The landscape of treatment options for rare diseases remains profoundly challenging, as over 90 percent of the approximately 7,000 identified rare conditions lack available therapies. Among the potential avenues for addressing these unmet medical needs are cell and gene therapies (CGTs), which have shown transformative promise by correcting underlying genetic and cellular abnormalities responsible for these disorders. These innovative treatments, while often life-altering and sometimes curative, carry exorbitant costs that strain healthcare systems, payers, and patients alike. The financial implications restrict access, fostering a pressing debate about the sustainability and equity of deploying CGTs on a broad scale.
As of May 2025, the United States Food and Drug Administration (FDA) had approved 46 distinct CGTs, reflecting an accelerating pace of innovation within this therapeutic realm. Market projections anticipate that these therapies will generate annual list price revenues nearing $24 billion by the year 2030 in the US alone. This rapid market expansion places enormous pressure on existing insurance frameworks, which are ill-equipped to manage the substantial up-front expenses associated with CGT administration. The economic burden is especially significant because these therapies often involve one-time or short-duration interventions with lifelong benefits, distinguishing them from chronic treatment modalities.
A perspective article recently published in JAMA Internal Medicine on October 20, 2025, by Dr. Audun Brendbekken of the University of Bergen, Norway, and Dr. Stacie Dusetzina of Vanderbilt University, USA, critically examines the divergent regulatory and reimbursement landscapes for CGTs in the US and Europe. The authors underscore the inherent complexities in these systems, noting the fragmented nature of the American health insurance market and the comparatively centralized European regulatory approach. Their analysis points to the urgent need for systemic reform in the US to ensure equitable access to life-saving therapies without imposing untenable costs on the system.
In Europe, the European Medicines Agency (EMA) has granted approval to 19 CGTs, with most Western European countries achieving reimbursement for these therapies within two years post-approval. European health systems typically base reimbursement decisions on stringent evaluations of a therapy’s added value against established standards of care. This value-based pricing model fosters negotiations that stabilize or reduce prices over time, enhancing long-term affordability. The iterative renegotiation process, conducted under the auspices of uniform pricing policies, ensures that payers minimize financial risk while maintaining patient access.
Conversely, the US healthcare ecosystem’s patchwork private insurance market introduces barriers to consistent CGT coverage. High up-front costs dissuade coverage by insurers, especially as patients frequently change insurance providers, complicating continuity of care and reimbursement frameworks. Moreover, budgetary limitations in state Medicaid programs further restrict access for vulnerable patient populations. These systemic challenges produce inequities in therapy penetration, disproportionately affecting those without stable insurance or adequate coverage provisions.
To address these disparities, the authors advocate for a single-payer insurance model within the US, particularly tailored to high-cost, short-duration therapies such as CGTs. A nationwide single-payer system could streamline coverage, reduce administrative fragmentation, and consolidate negotiating power to drive down prices. Such a model would diminish inequalities in patient access by offering universal coverage, thereby ensuring that life-saving interventions are not withheld due to payer intricacies or cost concerns. For manufacturers, this paradigm promises a more predictable market environment conducive to sustained innovation.
However, despite the potential benefits, the transition to a single-payer framework raises critical questions about funding sources, eligibility criteria, and implementation strategies. Deliberation on how to equitably finance this model, allocate resources, and define beneficiary populations remains paramount. These considerations must balance fiscal sustainability with ethical imperatives to provide timely access to groundbreaking treatments for rare disease patients.
The technical underpinnings of CGTs involve sophisticated genetic engineering and cellular manipulation methods, including viral vector-mediated gene insertion, CRISPR-based gene editing, and autologous stem cell transplantation. These therapies often operate by either adding functional copies of defective genes, removing deleterious mutations, or modulating immune responses to eradicate pathological cells. The complexity of manufacturing these therapies, coupled with stringent safety and efficacy testing mandated by regulatory authorities, contributes to their elevated costs.
Additionally, the manufacturing and distribution processes for CGTs diverge markedly from traditional pharmaceuticals. Many CGTs require individualized production cycles tailored to each patient, particularly autologous cell therapies, which utilize the patient’s own cells as raw material. This bespoke approach necessitates specialized facilities, rigorous quality control, and cold chain logistics, all of which compound financial and operational challenges.
Economic evaluations of CGTs further complicate reimbursement decisions. Standard cost-effectiveness models falter when applied to one-time treatments with potentially lifelong benefits, as traditional metrics often undervalue long-term improvements in patient quality of life and health system savings. Innovative payment frameworks, including outcomes-based agreements and annuity payment models, are emerging to reconcile these challenges but have yet to achieve widespread adoption in the US market.
From a policy perspective, the US faces significant hurdles in harmonizing payer systems, generating legislative consensus, and overcoming entrenched interests resistant to systemic overhaul. Lessons from Europe’s coordinated access pathways, health technology assessments, and centralized negotiation mechanisms offer valuable templates. These models emphasize collaboration among regulators, payers, manufacturers, and patient advocacy groups to optimize therapeutic access while containing costs.
In summation, the promise embodied by CGTs to revolutionize treatment for rare, otherwise untreatable diseases stands in stark contrast to the formidable economic and structural barriers limiting patient access in the US. The call for a nationwide single-payer model articulated by Brendbekken and Dusetzina represents a visionary but pragmatic proposal to reconcile innovation with affordability. As the pipeline of CGTs burgeons, proactive policy architecture will be indispensable to fulfill the therapeutic potential of these advances for all patients, regardless of socioeconomic status.
The imperative for action is clear: without decisive reform, the US risks perpetuating inequities in healthcare access and squandering opportunities to alleviate profound medical unmet needs. Bridging the gap between scientific breakthroughs and equitable clinical implementation demands integrating policy innovation, economic foresight, and ethical commitment. Only through such a multifaceted approach can the transformative promise of cell and gene therapies be realized on a national scale, ushering in a new era of precision medicine for rare disease populations.
Subject of Research: People
Article Title: Advancing a US Single-Payer Model for Cell and Gene Therapy
News Publication Date: 20-Oct-2025
Web References: https://doi.org/10.1001/jamainternmed.2025.5058
References: Brendbekken A, Dusetzina S. Advancing a US Single-Payer Model for Cell and Gene Therapy. JAMA Internal Medicine. 2025;DOI:10.1001/jamainternmed.2025.5058.
Image Credits: UiB
Keywords: Cell and gene therapy, rare diseases, single-payer system, health insurance, reimbursement, healthcare policy, precision medicine, innovative therapies, economic evaluation, healthcare access, regulatory approval, US healthcare system
