A preliminary application of a haemophilia value framework to emerging therapies in haemophilia
A preliminary application of a haemophilia value framework to emerging therapies in haemophilia
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DOI：
10.1111/hae.14511
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发表时间：
2022-03-01
期刊：
影响因子：
3.9
通讯作者：
O'Mahony, Brian
中科院分区：
文献类型：
--
作者：
Skinner, Mark W.;Dolan, Gerry;O'Mahony, Brian
前言预计新疗法的出现将改善血友病患者的临床结局和生活质量。在罕见疾病中进行随机临床试验的挑战导致缺乏直接的头对头比较，以支持不同治疗之间基于价值的决策。方法：我们对新的和新兴的血友病A和B治疗（延长半衰期[EHL]替代因子，非替代疗法[NRT]和基因疗法[GT]）进行了文献综述，以确定先前在血友病价值框架中定义的以患者为中心的差异性结局。由于文献包括所有出版物类型（例如，调查、建模研究、评论/评论），收集的数据被分配证据等级分数。结果在不同类别的治疗中，出血被确定为最常报告的分化结局，EHL、NRT和GT均优于对照替代疗法。EHL治疗血友病A和B以及NRT治疗血友病A在第1层结局中表现出良好的代表性（达到/保持健康状态），而更多的出版物被确定为第2级NRT的（恢复过程）结局优于EHL或GT。（健康的可持续性），突破性出血的频率代表了EHL的差异性结局（A型和B型血友病）、NRT（仅A型血友病）和GT（仅B型血友病），而持续良好的健康状况是大多数比较的区别。结论：我们证明了血友病价值框架作为有效比较治疗的共同核心结局集的实用性。该框架的应用将作为一个有用的决策工具，为患者，临床医生，并在卫生技术评估。考虑要点随着多个医学领域出现高成本、改变范式的治疗方法，我们血友病界需要配备评估人员所期望的工具，以满足日益增长的更严格循证价值评估的需求。传统的获取工具箱需要发展，以满足治疗方案的范式转变。价值不再仅由年出血率定义。为了实现新疗法的全面影响，我们需要利用价值框架等工具来组织证据，识别数据差距，并在多方面评估患者定义的有意义的结果。血友病价值框架是组织现有证据和识别证据差距的有效工具。这可用于评估血友病新兴疗法的价值，利用通过随机临床试验和真实的世界证据生成产生的数据。这是一个将价值框架纳入官方提交给当局的呼吁，因为它捕捉了更广泛的结果，包括患者有意义的结果，以更好地评估新疗法的潜在益处。
Introduction Emergence of new therapies are anticipated to improve clinical outcomes and quality of life of persons with haemophilia. Challenges in conducting randomized clinical trials in rare diseases have resulted in a lack of direct head-to-head comparisons to support value-based decision-making between different treatments. Methods We conducted a literature review for new and emerging haemophilia A and B therapies (extended half-life [EHL] replacement factor, non-replacement therapies [NRT], and gene therapies [GT]) to identify differentiating patient-centred outcomes defined previously in a haemophilia value framework. Since the literature included all publication types (e.g., surveys, modelling studies, commentaries/reviews), collected data were assigned level of evidence scores. Results Across different classes of therapies, bleeding was determined as the most frequently reported differentiating outcome, with EHL, NRT, and GT each demonstrating an advantage over comparator replacement therapies. EHL therapies for haemophilia A and B and NRT for haemophilia A showed good representation across Tier 1 outcomes (health status achieved/retained), while more publications were identified with Tier 2 (process of recovery) outcomes for NRT than EHL or GT. In Tier 3 (sustainability of health), frequency of breakthrough bleeds represented a differentiating outcome for EHL (both haemophilia A and B), NRT (haemophilia A only), and GT (haemophilia B only), whereas sustained good health was differentiating for most comparisons. Conclusions We demonstrate the utility of the haemophilia value framework as a common core outcome set for effectively comparing therapies. Application of this framework will serve as a useful decision-making tool for patients, clinicians, and within health technology assessments. KEY POINTS OF CONSIDERATION With the emergence of high-cost, paradigm changing treatments across multiple areas of medicine, we, the haemophilia community, need to be equipped to meet the growing demands for more rigorous evidence-based value assessments using the tools expected by assessors. The traditional access toolbox needs to evolve to meet the paradigm shift in treatment options. Value can no longer be defined by annualized bleed rates alone. To realize the full impact of new therapies, we need to utilize tools, such as a value framework, to organize evidence, identify data gaps, and assess patient-defined, meaningful outcomes across a multi-faceted dimension. The haemophilia value framework is an effective tool for organizing the available evidence and identifying gaps in the evidence. This can be used for assessing the value of emerging therapies in haemophilia utilizing data generated through randomized clinical trials and real world evidence generation. This is a call for incorporating the Value Framework into official submissions to authorities, as it captures a broader range of outcomes, including patient meaningful outcomes, in ways that better assess the potential benefits of new therapies.