Gene therapies that cure rare diseases cost $2-4 million per dose, and no one has figured out how to pay for them

Lenmeldy, a gene therapy for metachromatic leukodystrophy, costs $4.25 million for a single dose. Roctavian for hemophilia A costs over $2 million. Zolgensma for spinal muscular atrophy costs $2.1 million. These are one-time treatments that can functionally cure diseases that would otherwise require millions in lifetime care -- or kill the patient. The clinical value is real. The problem is that the entire insurance payment infrastructure was built for chronic treatments billed monthly, not one-time curative doses billed all at once. A single patient receiving Lenmeldy can blow through a small employer's entire annual health budget. The downstream effects ripple through the system. Insurers respond by increasing premiums, raising deductibles, excluding gene therapies from formularies, or steering patients toward manufacturer patient assistance programs -- effectively making the pharma company the insurer of last resort. Medicaid programs, which cover many rare disease patients, face even steeper barriers: state budgets aren't designed to absorb $4 million line items. The result is that gene therapies exist, are FDA-approved, and demonstrably work, but patients can't access them because the payment plumbing can't handle the transaction. Only 5% of FDA-approved gene therapies have been reimbursed across global health systems. The CMS Cell and Gene Therapy Access Model, launched with 33 states plus DC and Puerto Rico, represents the first federal attempt to solve this by negotiating pricing deals with manufacturers on behalf of state Medicaid programs, starting with sickle cell disease. But it covers only Medicaid, only specific conditions, and only treatments approved before May 2024. Private insurance remains the wild west. Proposed solutions -- installment payments, outcomes-based agreements where the manufacturer refunds money if the therapy doesn't work, annuity models -- all exist in theory but require infrastructure (patient tracking over decades, inter-payer data sharing, legal frameworks for refunds) that doesn't exist yet. The structural problem is that curative medicine broke the actuarial model that funds American healthcare. Until someone builds a new financial architecture, million-dollar cures will continue to sit on pharmacy shelves while the patients they could save go without.