Cell and Gene Therapies Can Cure Disease. Can We Afford to Pay for Them?

Imagine a disease that has shaped every year of your life, one dose away from being gone for good. That is not a hypothetical anymore. For people living with sickle cell disease, spinal muscular atrophy, hemophilia, and certain blood cancers, a new class of medicine called cell and gene therapy has turned what used to be science fiction into something close to a cure.

These therapies work by fixing the problem at its source. Depending on the approach, a faulty or missing gene gets repaired, reprogrammed, or replaced, sometimes inside the body, sometimes in a lab before being reinfused. The result, in many cases, is a single treatment that does the work of a lifetime of pills, infusions, and hospital visits. There are thousands more of these therapies in development, aimed at conditions far more common than today's rare-disease targets, including type 1 diabetes, multiple sclerosis, and Parkinson's. Researchers are even exploring versions that could someday replace GLP-1 drugs for weight management.

Because the science is so new, we do not yet have decades of data on long-term side effects or durability. That uncertainty is real. But for families watching a child struggle with a disease that used to have no real answer, the hope these therapies offer is not abstract. It is the difference between a life sentence and a second chance.

Here is the catch. A single course of treatment can cost more than 4 million dollars, according to the American Society of Gene and Cell Therapy. That number sounds staggering until you compare it to something else: the lifetime medical costs of managing a disease like hemophilia without a cure, which can actually run higher over time. The therapy is not necessarily more expensive. It just asks you to pay all of it at once, upfront, instead of spreading it across a lifetime of care.

That timing problem is where the whole system starts to strain. No individual patient can absorb a bill like that, so the cost lands on whoever insures them, whether that is Medicare, Medicaid, a commercial insurer, or an employer that self-funds its health plan. Unlike a lifetime of smaller costs that get shared across a large pool of people over many years, this cost hits one payer, all at once, for one person. Health systems face a similar bind. Standing up a treatment center for a single rare therapy can cost millions of dollars, and because the patient population is so small, hospitals often have to negotiate drug prices case by case and carry the financial burden on their books for months before insurance reimbursement arrives.

A System Pulling in Opposite Directions

Think of it like the old Tragedy of the Commons, the idea that a shared resource gets ruined not by bad intentions but by everyone protecting their own narrow interest. Apply that logic here and the outcome is grim. Insurers hesitate to authorize a treatment, even one that could transform a member's life, because the cost could destabilize coverage for everyone else in the plan. Hospitals hesitate to offer the therapy, even when it is exactly the kind of lifesaving care they exist to provide, because the financial risk could threaten the whole institution. Drug makers, having poured enormous resources into developing these treatments, resist lowering prices, which only deepens the strain on payers and providers.

Nobody in this picture is acting out of malice. Everyone is behaving rationally within a system that was never built for medicines like this. But if each party keeps protecting itself, patients lose access to treatments that could save or transform their lives, and drug developers lose the financial incentive to keep innovating. The entire ecosystem that made these breakthroughs possible starts to erode from the inside.

Will Shrank, a physician and CEO of Aradigm Health, has proposed something that has never really been tried for an entire class of drugs: treat cell and gene therapies as a separate category outside the normal insurance and reimbursement system entirely. Under this model, insurers and employers would pool money into a shared fund specifically for these high-cost treatments, spreading the financial risk across many payers instead of concentrating it on whoever happens to insure the patient. That kind of pooling stabilizes premiums for everyone and caps how much exposure any single payer has to carry alone.

Drug manufacturers would adopt transparent cost-plus pricing, tied to patient outcomes, which protects their ability to profit while easing the burden on the rest of the system. Hospitals and health systems would coordinate their investments in treatment centers rather than each building one independently, making access more consistent for patients regardless of where they live.

It is an unusual idea for a free-market healthcare system, but it is not unprecedented elsewhere. Think about how we pay for electricity. Building power plants and grids is enormously expensive, yet we do not let individual utilities absorb that cost alone or pass it entirely onto whichever customer happens to need power that month. Rate-based financing spreads the cost of that infrastructure over time and across the public, because we have decided, as a society, that reliable power access matters enough to warrant that kind of collective investment. Cell and gene therapy may need the same kind of thinking.

Some insurers and startups are already experimenting with risk-pooling arrangements for these therapies. That is encouraging, but a patchwork of separate private solutions carries its own danger: some patients and some diseases could end up covered well, while others get left behind entirely, depending on which insurer or employer they happen to have. Consolidation around a shared approach would smooth out that unevenness and make outcomes less dependent on luck.

Nothing about this should be treated as permanent. As more of these therapies reach the market and treatment infrastructure matures, prices are likely to fall. Any shared-risk framework will need to evolve alongside that, adjusting its structure for different drugs as the economics shift over time.

Why It Matters

What is really at stake here is not just one category of expensive drugs. It is whether a healthcare system built on fee-for-service economics can accommodate a genuinely new kind of medicine, one that front-loads a lifetime of value into a single moment of treatment. Get this wrong, and families in desperate need of a cure may never gain access to one that already exists. Get it right, and we preserve something precious: an innovation ecosystem capable of turning yesterday's science fiction into tomorrow's standard of care.