Happy Thursday, where this morning we’re reading about streaming TV’s existential crisis. Tips on show recs or otherwise to firstname.lastname@example.org.
FDA advisors have given thumbs-up to a second controversial drug
The Food and Drug Administration got a critical greenlight to approve a hotly debated experimental treatment for the debilitating disease ALS.
In a stunning reversal yesterday, the agency’s independent advisers recommended approval of the drug despite uncertainties about its effectiveness. Less than six months ago, the panel took a different tact, narrowly voting against FDA signing off on the medication, our colleague Laurie McGinley reports.
But the final call is ultimately up to agency staff, who are expected to make a decision by Sept. 29. It’s the second time in roughly 15 months where the FDA has been tasked with deciding whether to approve a controversial drug for a devastating illness.
Holly Fernandez Lynch, of the University of Pennsylvania:
Prior vote was 6-4 in the opposite direction. Big change. https://t.co/RIGRgjXLG3— Holly Fernandez Lynch (she/her) (@HollyLynchez) September 7, 2022
ALS — sometimes referred to as Lou Gehrig’s disease — destroys nerve cells in the brain and spinal cord. Roughly 30,000 Americans have the disease, which can kill people within three to five years after symptoms first appear. As of now, there are two FDA-approved drugs on the market, but they have limited effectiveness.
Enter Amylyx. The Cambridge-based company is seeking approval for a treatment called AMX0035. As Laurie explains, the drug has amassed support from an array of patients, who deluged the FDA with thousands of emails and personal testimonies in a plea for approval.
The FDA previously expressed skepticism about the drug’s effectiveness. And so did its panel of expert advisers, who in late March concluded evidence from a single clinical trial with just 137 patients and some additional data wasn’t proof enough that the drug worked.
But the agency convened a rare second meeting yesterday. After hours of debate, the panel ultimately voted 7-2 to recommend the FDA approve the drug. Here’s what advisers said changed their minds:
- Compelling patient testimony
- Additional analyses submitted by the drugmaker
- And an unusual move by a top federal regulator
That move was made by Billy Dunn, director of the FDA’s Office of Neuroscience. The drug manufacturer is conducting a large trial slated to wrap up late next year or in early 2024. Dunn asked company officials if they would voluntarily withdraw the treatment if it was approved now but the bigger trial later failed to show the drug worked. A top Amylyx official said they would “do what is right for patients, which includes withdrawing the product from the market,” Laurie reports.
But some experts expressed alarm at the move. It gave “false reassurance” to the advisory committee that the drugmaker would abide by a “gentleman's promise,” Reshma Ramachandran, an assistant professor of medicine at Yale School of Medicine, told The Health 202.
Stat's Adam Feuerstein:
$AMLX - Fascinating opening remarks by FDA's Billy Dunn, focused mostly on the agency's legal authority to apply "broadest regulatory flexibility" for drugs that treat fatal neurological diseases like #ALS.— Adam Feuerstein (@adamfeuerstein) September 7, 2022
The ghost of Aduhelm
The deliberations are reminiscent of the controversy over a new Alzheimer’s drug called Aduhelm.
Last summer, the FDA approved the drug amid intense pressure from patient groups. But the move sparked backlash from some health experts who expressed concern the drug wasn’t safe or effective. The decision even prompted several members of the agency’s advisory committee to resign in protest and for Medicare to decline to cover the costly medication for most patients.
But there are several key differences. For one, the experimental ALS drug is considered safe. And the diseases can affect different patient populations, said Rachel Sachs, a professor at Washington University in St. Louis School of Law.
- “There wasn't a significant safety problem observed here, in the way that there was for Aduhelm,” Sachs said. “One of the big issues [with Aduhelm] was this really significant question about the potential harm to patients, and here that just doesn't seem to have been part of the analysis.”
In the courts
Obamacare can’t require coverage for some HIV drugs, federal judge rules
A federal judge agreed with a Christian-owned company that parts of the Affordable Care Act mandating private insurance plans cover certain preventive drugs and services at no cost to patients are unconstitutional, The Post’s Mark Johnson reports.
The ruling could threaten access to certain care for more than 150 million working Americans who receive health care through their employer. The federal government is likely to appeal the decision, though a Biden administration official said the decision was under review.
U.S. District Judge Reed O’Connor in Fort Worth, Texas, ruled that the requirement that employers cover HIV pre-exposure prophylaxis, or PrEP, violates the Religious Freedom Restoration Act. Plaintiffs in the case had previously argued that the mandate made them “complicit in facilitating homosexual behavior” contrary to their faith and personal values.
Also: O’Connor found that the U.S. Preventive Services Task Force cannot decide what services are required to be fully covered under the ACA because it violates the Constitution’s Appointments Clause. Mandates that insurers cover contraceptives, vaccines and some children’s services still stand for now.
