PDF(Pubmed)
Abstract：
In this market insights program, AMCP brought together a panel of experts representing various stakeholders: national and regional health plans, integrated health care systems, employer benefits groups, clinical experts, the Centers for Disease Control and Prevention, and patient advocacy organizations. The objectives were to gain insights into the current and evolving treatments in hemophilia, sickle cell disease, and β-thalassemia; measure the effects of recently approved therapies on clinicians, payers, and patients; recognize emerging trends within the stop-loss market; address potential issues and obstacles related to monitoring and reporting outcomes; and identify concerns associated with both existing and emerging contracting and reimbursement models. This article aims to summarize expert perspectives on health care system challenges and strategies concerning the management of inherited blood disorders and to advance managed care professionals\' understanding of their role in supporting care for these patients. The experts emphasized that when shaping coverage policies, a patient-centered approach is crucial, focusing on preserving organ function to maintain eligibility for future gene therapies among individuals with inherited blood disorders. These strategies, including benefit design modifications, specialized provider networks, and centralized mechanisms like registries, are vital for evaluating effectiveness, facilitating decision-making, and managing costs and risks associated with new and emerging treatment options for inherited blood disorders.
摘要：
在这个市场洞察计划中，AMCP召集了代表各种利益相关者的专家小组：国家和区域卫生计划，综合医疗保健系统，雇主福利团体，临床专家,疾病控制和预防中心,和耐心的倡导组织。目标是深入了解当前和不断发展的血友病治疗方法，镰状细胞病,和β-地中海贫血；测量最近批准的疗法对临床医生的影响，付款人,和患者；认识止损市场内的新趋势；解决与监测和报告结果相关的潜在问题和障碍；并确定与现有和新出现的合同和报销模式相关的问题。本文旨在总结有关遗传性血液疾病管理的医疗保健系统挑战和策略的专家观点，并促进管理式护理专业人员对他们在支持这些患者护理中的作用的理解。专家们强调，在制定覆盖政策时，以患者为中心的方法至关重要，专注于保护器官功能，以维持遗传性血液疾病患者未来基因治疗的资格。这些策略,包括福利设计修改，专业提供商网络，和像登记处这样的集中机制，对于评估有效性至关重要，促进决策，以及管理与遗传性血液疾病的新的和新兴的治疗方案相关的成本和风险。
