When Maisie Green was born in 2017 she was fragile and floppy. Doctors eventually diagnosed spinal muscular atrophy (SMA), a genetic disorder that destroys motor neurons and often kills children by age two. In 2019 the FDA approved Zolgensma, a one‑time gene replacement therapy that can stop the disease by supplying a working copy of the missing gene. The drug’s list price: $2 million for a single dose.
Maisie’s mother, Ciji Green, was elated — and then devastated. Maisie was covered by Medicaid, but the insurer managing her case refused to pay for Zolgensma. Ciji launched a campaign: GoFundMe appeals, meetings with the insurer and public pressure. The board reconsidered. Maisie received the treatment. The result: progression of the disease appears to have stopped. She can’t walk — damage sustained before the drug is irreversible — but she’s thriving in school and has far better prospects than she did before the infusion.
Maisie’s story is a snapshot of a dawning problem in American health care. A new generation of genetic and cell therapies can be curative — often with a single administration — but their prices can reach millions of dollars. Payers, employers and state budgets are still struggling to decide who pays and how.
How we got here
Drug companies and developers point to the extraordinary science and expense behind these therapies. Doug Ingram, CEO of Sarepta Therapeutics, described years of basic research, the need to build manufacturing capacity almost from scratch and the billions required to develop treatments such as Elevidys, a gene therapy for Duchenne muscular dystrophy that Sarepta priced at about $3.2 million per dose. Ingram said it took roughly $3 billion to create Elevidys and that development timelines, regulatory demands and the high cost of failure contribute to high launch prices. He argued prices may fall with manufacturing scale, competition and regulatory streamlining.
Economists and policy experts warn of a “coming tsunami.” Jonathan Gruber, an MIT economist who helped design the Affordable Care Act, says more than 300 high‑cost gene therapies are in clinical trials. Many target diseases that are much more common than rare pediatric disorders. He and others worry that employer‑sponsored plans and self‑insured companies — which cover roughly two‑thirds of insured Americans — will be unable to absorb the cost of increasingly high‑priced single‑dose treatments without major premium hikes or financial strain.
Real cases and hard choices
Hospitals and health systems are already making stark decisions. Mike Poore, CEO of Mosaic Life Care, a nonprofit hospital system in Missouri, told reporters his organization stopped covering gene therapies in 2023 because the projected cost would have driven up employee premiums by roughly $125 a month. Months later, a staff member’s newborn twins were diagnosed with SMA and needed gene therapy estimated at $4.2 million for both children. Mosaic denied coverage. The family went public; the CEO faced anger and threats. After appeals to philanthropists and state leaders, Missouri’s Medicaid paid to treat the twins.
Poore’s experience underlines the policy dilemma: employers and their self‑insured plans face a choice between absorbing enormous costs that could threaten their financial viability or denying coverage for potentially lifesaving care.
How companies justify prices
Drugmakers defend high launch prices by pointing to development risk and small patient populations for many rare disease therapies. Ingram said manufacturers must raise billions to fund long development programs, in some cases with low probabilities of success. He also said manufacturing gene therapies required building new capacity and expertise, pushing up early costs. Executives claim prices reflect that investment and enable future innovation.
Critics say manufacturers charge what the market will bear, and that single‑dose price tags are politically and ethically fraught when they determine who lives and who dies.
What we still don’t know
Long‑term effectiveness and safety remain uncertain for many recently approved gene therapies. Sarepta’s Elevidys has treated about 1,100 patients but carries risks: there have been reports of liver failure leading to deaths. Regulators and clinicians continue to collect data on real‑world outcomes. For some patients the treatments are transformative; for others the benefits are still being evaluated.
Coverage, payment models and policy options
Experts suggest multiple approaches to prevent these therapies from destabilizing health coverage: government negotiation of prices, new public funding mechanisms, reinsurance or other risk‑spreading programs, outcome‑based payment contracts between payers and manufacturers, and changes in employer‑sponsored insurance design. Jonathan Gruber said government support and negotiated prices will likely be necessary to absorb costs at scale.
For now, families improvise. Ciji Green raised money, lobbied for coverage and ultimately secured Zolgensma for Maisie. Other parents may not find the same path. As more high‑cost one‑time therapies reach the clinic and market, the nation faces a choice: create systems to finance these advances equitably, or let coverage decisions — by employers, payers and states — determine access to potentially life‑saving treatments.