When Maisie Green was born in 2017 she was weak and floppy. Doctors eventually diagnosed spinal muscular atrophy (SMA), a genetic disease that destroys motor neurons and often proves fatal in early childhood. In 2019 the FDA cleared Zolgensma, a one‑time gene replacement therapy that supplies a functioning copy of the missing gene and can halt disease progression. The list price for a single infusion: about $2 million.
Maisie’s mother, Ciji Green, thought the drug could save her daughter — but the managed Medicaid plan covering Maisie initially refused to pay. Ciji launched fundraising appeals, met with the insurer and pushed the case into the public eye. The plan reversed course and Maisie received the treatment. The infusion appears to have stopped the disease’s progression: she cannot walk because of damage that occurred before treatment, but she is doing well in school and has markedly better prospects than before the therapy.
Maisie’s experience is a window into a growing problem. A new wave of gene and cell therapies can be curative — often after just one dose — but launch prices run into the millions. Payers, employers and state budgets are still wrestling with who should cover these therapies and how to pay for them without collapsing other parts of the health system.
Why prices are so high
Manufacturers say the costs reflect years of basic research, lengthy development programs, the need to build specialized manufacturing capacity and the high risk of failure. Executives have pointed to multibillion‑dollar development bills for drugs like Elevidys, a gene therapy for Duchenne muscular dystrophy priced at roughly $3.2 million. Companies argue that prices support current programs and fund future innovation, and that costs could fall as manufacturing scales, competition grows and regulators streamline approvals.
Economists and policy analysts, however, warn of a “coming tsunami.” More than 300 high‑cost gene therapies are in clinical development, many aimed at diseases far more common than the rare pediatric conditions that dominated early approvals. Because about two‑thirds of insured Americans are covered through employer‑sponsored or self‑insured plans, those entities could face steep financial exposure — potentially forcing premium hikes or other cuts if many such treatments are approved and used.
Real cases, hard choices
Hospitals and employers are already making painful decisions. In 2023 Mosaic Life Care, a nonprofit health system in Missouri, stopped covering gene therapies after leaders estimated the cost would raise employee premiums by roughly $125 per month. Months later, twins born to a staff member were diagnosed with SMA and needed gene therapy that would cost an estimated $4.2 million for both children. Mosaic declined coverage; after public outcry and appeals to philanthropists and state officials, Missouri’s Medicaid ultimately paid for the treatments. The episode underscores the dilemma facing self‑insured employers: absorb massive costs and risk financial instability, or deny coverage for potentially lifesaving therapies.
What remains uncertain
Long‑term safety and effectiveness are still being defined for many recently approved gene therapies. Some treatments have produced transformative benefit for patients; others carry serious risks. For example, there have been reports of severe liver injury after certain therapies, and regulators continue to gather real‑world data to understand outcomes and rare adverse events.
Policy responses and payment ideas
Experts have proposed several approaches to prevent these therapies from destabilizing coverage: government negotiation of prices, new public funding streams, reinsurance or risk‑pooling programs to spread cost, outcome‑based contracts tying payment to real patient benefit, and redesigns of employer coverage. Many analysts say government involvement and negotiated prices will likely be necessary to absorb costs at scale.
For now, families improvise. Ciji Green raised money, lobbied, and won coverage for Maisie; other parents may not have the same options. As more one‑time, high‑cost therapies reach the clinic and market, the country faces a choice: create financing systems that fairly and sustainably expand access, or leave life‑and‑death treatment decisions to the budgets of employers, insurers and states.