Opinion: $2 million gene therapy cures require a financing model

OpinionOpinions+ $2 million gene therapy cures require a financing model Decades of value in a single intervention mean we need a new approach Manage alerts for this article Email this article Share this article By William V. PadulaJune 8, 2026 Padula is a senior scholar at the USC Schaeffer Center for Health Policy & Economics, an associate professor of pharmaceutical and health economics at the USC Mann School of Pharmacy and Pharmaceutical Science, and chief scientific officer of Stage Analytics. A new class of medicines is transforming health care. Gene therapies can now cure diseases like sickle cell with a single treatment, but they come with a price tag that would have been unthinkable a decade ago — often $2 million or more per patient. In a way, this cost makes sense: These are potential cures that can avoid years of hospitalizations, complications, and lost productivity. In many cases, they concentrate decades of value into a single intervention.Advertisement Yet many eligible patients are not receiving them, because America’s health care system is not built to pay for cures. The central problem is financing: The health system lacks mechanisms to absorb large upfront costs for treatments that deliver value over decades. Even as federal and state policymakers roll out new models to expand access to gene therapies, many patients still cannot receive them. The result is not just limited access, but a structural market failure: Cures exist, but the system cannot deliver them at scale. Under the Biden administration, the Centers for Medicare and Medicaid Services launched the Cell and Gene Therapy Access Model, a voluntary program designed to help state Medicaid agencies improve access to high-cost gene therapies through multistate purchasing and outcomes-based payment arrangements. The Cell and Gene Therapy Access Model reflects growing recognition of the problem. By enabling states to pool risk and negotiate arrangements that tie payment to patient outcomes for high-cost therapies, it aims to expand access within Medicaid.Advertisement But these efforts remain constrained by the same underlying challenge: the need to absorb large, upfront costs in a system designed around annual budgets. Early analyses suggest that while these models may improve access at the margins, they do not fundamentally change how these costs are financed. States remain constrained by budget cycles, and manufacturers face uncertainty in scaling treatment, leaving access behind scientific capability. Insurers and state Medicaid programs operate on those annual budgets. A $2 million upfront cost, even for a therapy that delivers long-term value, is difficult to absorb. Coverage remains limited, approvals are slow, and access is constrained, especially outside the rarest conditions. Some policymakers argue that prices will fall as these therapies scale, but this assumes a cost structure that does not apply. Gene therapies are not mass-produced pills. In many ways, they are closer to organ transplants, requiring specialized centers, highly trained teams, and limited clinical capacity. Lowering price alone does not resolve these constraints. It can shift the bottleneck elsewhere. A tenfold reduction in price, from millions to hundreds of thousands per patient, would require a corresponding expansion in patient identification, treatment capacity, and clinical delivery, none of which scales automatically. We do not lack cures. We lack the infrastructure to pay for and deliver them. Indeed, the FDA recently issued draft guidance intended to accelerate the development and review of cell and gene therapies, underscoring how rapidly the science continues to advance. Yet faster approvals alone will not solve the financing and delivery challenges that determine whether patients can ultimately access these treatments. This helps explain current market behavior. Companies often treat fewer patients at higher prices rather than pursue larger volumes at lower prices, because expanding access requires building the delivery system, not just adjusting price.Advertisement The core problem is the absence of a mechanism to finance large, upfront investments in curative care while aligning incentives across payers, providers, and manufacturers. Other sectors solve this routinely. Infrastructure, energy, and housing projects use financing structures that spread costs over time and tie returns to long-term value. Health care remains one of the few sectors without a financing model for life-changing products. In any other sector, an asset delivering decades of value would never be financed in a single year. A more effective model would treat access as a financing problem. Private capital could fund treatment upfront, with repayment linked to outcomes and long-term savings. For example, a third-party intermediary could finance gene therapies at scale and recover costs over time through contracts tied to patient outcomes or reductions in downstream spending. In practice, this could function much like amortizing a large capital investment, spreading costs over years rather than forcing payers to absorb them in a single budget cycle. This would allow Medicaid programs and insurers to align payment with realized value while expanding access and enabling providers to scale capacity. What might this look like in practice? Imagine a state Medicaid program financing a $2 million gene therapy much as a homeowner finances a mortgage. Rather than paying the full cost in a single year, Medicaid could access upfront capital through a specialized financing vehicle and repay that investment over time using a portion of the future savings generated by avoiding years of hospitalizations, emergency care, and chronic disease management. For patients, the result could be faster access to curative therapies rather than waiting for annual budgets, coverage decisions, and funding constraints to catch up with scientific advances. The primary barriers are not scientific, but institutional. Such a model would require new financial and regulatory infrastructure. Current payment systems are built around annual budgets and fragmented insurance coverage, making it difficult to spread costs over time or assign responsibility for long-term outcomes when patients change health plans.Advertisement Policymakers also would need safeguards to ensure that repayment remains tied to clinical success, so that manufacturers, providers, and financiers share some responsibility when therapies fail to deliver their expected benefit. Yet these are financing challenges, not limitations of the therapies themselves. The United States has already invested in developing curative therapies. Without a financing model that matches their value, those cures will remain out of reach for many patients. The next step is not just scientific innovation, but building a system that can finance and deliver cures at scale. William Padula, Ph.D., is a senior scholar at the USC Schaeffer Center for Health Policy & Economics, an associate professor of pharmaceutical and health economics at the University of Southern California Mann School of Pharmacy and Pharmaceutical Science, and chief scientific officer of Stage Analytics. Letter to the editor Have an opinion on this essay? Submit a letter to the editor. Gene therapy, health care costs, Medicaid, STAT+ Submit a correction requestReprints William V. Padula Newsletter The smartest thinkers in life sciences on what's happening — and what's to come Recommended Pharma June 6, 2026 STAT Plus: Lilly shares safety, tolerability data on its next-gen obesity drug Pharma June 6, 2026 STAT Plus: Detailed data show Pfizer’s monthly obesity drug continues to show potential Advertisement The Readout June 5, 2026 STAT Plus: Will Makary’s FDA voucher program survive? 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