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The Health 202

A newsletter briefing on the health-care policy debate in Washington.

The Makena controversy puts FDA's accelerated drug approval program under scrutiny

The Health 202

A newsletter briefing on the health-care policy debate in Washington.

Happy Thursday morning, where there were “sneaky snow flurries” in the D.C., Maryland and Virginia area this morning. Send news tips and flurry sightings to

Today’s edition: A false claim that the CDC would require coronavirus vaccines for kids went viral. A potential preview of Republican investigations if they win the House. But first …

The debate over the effectiveness of a preterm labor drug highlights frustrations with FDA's fast-track program

The Food and Drug Administration advisers’ recommendation to withdraw a preterm pregnancy drug from the market put a spotlight on the program used to approve it. 

The 14-1 vote came at the end of an emotional three-day hearing over whether the FDA should rescind approval of the drug Makena, which was initially approved through a fast-track process but whose effectiveness hasn't been confirmed in follow-up studies, Ariana Eunjung Cha and I reported yesterday.

The debate over the drug’s effectiveness over a decade after its approval raises questions about the FDA’s accelerated approval program and underscores just how long it can take the agency to pull a drug from the market even when officials believe it doesn’t work.

In 2011, Makena was approved under the FDA’s fast-tracked process meant to expedite the availability of drugs that treat serious conditions in need of more treatments. But such approval requires drug companies to conduct follow-up studies to confirm the medicine’s benefits. 

Lawmakers on Capitol Hill, experts and even top FDA officials have said they want reforms to the program, which critics say is cumbersome and doesn’t incentivize drugmakers to perform additional clinical trials on time. 

A case study

Makena’s original trial was viewed as promising, but not definitive. However, a larger confirmatory trial out in 2019 was broadly disappointing and didn’t show the drug was effective, Ariana noted earlier this week.

The FDA’s Center for Drug Evaluation and Research proposed withdrawing it from the market in October 2020 — a move that followed an expert advisory panel’s 9-7 vote a year earlier to recommend yanking the treatment. But regulatory requirements, as well as the pandemic, slowed the process.

But drugmaker Covis Pharma, in pushing back against FDA, has argued there is evidence to suggest Makena may work in a narrower population that includes Black women at high risk of giving birth too soon. The company suggested the larger study may have missed benefits in high-risk patients in the United States because participants in the trial were mostly Eastern European, only 7 percent Black and lower risk.

That didn’t sway the FDA. Throughout this week’s meeting, top officials were steadfast in their opinion that the drug should be removed. The agency's advisers agreed, saying it was imperative to withdraw drugs without a proven benefit from the market or risk setting a bad precedent.

Now, it’s up to FDA Commissioner Robert Califf and the agency’s chief scientist to make the final decision on what to do with the drug.

Pink Sheet's Sarah Karlin-Smith:

Under scrutiny

The FDA’s process to withdraw drugs under the expedited pathway can be “lengthy and contentious,” the watchdog for the Department of Health and Human Services wrote in a recent report.  

  • “These challenges can result in drugs staying on the market — and being administered to patients — for years without the predicted clinical benefit being verified,” the report states.
  • Meanwhile, more than one-third of drugs that haven’t wrapped up their confirmation trials are past their original completion date. More than five years have gone by for four medications.

The notion of changing the accelerated approval process has intensified on Capitol Hill. Reforms were included in initial versions of legislation to reauthorize critical FDA user fees, but were ultimately left on the cutting room floor.

Meanwhile, the issue also cropped up during the lengthy confirmation battle for FDA commissioner. Senate Finance Committee Chair Ron Wyden (D-Ore.) asked Califf to affirm he would hold drug companies accountable for delivering follow-up data under the accelerated pathway program.

Ensuring drugmakers conduct additional studies would be a “high priority,” Califf wrote in a letter to Wyden last February. “Accelerated approval can facilitate the development of therapies to address unmet needs. However, it is incumbent upon FDA to ensure that the work does not end with the initial approval.”

Zach Brennan, Endpoints News:


False claim that CDC would require coronavirus vaccines for kids goes viral

A false claim that the Centers for Disease Control and Prevention was set to mandate that schoolchildren get coronavirus vaccines spiraled out of control Tuesday, making it onto the nation’s most popular cable news show, our colleagues Dan Diamond and Lena H. Sun report.

The erroneous claim spread by Fox News personalities is the latest example of how critics can twist the facts about the CDC and the coronavirus, potentially contributing to lower vaccination rates, fading trust in federal health officials and other consequences for public health.

