December 15, 2023

Happiest of Fridays, Vitals crew. Today's newsletter is 1,016 words or a 4-minute read.

1 big thing: Trans care advocates look to FDA

Illustration: Aïda Amer/Axios

An effort to get FDA approval of hormone therapies for gender-affirming treatment could help preserve patients' access to the therapies as states restrict them, Maya writes.

The big picture: Transgender patients can receive hormone therapies "off label" — a common practice in which doctors prescribe treatments for a use the agency hasn't approved.

  • A small nonprofit formed this year with the goal of obtaining FDA approval of the drugs for gender-affirming care believes the agency's formal OK could strengthen legal protections for the treatments and boost insurance coverage.
  • In recent years, Republican lawmakers in at least 22 states have passed laws banning or limiting gender-affirming care for minors, some of which include restrictions on hormone therapy.

Driving the news: The Research Institute for Gender Therapeutics (RIGT) has proposed a late-stage clinical trial of estradiol, a common form of estrogen, as a treatment for gender incongruence. The FDA last month provided initial feedback on the trial design.

  • "We're excited that now we have really good visibility on how to go about pursuing" full FDA approval, said RIGT co-founder Brad Sippy.
  • An FDA spokesperson said the agency is limited in what it can say about a drug sponsor's development program, while noting that FDA's advice "should not be interpreted as FDA necessarily proposing or recommending a different approach."

The nonprofit, which is aiming to start the trial in 2025, still needs to secure some approvals to run it and also raise money for the research. It hopes to launch a similar effort for testosterone treatment.

FDA approval of hormone therapies for gender-affirming care wouldn't change the way doctors prescribe them or patients use them, but it would bring additional legitimacy to the treatment and could make insurance companies more willing to cover the drugs, said Madeline Deutsch, director of the University of California, San Francisco's gender-affirming care treatment program.

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2. Concern about cash flow to pharmacies

Illustration: Sarah Grillo/Axios

CMS is urging health plans and other payers not to put pharmacies in a cash squeeze when a new policy kicks in that may reduce how much they're paid upfront for dispensing drugs.

  • CMS on Thursday wrote to pharmacy benefit managers and insurers suggesting they make special payment arrangements with pharmacies before the policy takes effect on Jan. 1, Maya writes.

Catch up quick: The change is aimed at lowering patients' drug costs by keeping Medicare prescription drug plans and PBMs from retroactively recouping performance-based adjustment fees from pharmacies.

  • Payers will still be able to adjust pharmacy reimbursement for quality performance at the point of sale.
  • Medicare originally planned to start the policy in 2023 but delayed it a year after pharmacies raised concerns that the change would slash their cash on hand at its outset.

Medicare officials have reason to be concerned. Hundreds of pharmacies have closed this year, and research shows that nearly a quarter of U.S. neighborhoods are in "pharmacy deserts."

The other side: PBMs oppose the policy change and have "raised countless times our concerns about the likely consequences of the new rule," said Greg Lopes, spokesperson for the Pharmaceutical Care Management Association.

More here

3. What the CDC found on accidental gun deaths

Data: U.S. National Violent Death Reporting System; Chart: Axios Visuals

Children and teens involved in unintentional fatal shootings most commonly found the gun inside or atop a nightstand, under a mattress or pillow, or on top of a bed, according to a new federal study.

Why it matters: CDC researchers said the data, which covers nearly 20 years of deadly firearm accidents among America's youth, demonstrates why putting a gun out of sight or out of reach is not "safe storage," Tina writes.

  • It underscores the need for policymakers, health experts and parents to promote safe gun storage, they said.

The big picture: Researchers identified more than 1,250 unintentional gun deaths among kids between 2003-2021.

  • The vast majority involved guns that were unlocked (76%), and most of those unlocked firearms were also loaded (91%).
  • Two-thirds (67%) of unintentional gun injury deaths among kids occurred when the shooter was playing with the gun or showing it to others.
  • In 30% of deaths, guns were found around nightstands and other sleeping areas.
  • Guns were also most commonly found on top of a shelf or inside a closet (18.6%) or inside a vehicle (12.5%).

Read here

4. 1 big number: The Oprah effect

Oprah Winfrey at the Smithsonian's National Portrait Gallery in Washington on December 13. Photo: Tom Brenner for The Washington Post via Getty Images

After Oprah revealed this week that she's taking an anti-obesity medication,
online appointment booking company Zocdoc on Wednesday saw a 30% spike in bookings among patients seeking semaglutidethe active ingredient in Wegovy and Ozempic — compared with the previous four Wednesdays.

  • Now that's market power.

5. Health impacts of a record wildfire season

The 2023 wildfire season more than doubled the previous record for fire-related air pollution in the U.S., report Axios' Will Chase, Erin Davis and Kavya Beheraj.

  • The average American was exposed to 66% more air pollution than the previous record year, driven by the worst wildfires in Canada's history, according to data from the Stanford Environmental Change and Human Outcomes Lab.

Why it matters: Exposure to fine-particle pollution (PM2.5) from wildfire smoke irritates the throat and eyes, causes breathing problems, and contributes to long-term mortality by exacerbating heart disease and respiratory illness.

  • Wildfire smoke has become a fact of life in the American West, but 2023 saw record-breaking smoke exposure on the East Coast and mid-Atlantic, affecting major population centers.
  • Future warming will cause even more frequent fire weather conditions, according to the latest reports from the Intergovernmental Panel on Climate Change.

See how wildfire smoke affected you this year

6. Catch up quick

💊 48 drugs could face Medicare rebates in 2024's first quarter because their prices rose faster than inflation. (Fierce Pharma)

🧬 Bluebird Bio said a large insurer has agreed to cover its new sickle cell gene therapy, easing some investor concerns that payers would balk at the treatment's $3.1 million price tag. (Reuters)

💪 Kansas' Democratic governor's latest pitch for Medicaid expansion includes a work requirement. (Kansas Reflector)

🦾 Nearly 30 health care companies have joined the Biden administration's voluntary safeguards on AI use. (Bloomberg)

7. Dog of the week

Luna. Photo: Krish Ramadurai

Meet Luna, who we hear is a big fan of puppuccinos and regular power naps.

  • Her human, Krish Ramadurai, also tells us: "She loves hanging with her human friends, watching movies of all genres, and playing with her favorite dinosaur toys."

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