National Marrow Donor Program/Be The Match, Minneapolis, Minnesota; Center for International Blood and Marrow Transplant Research, Minneapolis, Minnesota; Aflac Cancer and Blood Disorders Center, Department of Pediatrics, Division of Hematology/Oncology/BMT, Emory University School of Medicine, Atlanta, Georgia.
National Marrow Donor Program/Be The Match, Minneapolis, Minnesota; Center for International Blood and Marrow Transplant Research, Minneapolis, Minnesota; Aflac Cancer and Blood Disorders Center, Department of Pediatrics, Division of Hematology/Oncology/BMT, Emory University School of Medicine, Atlanta, Georgia.
Transplant Cell Ther. 2021 Apr;27(4):345-351. doi: 10.1016/j.jtct.2021.01.022. Epub 2021 Jan 29.
Sickle cell disease (SCD) is the most common inherited hemoglobin disorder, affecting approximately 100,000 people in the United States. Allogeneic hematopoietic cell transplantation (alloHCT), also known as bone marrow transplant (BMT), is currently the only established curative option for SCD. However, alloHCT is an optional benefit under Medicaid. This study of alloHCT coverage for patients with SCD aims to understand the scope of state Medicaid coverage benefits and BMT financial coordinators' experience working with their state Medicaid programs. States estimated to have more than 50 newborns diagnosed with SCD in 2016 and at least one active BMT Clinical Trials Network (1503 [STRIDE 2], NCT02766465) transplant center (TC) were eligible to participate in this study. Qualitative, semi-structured interviews 30 to 60 minutes in length were conducted with BMT financial coordinators via telephone between May and October 2019. A total of 10 BMT financial coordinators from 10 TCs representing eight states (Florida, Georgia, Illinois, Michigan, New York, Pennsylvania, Texas, and Virginia) participated in the semi-structured interviews. Coordinators in all of the included states reported that alloHCT in children with SCD with a human leukocyte antigen-matched sibling donor was covered by their state Medicaid programs. However, only two states (Florida and Texas) had legislative policies mandating coverage of routine medical costs for patients in clinical trials. TCs in two states (Illinois and Pennsylvania) reported accepting out-of-state Medicaid insurance, but only one state (Michigan) covered both travel and lodging for the patient and one caregiver. Four themes emerged when coordinators were asked about their perspectives and experiences working with their corresponding state Medicaid programs: (1) state Medicaid eligibility criteria based on disability were perceived as being restrictive, and Medicaid reimbursement rates were reported to be low; (2) Medicaid fee-for-service plans were perceived as being more comprehensive and easier to navigate compared to comprehensive managed care (CMC) plans; (3) there is a need to address caregiver and financial assistance beyond the health care costs; and (4) completing the insurance authorization process leading up to alloHCT is critical, including peer-to-peer reviews. There is limited legislative policy to help ensure access to clinical trials and provide out-of-state benefits and travel and lodging for Medicaid enrollees with SCD. These data provide insight into potential areas that could influence changes in policy to enhance access to curative therapy for SCD.
镰状细胞病 (SCD) 是最常见的遗传性血红蛋白疾病，影响美国约 10 万人。同种异体造血细胞移植（alloHCT），也称为骨髓移植（BMT），是 SCD 目前唯一公认的治愈方法。然而，alloHCT 是医疗补助计划下的一种可选福利。这项针对 SCD 患者 alloHCT 覆盖范围的研究旨在了解州医疗补助计划覆盖福利的范围以及 BMT 财务协调员与他们所在州医疗补助计划合作的经验。有资格参与这项研究的州，预计在 2016 年有超过 50 名新生儿被诊断患有 SCD，并且至少有一个活跃的 BMT 临床试验网络（STRIDE 2，NCT02766465）移植中心（TC）。2019 年 5 月至 10 月期间，通过电话对来自 10 个 TC 的 10 名 BMT 财务协调员进行了 30 至 60 分钟的定性、半结构化访谈。来自八个州（佛罗里达州、佐治亚州、伊利诺伊州、密歇根州、纽约州、宾夕法尼亚州、得克萨斯州和弗吉尼亚州）的 10 名 TC 财务协调员参加了半结构化访谈。所有参与的州的协调员都报告说，他们所在州的医疗补助计划涵盖了 HLA 匹配的同胞供体儿童的 alloHCT。然而，只有两个州（佛罗里达州和德克萨斯州）有立法政策要求为临床试验中的患者支付常规医疗费用。两个州（伊利诺伊州和宾夕法尼亚州）的 TC 报告说接受外州医疗补助保险，但只有一个州（密歇根州）为患者和一名护理人员支付旅行和住宿费用。当协调员被问及他们与所在州医疗补助计划合作的观点和经验时，出现了四个主题：(1) 基于残疾的州医疗补助资格标准被认为具有限制性，医疗补助报销率被报告较低；(2) 与综合管理保健（CMC）计划相比，医疗补助按服务收费计划被认为更全面，更容易操作；(3) 需要解决护理人员和经济援助问题，而不仅仅是医疗费用；(4) 在 alloHCT 之前完成保险授权过程至关重要，包括同行评审。为了帮助确保 SCD 患者获得临床试验机会并提供州外福利以及旅行和住宿，没有多少立法政策。这些数据提供了一些潜在领域的见解，这些领域可能会影响政策的改变，以加强 SCD 的治愈疗法的可及性。