What happened: On Tuesday morning, Fox News contributor and radiologist Nicole Saphier tweeted the false claim that CDC advisers would be voting on whether to require children get vaccinated against the coronavirus to attend school. The claim was picked up by conservative commentator Tucker Carlson and amplified to millions more on social media. (He doubled down again on his show last night.)

In reality, the panel was voting to add the shots to the federal Vaccines for Children program, which provides the vaccines at no cost for low-income families. The CDC cannot mandate that students receive vaccines — that responsibility is left up to states and local jurisdictions.

Administration officials were initially hesitant to criticize the false claims in fear of amplifying them. But yesterday, the CDC took to Twitter, pointing out that such a mandate for children isn’t within the agency’s authorities and noting that its independent vaccine advisory committee is set to weigh today whether to add approved or authorized coronavirus vaccines to its 2023 childhood and adult immunization schedules, which acts as a guide for health-care providers.

Kavita Patel, a physician and former Obama administration official, rebuffed the claims: 

On the Hill

First in The Health 202: House Republicans press Biden administration on covid test manufacturing

Republican lawmakers on the House Committee on Oversight and Reform are pressing the Department of Health and Human Services to turn over documents regarding the Biden administration’s purchase of coronavirus tests from a company headquartered in China in a letter sent yesterday. The move could serve as a preview of the kind of investigations Republicans may launch should the party take control of the House this November.

The details: The inquiry centers on the administration’s nearly $1.3 billion contract with iHealth Labs to manufacture at-home coronavirus tests. The agreement made the California-based subsidiary of China’s Andon Health part of the push to make free kits available to Americans.

Former White House press secretary Jen Psaki previously acknowledged the deal, saying at a February press briefing that the purchase was necessary to meet the nation’s needs amid a shortage of tests that outpaced U.S. manufacturing capacity. HHS didn’t immediately respond to The Health 202’s request for comment.

Midterms watch

Fetterman’s doctor says he has no work limits

The primary care physician for Pennsylvania Democratic Senate nominee John Fetterman said yesterday that he is recovering well from his stroke and has no work restrictions, our colleague Colby Itkowitz reports.

University of Pittsburgh Medical Center’s Clifford Chen wrote in a letter Saturday that Fetterman’s vital signs were normal. Chen said his communication has significantly improved since his stroke in May after speech therapy, but noted that the candidate continues to show symptoms of “an auditory processing disorder which can come across as hearing difficulty.” 

Fetterman and his aides have often mentioned this condition, but the report from Chen marks the most detailed information his campaign has provided from a doctor since early June. His GOP opponent, Mehmet Oz, has openly questioned whether his challenger’s health makes him fit for office, and has repeatedly attacked him for not releasing more detailed medical records or making his doctors available for interviews with the press. 

More from Fetterman: 

Industry Rx

Black men are less likely to donate sperm

Black men account for less than 2 percent of sperm donors at cryobanks across the nation, our colleague Amber Ferguson writes. 

By-the-numbers: There are more than 20 cryobanks in the country, four of which have more than 100 donors. Among the four, as of Oct. 11, there were only 12 Black donors out of a total of 748 — and Black women vying for sperm from Black donors say vials typically sell out within minutes.

At California Cryobank, the waiting list for an in-demand White donor is generally three months, according to Jaime Shamonki, the chief medical officer. The wait for a Black donor there can stretch as long as 18 months. 

Read more from Amber and her multipart series on sperm donors here. 

In other health news

  • The FDA authorized Novavax’s coronavirus booster shot for adult use yesterday, and the CDC recommended the shot shortly thereafter, The Post’s Laurie McGinley reports.
  • Abbott Laboratories announced yesterday that it has made leadership changes at its major baby formula manufacturing plant and in its quality organization, following a temporary shutdown of a site earlier this year that fueled a nationwide formula shortage, the Wall Street Journal reports.
  • The coronavirus contributed to a quarter of maternal deaths in the first two years of the pandemic, according to a report from the Government Accountability Office yesterday.

Health reads

Adderall shortage is so bad some patients can’t fill their prescriptions (Fenit Nirappil l The Washington Post)

Kids’ Mental Health Care Leaves Parents in Debt and in the Shadows (By Yuki Noguchi, NPR News | Kaiser Health News)

These Doctors Admit They Don’t Want Patients With Disabilities (By Gina Kolata | The New York Times)

Sugar rush

Thanks for reading! See y'all tomorrow.