December 11, 2023

Happy Monday, Vitals readers. Today's newsletter is 1,086 words or a 4-minute read.

1 big thing: What's next for CRISPR

A patient suffering from complications of sickle cell disease gets an echocardiogram. Photo: Michael Williamson/The Washington Post via Getty Images.

The approval of the first CRISPR-based gene editing treatment to address the excruciating symptoms of sickle cell disease was a landmark moment for the treatment of inherited disorders.

  • It also raises urgent questions about who may benefit from new cutting-edge treatments costing millions of dollars and what comes next for a technology discovered just over a decade ago, Axios' Adriel Bettelheim writes.

The big picture: Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is a one-time treatment in which stem cells are harvested from a patient's bone marrow, then edited to produce fetal hemoglobin, which can offset the effects of patients' defective hemoglobin.

  • It was approved by the FDA on Friday alongside Lyfgenia, another gene therapy treating sickle cell disease, which affects an estimated 100,000 Americans, mostly of African descent, and 20 million people globally.

Yes, but: The treatments aren't for everyone.

  • Patients first have to undergo extensive chemotherapy to rid their bodies of the defective cells and make way for reengineered ones — a process that could take months and may not be appropriate for older or frail patients.
  • Few hospitals can offer Casgevy. Just nine medical centers are now authorized by Vertex to provide the treatment, though the drugmaker will eventually authorize about 50, per New York Times.
  • There are other hurdles. For instance, about half of those living with sickle cell are lower-income individuals on Medicaid. The Biden administration launched a recent effort to help ensure this group can access new treatments.

What's next: Like sickle cell, more than 6,000 rare inherited diseases are caused by a single genetic mutation — CRISPR's molecular scissors could hold potential for addressing them, USA Today noted.

  • Researchers using a newer form of CRISPR last month reported that editing a gene inside the liver can significantly reduce levels of "bad cholesterol" in people who have a genetic condition that causes high cholesterol.
  • The study was small but highly anticipated, and researchers believe further study may show it's a powerful tool for reducing heart attacks and strokes, even in people who don't have the genetic condition, per NPR.
  • Researchers want to continue to perfect the technology and edit or insert bigger pieces of DNA to make it possible to treat a condition like cystic fibrosis that's caused by many mutations in a certain gene.

Go deeper

2. Cigna-Humana deal off the table

Photo: Joe Buglewicz/Bloomberg via Getty Images

Cigna is turning its focus toward smaller "bolt-on" acquisitions after a potential merger with health insurance giant Humana fell apart.

Driving the news: Cigna is walking away from its ambitions to acquire Humana with a cash-and-stock transaction after the two companies couldn't agree on price and other financial terms, the Wall Street Journal reported on Sunday.

Catch up quick: Such a deal would have created a health insurance giant valued at roughly $140 billion and further consolidated the pharmacy benefit manager industry. It also likely would've come under intense antitrust scrutiny.

  • The idea was unpopular with investors, with Cigna's share price dropping 10% in the two weeks since news of the potential acquisition broke.

What we're watching: Cigna has said it's planning to "use the majority of its discretionary cash flow" for an additional $10 billion stock buyback.

  • "We believe Cigna's shares are significantly undervalued and repurchases represent a value-enhancing deployment of capital," Cigna CEO David Cordani said in a statement.
  • He also said the company will be looking to make some "value-enhancing divestitures."
  • Cigna, which is a big player in the commercial insurance market, is still exploring a sale of its smaller Medicare Advantage business, the WSJ noted. But that could leave it out of a growing part of the insurance market.

3. Support for curbing facility fees

Illustration: Gabriella Turrisi/Axios

As more states weigh limits on "facility fees" hospitals can charge for outpatient care, new polling from a patient advocacy group shows widespread opposition to these tack-on charges.

By the numbers: More than three-quarters of voters support banning extra fees charged by hospitals for services at their off-campus clinics and doctor's offices, according to polling from United States of Care and Morning Consult shared first with Axios.

  • More than 80% of voters also support requiring medical providers to disclose facility fees, which can reportedly range from $15 to more than $1,000, before a patient comes in for care.
  • 13 states including Indiana, Florida, Minnesota and New York have passed legislation limiting facility fees or making them more transparent. Some have specifically banned the charges for telehealth services or COVID-19 treatments.
  • More states are expected to pursue legislation limiting facility fees next year, said USofCare senior director Lisa Hunter.

The other side: The American Hospital Association has said facility fees are needed to help pay for the overhead costs of running a hospital and to offset underpayments by insurers.

  • Meanwhile, critics say the fees drive up costs without improving patient care.

Of note: A health care transparency bill expected to move through the House of Representatives this week adds modest limits on how Medicare pays hospital outpatient facilities more for some services, but it doesn't touch facility fees.

  • A bipartisan Senate package introduced earlier this fall would ban certain facility fees, but its prospects are uncertain.

4. Calif. governor eyes changes to new wage law

Photo: Keith Birmingham/MediaNews Group/Pasadena Star-News via Getty Images

Big changes may be coming for California's new law mandating a minimum wage for health care workers, the first of its kind in the United States.

Driving the news: As the state stares down a projected $68 billion budget deficit, Gov. Gavin Newsom is seeking "major reforms" to the pricey law that raises the minimum wage to $25 an hour, the L.A. Times reports.

  • The measure approved this fall with the strong backing of labor unions is reportedly the most expensive from the last legislative session, estimated to cost the state $4 billion in the first full fiscal year it's implemented.
  • The law currently doesn't include a mechanism to pause wage hikes during economic downturns.

What's next: It's unclear whether Newsom is looking to narrow eligibility for wage hikes or push back its implementation, the Times notes.

  • There could be more clarity in the coming weeks, with Newsom expected to unveil his new budget plan on Jan. 10.

5. While you were weekending

Illustration: Annelise Capossela/Axios

Chronic fatigue syndrome affects an estimated 3.3 million U.S. adults, more than previously thought. (Axios)

Long wait times to obtain an abortion have grown more common, causing delays that can lead to abortions that are more complex, costly and sometimes riskier. (Associated Press)

Former and current patients of the Fred Hutchinson Cancer Center have received threatening emails from hackers after a cyberattack on the health system. (Seattle Times)

The immune system may play a much larger role in postpartum depression than previously known, emerging research shows. (Washington Post)

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