Financial Infrastructure Lacks Capacity for $2 Million Gene Therapies, Health Economist Warns
核心洞察
Health economist William Padula reports that the U.S. healthcare system lacks the financial infrastructure to support widespread delivery of gene therapies priced at $2 million or more per patient.
Current payment models built around annual budgets cannot absorb large upfront costs for one-time curative treatments, creating a structural market failure where cures exist but cannot be delivered at scale.
The CMS Cell and Gene Therapy Access Model attempts to address access through multistate purchasing and outcomes-based payments, but remains constrained by the same underlying financing challenges.
The current U.S. healthcare system lacks the necessary financial infrastructure to support the widespread delivery of high-cost gene therapies, according to health economist William Padula, Ph.D., of the USC Schaeffer Center for Health Policy & Economics (搜索). While medical researchers continue to develop curative treatments for diseases like sickle cell, existing payment models struggle to accommodate therapies carrying price tags that reach $2 million or more per patient.
"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," Padula writes in STAT News. "Yet many eligible patients are not receiving them, because America's health care system is not built to pay for cures."
The Structural Disconnect Between Innovation and Payment
Padula argues that the high upfront costs of these one-time treatments create significant budgetary challenges for insurers and healthcare providers. 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, many patients still cannot receive these therapies.
"The result is not just limited access, but a structural market failure: Cures exist, but the system cannot deliver them at scale," Padula states.
Insurers and state Medicaid programs operate on annual budgets, making a $2 million upfront cost difficult to absorb — even for a therapy that delivers long-term value. Coverage remains limited, approvals are slow, and access is constrained, especially outside the rarest conditions.
Policy Efforts and Their Limitations
Under the Biden administration, the Centers for Medicare and Medicaid Services (搜索) 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 model reflects growing recognition of the problem, enabling states to pool risk and negotiate arrangements that tie payment to patient outcomes.
However, Padula notes that these efforts remain constrained by the same underlying challenge. "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."
Why Lower Prices Alone Are Not the Solution
Some policymakers argue that prices will fall as these therapies scale, but Padula contends this assumption does not apply to gene therapies. "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," Padula writes.
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 Padula emphasizes that faster approvals alone will not solve the financing and delivery challenges that determine whether patients can ultimately access these treatments.
A Proposed Financing Model
Padula proposes that 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. 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," Padula explains. This would allow Medicaid programs and insurers to align payment with realized value while expanding access and enabling providers to scale capacity.
He illustrates the concept by comparing it to 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.
Institutional Barriers and Safeguards
Padula acknowledges that 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.
Policymakers would also 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," Padula notes.
"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."