Toward Equitable Access to Cell and Gene Therapies: Rethinking Co-Payments

ABSTRACT

Cell and gene therapies represent a transformative advance in the treatment of inherited disorders, hematologic malignancies, and progressive neuromuscular diseases, offering the potential for durable remission or cure. However, with onetime therapies now routinely priced above $3 million, their integration into the US health care system presents significant challenges related to affordability, access, and equity. Although patient cost sharing represents a small fraction of total cell and gene therapy spending, deductibles and coinsurance can expose commercially insured and Medicare beneficiaries to thousands of dollars in out-of-pocket costs, creating meaningful financial barriers for patients and families already burdened by illness-related economic strain.

This article examines the role of co-payments in the context of high-cost, physician-administered, curative therapies and evaluates whether traditional cost-sharing rationales remain applicable. Drawing on emerging evidence from chimeric antigen receptor T-cell therapy utilization, we highlight persistent disparities in access by race, socioeconomic status, geography, and insurance type and discuss how patient cost sharing may exacerbate inequities in a category characterized by strict clinical eligibility, intensive oversight, and minimal risk of inappropriate use.

Am J Manag Care. 2026;32(5):257-259. https://www.doi.org/10.37765/ajmc.2026.89933

Takeaway Points

• High co-payments for potentially curative cell and gene therapies create avoidable access barriers.
• Value-based insurance design should eliminate patient cost sharing for these therapies.

Cell and gene therapies represent one of the most extraordinary scientific advances in modern medicine. For patients with devastating inherited disorders, hematologic malignancies, or progressive neuromuscular disease, these therapies offer something frequently lacking from clinical care: the possibility of durable remission or even cure. These therapies are remarkable both in their promise and their price, with onetime treatments now routinely exceeding $3 million per patient, presenting a defining challenge for a strained US health care system that is already confronting an affordability crisis.

As these therapies move to standard of care for a greater number of, and more common, chronic conditions, the question is no longer whether the science works. It is whether our health system can provide access to those who can benefit and deliver them equitably and affordably.

Co-Payments in the Era of Million-Dollar Therapies

Today, patient cost sharing for cell and gene therapies varies widely by insurance type and benefit design. For Medicaid beneficiaries, out-of-pocket costs are often minimal or zero, although other utilization management strategies (eg, prior authorization, step edits) restrict access. For patients with commercial insurance or Medicare Advantage, however, cost sharing frequently takes the form of deductibles and coinsurance that quickly push patients to their health plan’s annual out-of-pocket maximum, not including associated costs such as travel and caregiving. Even traditional Medicare beneficiaries without supplemental coverage may face substantial financial exposure.

These out-of-pocket costs for patients and families—many already burdened by chronic illness, disability, and lost income—often lead to online fundraisers, crippling medical debt, and personal bankruptcy. Given the detrimental impact of these outcomes and the fact that patient contributions represent a small fraction of total cell and gene therapy spending, a deeper question arises: What purpose does cost sharing serve in this category of care?

Evidence of Access Barriers and Disparities

Access to cell and gene therapies remains uneven. The best evidence to date comes from studies about utilization of chimeric antigen receptor T-cell therapy, whose findings demonstrate disparities by race, socioeconomic status, geography, and insurance type.^1,2 Patients treated at well-resourced academic centers, with generous insurance (often employer-sponsored) coverage and care navigation support, are far more likely to receive these therapies,³ consistent with other expensive, innovative technologies.

Patient cost sharing is not the sole driver of these disparities, but it is a visible and avoidable barrier layered on top of others (eg, limited center availability, complex referral pathways, and payer prior authorization processes). There is extensive evidence that even small co-payments meaningfully influence utilization, even for highly effective, essential therapies.⁴ Even small reductions in co-payments have been shown to reduce disparities in use and outcomes.⁵ In a category where eligibility is narrowly defined, clinical oversight is intense, and inappropriate use or overuse is extremely unlikely, adding financial barriers risks undermining access to these therapies for populations of patients who stand to benefit most.⁶

Do Co-Payments Make Sense for Curative Therapies?

Patient cost sharing is traditionally justified as a tool to reduce low-value utilization and encourage consumer price sensitivity. That logic collapses when applied to a $3 million physician-administered therapy delivered at a handful of specialized centers to patients who meet strict clinical criteria.

It is reasonable to posit that consumption of gene therapy is not discretionary. Patients do not “overuse” cures, in particular when there are no other alternative therapies. Once a therapy has cleared stringent regulatory thresholds for safety and effectiveness, patients’ out-of-pocket obligation no longer functions as a value signal but punitively operates as a rationing mechanism. It is difficult to justify shifting thousands of dollars of financial responsibility onto patients—often those who cannot afford this burden—who have no meaningful clinical alternative.

