approval therapies work after they reach the market or risk the FDA revoking their approval. But the trials can take years to complete and are often delayed.

Congress granted the FDA additional powers last year to pull unproven drugs from the market if manufacturers fail to provide additional evidence quickly enough. But the agency hasn’t put those powers into action.

“We are considering our new statutory authorities and working to implement them,” Cherie Duvall-Jones, an FDA spokesperson, said.

Patients without other options see drugs with expedited approvals as their only hope. Many of the diseases they treat are so rare and are fatal so quickly, it would be impossible to run a traditional clinical trial, said Paul Melmeyer, vice president of public policy and advocacy at the Muscular Dystrophy Association. “We don’t have existing options,” he said.

IT’S FRIDAY. WELCOME BACK TO PRESCRIPTION PULSE. Your co-author is drafting his fantasy basketball team on Monday.

Send news, tips and drafting tips to Lauren Gardner ([email protected] or @Gardner_LM), David Lim ([email protected] or @davidalim) and Katherine Ellen Foley ([email protected] or @katherineefoley).

TODAY ON OUR PULSE CHECK PODCAST, host Kelly Hooper talks with Katherine about the FDA's accelerated approval process for neurological disease treatments and why some experts fear it can result in giving patients false hope.

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340B hospitals serve as a vital safety net for essential health care. 340B hospitals provide 77% of all hospital care to Medicaid patients and 67% of all unpaid hospital care in the U.S. Protecting 340B is crucial for the health of patients and communities. Learn more.

Paxlovid's high price tag has drug pricing experts concerned about its future accessibility. | Joe Raedle/Getty Images

PAXLOVID PRICE DRAWS FIRE — Pfizer’s announcement that its Covid-19 antiviral Paxlovid will cost $1,390 is raising concern among drug pricing experts who point out the Institute for Clinical and Economic Review estimated the treatment would be cost-effective at between $563 to $906.

“Welcome to our screwed-up health care system,” said a former Biden administration official granted anonymity to discuss the Paxlovid list price. “What’s on full display is the difference between the public health response and a fragmented health care system. Pfizer deserves enormous credit, but come on.”

Pfizer will start selling Paxlovid in 2024 to privately insured patients after negotiating prices with insurers. The company said Thursday the $1,390 list price reflects the value of the drug’s role in reducing Covid-related hospitalizations and deaths despite being more than double the lower end of ICER’s estimated cost-effective price range.

Pfizer noted it plans to offer a co-pay assistance program for the drug and is pushing for the drug to be added to insurers’ lists of covered drugs in a way that would help keep patient out-of-pocket costs low.

But patient advocates worry the higher-than-expected list price will make it more difficult for patients to obtain a life-saving drug when government-funded access to the drug lapses.

“Here at home, doubling the U.S. price will reduce access,” said Peter Maybarduk, director of advocacy group Public Citizen’s access to medicines unit. “As with the Pfizer and Moderna vaccine price spikes, it makes it that much harder for people and payers to keep up the fight against Covid at a time when public health investment and protections are falling.”

Merck’s Covid antiviral, Lagevrio, is slated to hit the commercial market soon. A company spokesperson told Prescription Pulse it will disclose the market price in the coming weeks.

GO INSIDE THE MILKEN INSTITUTE FUTURE OF HEALTH SUMMIT: POLITICO is proud to partner with the Milken Institute to feature a special edition of our Future Pulse newsletter at the 2023 Milken Institute Future of Health Summit from November 6-8. The newsletter takes readers inside one of the most influential gatherings of global health industry leaders and innovators solving the biggest public health issues to ensure a healthier, more resilient future for all. SUBSCRIBE TODAY TO RECEIVE EXCLUSIVE COVERAGE.

NIH UPDATES GRANT REVIEW PROCESS — The NIH will roll out changes to the research grant application process early next year that aim to prevent an institution’s or investigator’s reputation from skewing NIH decisions.

“Studies have shown that consideration of reputation of the institution or investigator in the grant review process could affect assessment of scientific merit, potentially giving reputation greater weight than other factors,” NIH Acting Director Lawrence Tabak said in a statement.

Under the new system, expertise and resources will be evaluated for “sufficiency only,” leaving the importance of the research and rigor and feasibility to be scored.

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PFIZER GETS EU CLEARANCE FOR SEAGEN DEAL — Pfizer’s $43 billion deal to buy cancer drugmaker Seagen won EU antitrust approval Thursday after the European Commission found that the takeover would “not lead to loss of innovation,” POLITICO’s Giovanna Faggionato reports.

WHO UPDATES ESSENTIAL DIAGNOSTICS LIST — Three tests for hepatitis E virus and personal-use glucose-monitoring devices were added to the World Health Organization’s 2023 update to its essential diagnostics list.

MID-STAGE TRIAL OF MARBURG VACCINE KICKS OFF — The Sabin Vaccine Institute announced Thursday it had begun dosing volunteers in its Phase II clinical trial of a vaccine candidate for Marburg virus disease. There are no approved vaccines or antiviral treatments for the filovirus, which has a fatality rate of up to 88 percent, according to the nonprofit organization.

A NEW POLITICO PODCAST: POLITICO Tech is an authoritative insider briefing on the politics and policy of technology. From crypto and the metaverse to cybersecurity and AI, we explore the who, what and how of policy shaping future industries. We’re kicking off with a series exploring darknet marketplaces, the virtual platforms that enable actors from all corners of the online world to traffic illicit goods. As malware and cybercrime attacks become increasingly frequent, regulators and law enforcement agencies work different angles to shut these platforms down, but new, often more unassailable marketplaces pop up. SUBSCRIBE AND START LISTENING TODAY.

CVS said it will voluntarily remove oral cold medicines from its shelves that only have phenylephrine as an active ingredient following an FDA advisory committee meeting last month determining that phenylephrine is ineffective, The Wall Street Journal’s Jared Hopkins reports. The FDA has not yet asked retailers or drugmakers to remove such products from shelves.

Merck filed its reply brief to the government’s argument against the pharmaceutical company’s lawsuit fighting the implementation of Medicare drug price negotiations.

The FDA published guidance on its standards recognition program for regenerative medicines.

The FDA published guidance on its enforcement policy for noninvasive remote monitoring devices, outlining Covid-era policies that will continue.

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