Next steps: It remains to be seen whether the ruling will apply only to the Texas-based Christian for-profit corporation Braidwood Management Inc. or have greater consequences across the country. O’Connor has yet to issue a nationwide injunction or vacate the rule, but has requested both sides file supplemental briefings by Friday.
The ruling is the latest in a series of challenges to the ACA. O’Connor himself ruled the entire law unconstitutional in late 2018 — a decision that didn’t stand after the Supreme Court upheld the law 7-2 last year, the third time the justices had considered a challenge to the Obama administration’s most significant domestic policy.
Karine Jean-Pierre, White House press secretary:
The Administration is reviewing today’s decision by the Northern District of Texas. The Affordable Care Act has been the law of the land for over 10 years.— Karine Jean-Pierre (@PressSec) September 7, 2022
Michigan judge strikes down 1931 abortion ban
Michigan’s 1931 near-total ban on abortion violates the tenets of the state’s constitution, a judge ruled yesterday after temporarily suspending the law earlier this year, the Detroit Free Press reports.
Michigan Court of Claims Judge Elizabeth L. Gleicher ruled that enforcing the law would violate the right to bodily integrity and equal protection afforded to pregnant women. The decision will almost certainly be appealed to a higher court that has already weakened Gleicher’s previous abortion rulings.
The ruling doesn’t change access to the procedure in the state. Prosecutors haven’t been able to enforce the pre-Roe ban, which criminalizes abortion unless it is necessary to save the patient’s life.
A decision in the shadow of a deadline: The state Supreme Court has until Friday to decide whether to place a constitutional amendment enshrining abortion access into law on the November ballot.
Michigan Gov. Gretchen Whitmer, a Democrat:
Today, the courts ruled once again Michigan women have the right to make medical decisions for themselves.— Governor Gretchen Whitmer (@GovWhitmer) September 7, 2022
However, this decision is likely to be challenged, and we know that there are extremists who will stop at nothing to ban abortion even in cases of rape and incest.
New York drops 28-month-old mask mandate on public transportation
Gov. Kathy Hochul (D) announced yesterday that public transit riders will no longer be required to wear face coverings in New York, effectively ending one of the country’s longest-standing coronavirus mask mandates.
The announcement puts the state in line with many public transit systems nationwide, which overwhelmingly moved to drop their own face covering requirements after a federal judge overturned the nationwide mask mandate for transportation in April.
Masks will still be required at many health-care facilities across the state, but are now also optional in for-hire vehicles, airports, homeless shelters, correctional facilities and detention centers. The new rule took effect immediately.
The Metropolitan Transportation Authority:
Beginning today, masks are encouraged but optional on @NYCTSubway, @NYCTBus, @LIRR and @MetroNorth. pic.twitter.com/BMOea8nE67— MTA (@MTA) September 7, 2022
Transgender residents sue Florida over new Medicaid rule
A group of LGBT and health advocacy organizations sued Florida yesterday seeking to block a new rule excluding the state's Medicaid program from covering costs associated with gender-affirming care, our colleague Anne Branigin reports.
The lawsuit filed in Tallahassee federal court argues that Florida’s new policy, which went into effect last month, violates the plaintiffs’ constitutional rights and federal nondiscrimination statutes because it denies them treatment on the basis of their gender identity.
The state’s Medicaid exclusion comes amid a larger effort underway to roll back access to gender-affirming care, particularly for transgender youth. Florida is among nine states that explicitly exclude residents from using Medicaid to pay for the services, which can include voice therapy, hormone-blocking medication and, in some instances, surgery.
Florida officials defended the policy. “Under our rules, only treatments that are found to be safe, effective, and that meet medical necessity criteria may be covered,” Brock Juarez, a spokesperson for the state Agency for Health Care Administration, wrote in an email to Anne. “That is precisely what the Agency has done here.”
In other health news
- Health and Human Services Secretary Xavier Becerra signed a declaration yesterday permitting the FDA to issue emergency use authorizations for in vitro monkeypox diagnostics in an effort to expand the availability of tests.
- The reproductive health-care start-up Choix announced that it will start offering advanced provision abortion pills to people who aren’t pregnant so that they can stockpile them for potential future use.
- On the move: Families USA, a liberal consumer health lobby, announced it promoted Cheryl Fish-Parcham to serve as its new director of private coverage. Arielle Kane will also join the organization as its new director of Medicaid initiatives. She most recently served as director of health-care policy at the Progressive Policy Institute.
Postpartum women never lost Medicaid coverage during the pandemic. But the state told them they did. (By Isabelle Taft | Mississippi Today )
Organ Transplants Are Up, but the Agency in Charge Is Under Fire (By Blake Farmer | Nashville Public Radio and Kaiser Health News )
A suicidal son, an iconic bridge and the struggle to keep people from jumping (By William Wan | The Washington Post)
Thanks for reading! See y'all tomorrow.