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实现细胞和基因疗法的公平可及:重新思考共付费用

英文原题:Toward equitable access to cell and gene therapies: rethinking co-payments.

查看英文原题

Toward equitable access to cell and gene therapies: rethinking co-payments.

PubMed 2026/05/01(内容时间) Am J Manag Care Q2 · IF 2.1(JCR 2025)

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中文摘要

细胞和基因疗法代表了遗传性疾病、血液系统恶性肿瘤和进行性神经肌肉疾病治疗领域的变革性进展,具有实现持久缓解或治愈的潜力。然而,随着一次性疗法定价现已普遍超过300万美元,其融入美国医疗保健体系在可负担性、可及性和公平性方面面临重大挑战。尽管患者费用分摊仅占细胞和基因疗法总支出的一小部分,但免赔额和共保比例仍可能使商业保险和Medicare受益人面临数千美元的自付费用,为已被疾病相关经济负担所困的患者和家庭制造了实质性的经济障碍。本文探讨了共付额在高成本、医生施用的治愈性疗法背景下的作用,并评估传统的费用分摊理由是否仍然适用。基于CAR-T 细胞疗法利用的新兴证据,我们强调了在种族、社会经济地位、地理区域和保险类型方面持续存在的可及性差异,并讨论了在以严格临床准入标准、密集监管和极低不当使用风险为特征的这一疗法类别中,患者费用分摊如何可能加剧不平等。

展开英文摘要原文

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.

论文信息

作者
Shrank WH、Kelsay E、Fendrick AM
单位
Aradigm, 325 Hudson St, 4th Floor, New York, NY 10013. Email: will@aradigmhealth.com.United States
期刊
The American journal of managed care2026 May
原文标识
PubMed 42189073 · DOI 10.37765/ajmc.2026.89933