Important Exceptions and Nuance

This does not mean that all advanced therapies should be treated identically for every patient who is indicated. First, therapies with limited or uncertain effectiveness may warrant different treatment. For example, when clinical benefit is modest, heterogeneous, or where the evidence is still evolving (eg, existing therapy for Duchenne muscular dystrophy), stronger utilization controls or alternative payment structures (eg, outcome guarantees/warranties) may be appropriate. Coverage with evidence development has been applied in similar situations.

Second, conditions for which effective chronic therapies already exist present a different calculus. For example, curative therapy for type 1 diabetes, a condition that impacts more than 1 million Americans and has well-established, effective, long-term management strategies, is under development. Multimillion-dollar therapies may seem unfeasible, and therefore, some form of comparative value assessment is necessary, and cost-sharing requirements might be considered.

A Better Way to Put “Skin in the Game”

In general, patients are not the right locus for financial risk in this category. Government and private payers are largely responsible for paying for almost all the cost of these therapies today. Increasingly, these purchasers will require the use of high-quality, high-value accredited facilities that are equipped to make sound, data-driven decisions about appropriateness of utilization and contract with these providers in collaborative ways to promote efficient and sustainable care. Manufacturers are increasingly expected to take on some of the risk. Value-based contracts, in which the costs of therapy are recouped if therapies fail to produce expected outcomes, have become more commonplace and expected for this category of therapy. The binary nature of the outcomes for many of these therapies facilitates administration of these contracts, as outcome definitions are more straightforward to determine and measure for therapies that promise to cure the targeted condition.

One promising approach is to require long-term patient participation in outcomes registries as a condition of coverage and the elimination of cost sharing. Robust, longitudinal data collection will foster real-world evidence on durability, safety, and functional outcomes—data that are essential for refining pricing, informing future coverage decisions, and sustaining public trust. This approach asks patients to contribute, but in a nonfinancial way, aligning individual access with collective learning and a better understanding of the value of treatment.

True North

The health care system should establish value-based insurance design for cell and gene therapies as an explicit goal. A natural consequence would be the elimination of co-payments for high-value cell and gene therapies in specific clinical scenarios. Access to cures—with potential to alter the course of a lifetime—should be guided by probability of clinical benefit and not be gated by a patient’s ability to pay. As the pipeline for this category of therapy grows, we should establish reasonable norms now in order to realize the promise of this extraordinary new science and ensure that we narrow, rather than widen, the very inequities that these therapies have the power to eliminate.

Author Affiliations: Aradigm (WHS), New York, NY; Business Group on Health (EK), Washington, DC; Center for Value-Based Insurance Design (AMF), Ann Arbor, MI; Department of Internal Medicine, University of Michigan (AMF), Ann Arbor, MI.

Source of Funding: None.

Author Disclosures: Dr Shrank is CEO of Aradigm Inc, a company that manages the clinical and financial risk for cell and gene therapy; has equity in Humana and a16z; serves on the boards of RxCap, AnewHealth, NCQA, and the Brown University School of Public Health; and is an adviser to UpDoc and Thrive Health Tech. Dr Fendrick reports receiving consulting fees from AbbVie, Better Medicare Alliance, Centivo, Clover Insurance Company, Community Oncology Alliance, Eebu Health, Elektra Health, Employee Benefits Research Institute, Exact Sciences, GRAIL, Hopewell Fund, Johnson & Johnson, Medtronic, MedZed, Merck, Mother Goose Health, Phathom Pharmaceuticals, Proton Intelligence Inc, Sempre Health, Sera Prognostics, Silver Fern Health, UnitedHealth Group, Virginia Center for Health Innovation, Washington Health Benefit Exchange, and Wellth, and serving as a partner for VBID Health and co–editor in chief of The American Journal of Managed Care. Ms Kelsay reports no relationship or financial interest with any entity that would pose a conflict of interest with the subject matter of this article.

Authorship Information: Concept and design (WHS, EK, AMF); drafting of the manuscript (WHS, EK, AMF); critical revision of the manuscript for important intellectual content (WHS, EK, AMF); administrative, technical, or logistic support (WHS); and supervision (WHS).

Address Correspondence to: William H. Shrank, MD, MSHS, Aradigm, 325 Hudson St, 4th Floor, New York, NY 10013. Email: will@aradigmhealth.com.

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