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What do insulin and opioids have in common?

November 28, 2023
Reporter, Morning Rounds Writer
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exclusive

FDA gives feedback on proposed trial seeking approval for gender-affirming hormones  

In an unexpected turn of events, the FDA has responded to a non-profit's research proposal for a trial of gender-affirming hormones with advice to dispense with a placebo arm and expand participation beyond adults to include adolescents as young as 13. The nonprofit Research Institute for Gender Therapeutics was "positively surprised" by the agency's written response to the group, which ultimately plans to seek approval for estrogen to treat trans and gender-diverse patients. Gender-affirming hormones are now prescribed to patients off-label, without an explicit approval for trans health care.

"[The FDA's] feedback validates that this is a treatable condition and validates the existence of that population, and really gives us a pretty clear path to a federal approval for gender affirming care," said Brad Sippy, the group's founding director and president. The FDA did not explain the reasoning for its advice, but experts have warned that randomized controlled trials for hormone treatments may be both unethical and impractical, since the treatment can prevent serious harm for trans patients and its physical effects are hard to blind. STAT's Theresa Gaffney has more in this exclusive story.


drug pricing

A wave of lawsuits hitting insulin makers and sellers

So many states, counties, and cities are suing companies that make and sell insulin that it's reminiscent of legal actions taken during the opioid crisis, STAT's Ed Silverman and John Wilkerson suggest. Here's what prompts the comparison: In recent weeks, state officials in Utah and municipalities in New York, Virginia, Maryland, and Ohio have filed lawsuits alleging that the companies — drug makers Eli Lilly, Sanofi, and Novo Nordisk, and pharmacy benefit managers Express Scripts, OptumRx, and CVS Caremark — conspired to profit from a complicated scheme involving lockstep pricing, favorable insurance coverage, and secret rebate fees.

The litigation alleges the manufacturers tried to coordinate price hikes, even as the cost to produce insulin plummeted. The sheer volume of lawsuits recalls how the companies that made or distributed prescription opioids reached billions of dollars in settlements amid allegations the improperly promoted painkillers led to tens of thousands of deaths. Read more, including responses from Novo, Sanofi, and CVS.


research

California stem cell agency board members hint at issues behind CEO's surprise departure

Earlier this month, the California Institute for Regenerative Medicine unexpectedly announced that its president and CEO, Maria Millan, would be resigning. But the agency didn't provide a clear reason, telling STAT's Jonathan Wosen in emails that the decision was Millan's choice and that "she feels she has completed her work at CIRM and that we are better positioned to move into the next phase of our evolution."

Yesterday's meeting of CIRM's board hints that the agency's answer wasn't the full story. One board member remarked that he had been surprised by the "crisis situation that seems to exist at a staff level," adding that morale had dipped during Millan's tenure. Another alluded to "HR" issues within the agency, bankrolled by the people of California, who in 2020 narrowly voted to keep CIRM afloat with $5.5 billion in fresh funding.



opinion

Alzheimer's drugs' dangerous side effect deserves more attention, neurologist says

LEQEMBI Illustration: Alex Hogan/STAT; Photo: Eisai via AP 

Since the FDA approved lecanemab and Medicare decided to cover the drug, Johns Hopkins neurologist Madhav Thambisetty has met with many Alzheimer's patients and their families eager to know if this new treatment can help slow the advance of this disease. He meets their hopes with the drug's risk-to-benefit ratio, which includes both a modest slowing of the memory-robbing disease and the worrisome brain swelling and bleeding called ARIA — side effects also found in two similar drugs, aducanumab and donanemab.

Thambisetty is troubled by marketing campaigns that paint ARIA as mostly mild or even asymptomatic, often resolving spontaneously, even though 1% to 2% of patients in clinical trials didn't recover and got worse. Each of the three drugs has been linked to patient deaths, most of which came to light via investigative journalists, including at STAT. "Before deciding whether to offer these treatments to eligible patients, physicians should insist on full disclosure of all observed adverse events and their relationship, if any, to worsening memory impairment and functional abilities," Thambisetty writes in a STAT First Opinion.


inequity

STEM PhDs with disabilities are paid less in academia

Employment disparities for women and underrepresented racial minorities in science, technology, engineering, and medicine are all too familiar. Less well known may be the fact that scientists and engineers with disabilities are more likely to be unemployed than other people in the U.S. labor force. Now a new paper in Nature Human Behaviour finds that when people who have been disabled before age 25 complete their doctoral degrees and get jobs in STEM, they earn $10,580 less per year than their peers without a disability, after being matched for socioeconomic background, job, and degree-related characteristics.

In academia, they fare even worse, earning $14,360 less per year than their counterparts without a disability. The analysis of 1.15 million U.S. research doctorate recipients awarded degrees between 1973 and 2017 also found advancement to tenured positions or such higher faculty levels as deans and presidents was stalled if there was an early disability — but not if disability occurred later in life.

"People with disabilities are a national asset whose potential should not be underutilized," the authors write. "Lower representation and salaries for people with disabilities are unjust."


insurance

People with Medicaid report more coverage denials

more-than-half-of-medicaid-enrollees-report-having-experienced-a-problem-with-their-insurance-in-the-past-year

Most of the Medicaid news lately has documented how many people have lost coverage as enrollment has unwound along with the pandemic-era state of emergency. A new KFF survey asked people still on Medicaid what they thought about their coverage. By far, most respondents (83%) on Medicaid assessed their health insurance as "excellent" or "good." Even so, more than half (58%) had a problem in the past year, such as difficulty with prior authorization and trouble finding health care providers.

Other differences:

  • Medicaid enrollees are more likely to rate their physical or mental health status as fair or poor compared to adults with other coverage.
  • Medicaid enrollees are less likely than those with other insurance to report cost-related problems or difficulty understanding what they owe out of pocket.

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What we're reading

  • This boy was born without an immune system. Gene therapy rebuilt it, Washington Post

  • Need therapy? In West Africa, hairdressers can help, New York Times

  • Sales of antibiotics for food-producing animals dropped by more than half in Europe over the past decade, STAT
  • Dr. Chelsea Polis: 'The scientific world recognises when you stick your neck out and do the right thing,' The Guardian

  • Most clinical trials run in Canada failed to take basic steps toward transparency, analysis finds, STAT

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