This week in health care policy: September 21–25, 2026

Innovation watch

FDA approves the first treatment for Sanfilippo syndrome. On September 17, the FDA approved Ultragenyx’s Fayuvi, a one-time gene therapy for Sanfilippo syndrome type A, an inherited disease that progressively destroys cognitive function in young children. Until this week, there was no approved treatment at all.

BioNTech’s lung cancer candidate shows promising results. On September 14, BioNTech and partner OncoC4 reported that gotistobart extended median survival to 18.5 months, versus 10 months on standard chemotherapy, in squamous non-small cell lung cancer patients whose disease had already progressed after immunotherapy and chemo (Reuters). For those patients, median survival on existing treatment is under a year.

Novo Nordisk plans five-plus blockbuster launches by 2030. At its September 21 investor day, Novo laid out a pipeline aimed at more than five “multi-blockbuster” launches this decade.

Cancer death rate falls 35 percent in a decade. The American Association for Cancer Research’s annual Cancer Progress Report, released September 16, finds the US cancer death rate fell 35 percent from 2015 to 2024, averting more than 4.8 million deaths.

FDA panel backs premarket application for first multi-cancer blood test. On Wednesday, the Molecular and Clinical Genetics Devices Panel voted to approve GRAIL’s premarket approval application for the Galleri test. The test detects cancer signals from DNA and can identify several cancers from a single blood sample.

Drug pricing

Every state applies for “GENEROUS,” Medicaid’s most-favored-nation experiment. CMS announced that all 50 states, plus DC and Puerto Rico, have applied to the GENEROUS model.

No Surprises Act

An appeals court sides with Cigna over unpaid arbitration award. The US Court of Appeals for the Second Circuit affirmed a lower court decision to dismiss a plastic surgery group’s suit to collect $3 million in No Surprises Act arbitration awards from Cigna. This outcome reduces the number of enforcement mechanisms available to providers.

The bigger picture. A Becker’s roundup shows more developments regarding the state of the No Surprises Act.

Coverage

CMS cancels 760,000 improper ACA enrollments. On September 22, the administration announced it canceled roughly 315,000 marketplace policies covering about 760,000 people, citing unauthorized and fraudulent enrollments, and will run extra verification on another 419,000. Officials estimate the actions save $2.2 billion.

Uninsured rate holds steady. The Census Bureau reports that 26.7 million Americans, 7.9 percent, went without coverage for all of 2025, statistically unchanged from 2024. Beneath the flat topline, Medicaid coverage fell by 1.4 million people while Medicare rose by 2.2 million.

Administration and legislation

The White House backs off its NIH grant order. A planned executive order giving the White House more say over NIH grant decisions is being pulled back after Senate Appropriations Chair Susan Collins pressed OMB to drop it.

FDA nominee faces the Senate. On September 24, Heidi Overton, the nominee for FDA Commissioner, testified before the Senate HELP Committee, where senators pressed her on the administration’s childhood vaccine schedule overhaul and on mifepristone.

“Apples to Apples” advances. On September 16, Ways and Means ordered H.R. 4093 reported, 25–15, with a substitute amendment. Starting in 2030, the bill would require CMS to publish Medicare spending by county and metro area, broken out by enrollee type. It would also direct MedPAC to compare Medicare Advantage and fee-for-service spending every three years, counting only fee-for-service beneficiaries who have both Part A and Part B and could actually enroll in MA, adjusting for demographics and risk scores. An amendment also directs CMS to run the analysis both with and without favorable selection, which critics were concerned about.