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.
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.
