Introduction

Sickle cell disease (SCD) is the most common inherited hemoglobin disorder affecting approximately 100,000 people in the United States (U.S.). Allogeneic hematopoietic cell transplantation (alloHCT) is currently the only curative option for SCD. However, alloHCT is an optional benefit under Medicaid, the largest health program in the US. This means that individual state Medicaid programs can chose to cover alloHCT, define the indications, clinical trial coverage, and/or determine the scope of key health benefits, including donor search, cell procurement, medication, travel and lodging. This study of coverage for alloHCT for patients with SCD aims to understand 1) scope of state Medicaid coverage benefits 2) transplant center (TC) financial counseling practices and experience working with state Medicaid programs.

Methods

Financial coordinators at active Blood and Marrow Transplant Clinical Trial Network (BMT CTN) 1503 clinical trial TCs in 12 states (CA, FL, GA, IL, LA, MI, NC, NY, OH, PA, TX and VA) estimated to have more than 50 newborns diagnosed with SCD in 2016 were contacted. Qualitative, semi-structured interviews 30-60 minutes in duration were conducted via telephone using WebEx teleconference by trained interviewers from Be The Match – NMDP, between May and October 2019. Interviews were recorded, transcribed and analyzed using content analysis, using computer assisted qualitative data analysis software (CAQDAS), NVivo 10.

Results

Of the 26 TCs contacted, a total of 9 TCs representing 7 states participated in semi-structured interviews (Table 1). Preliminary data analysis of interviews revealed that travel and lodging was the most common benefit with limited or absent coverage for alloHCT patients with SCD but showed wide variation by state. Clinical trials were inconsistently covered with some states following Medicaid guidelines for covering alloHCT as part of a clinical trial but not as standard of care while other states covered alloHCT as a part of standard care but not as part of a clinical trial.

Conclusions

There is wide variation in state Medicaid coverage benefits particularly for clinical trial coverage and travel and lodging. These data provide insight into potential areas to influence changes in policy in order to enhance access to life altering curative therapy for SCD.

中文翻译：

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对镰状细胞病（SCD）患者进行同种异体造血细胞移植（alloHCT）的国家医疗补助覆盖范围受益的定性研究

介绍

镰状细胞病（SCD）是最常见的遗传性血红蛋白疾病，在美国（US）感染约100,000人。异基因造血细胞移植（alloHCT）目前是SCD的唯一治疗选择。但是，根据美国最大的医疗计划Medicaid，alloHCT是一项可选福利。这意味着各个州的医疗补助计划可以选择涵盖alloHCT，定义适应症，临床试验覆盖范围和/或确定关键健康益处的范围，包括捐献者搜索，细胞采购，药物，旅行和住宿。这项针对SCD患者的alloHCT承保范围的研究旨在了解1）国家医疗补助覆盖范围的范围2）移植中心（TC）财务咨询实践以及与国家医疗补助计划合作的经验。

方法

活跃的血液和骨髓移植临床试验网络（BMT CTN）1503临床试验TC的财务协调员在12个州（CA，FL，GA，IL，LA，MI，NC，NY，OH，PA，TX和VA）估计具有接触了2016年诊断为SCD的50多名新生儿。在2019年5月至2019年10月之间，由Be The Match – NMDP的受过训练的采访员使用WebEx电话会议，通过电话进行了30-60分钟的定性，半结构化访谈。使用内容分析，计算机辅助定性对访谈进行记录，抄写和分析数据分析软件（CAQDAS），NVivo 10。

结果

在联系的26个TC中，代表7个州的9个TC参加了半结构化访谈（表1）。访谈的初步数据分析显示，旅行和住宿是alloHCT伴SCD患者的最常见益处，覆盖范围有限或缺乏，但因州而异。在某些州，按照医疗补助准则将alloHCT涵盖为临床试验的一部分，但未作为护理标准，而对某些州的临床试验不一致，而其他州则将alloHCT列为标准护理的一部分，但未作为临床试验的一部分。

结论

州医疗补助覆盖利益的差异很大，尤其是在临床试验覆盖以及旅行和住宿方面。这些数据提供了对可能影响政策变化的潜在领域的洞察力，从而增加了改变生活习惯的治疗性SCD的途径。