The treatment landscape for haemophilia continues to rapidly develop, and expectations for future treatment success are high. There is limited information on the challenges to accessing new and innovative therapies. The aim of this study was to explore challenges with accessing haemophilia treatment from the perspective of healthcare professionals (HCPs). A crosssectional study design was used. A pilot-tested, online survey was distributed to haemophilia treatment centres in Australia, Canada, France, Italy, New Zealand, Republic of Ireland, Turkey, the United States, and the United Kingdom. The questionnaire covered questions on product access, economic considerations, health technology assessment requirements, and patient organization involvement. The results were analyzed descriptively using SPSS. A total of 154 HCPs completed the questionnaire. There was heterogeneity across countries, regions, and centres regarding HCPs' knowledge of access to novel recently developed treatments. Notable limitations to access were reported such as differences in access based on age of patient and type of product, economic considerations, and the growing influence of HTA bodies. Many countries have a hemophilia patient organization that does not have a vote at the decision-making table. There is a need to empower HCPs to better understand national healthcare structures and decisions that lead to access limitations. Requirements from HTA bodies must be understood to optimally design clinical studies and value generation of treatment options. This may strengthen the haemophilia treatment centre's voice to collectively mandate for exchange with key involved individuals, such as the payers and politicians for the provision of optimal therapy.

中文翻译：

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与在常规护理中获得最近开发的血友病治疗相关的挑战：医疗保健专业人员的观点。


血友病的治疗前景继续迅速发展，对未来治疗成功的期望很高。关于获得新的和创新疗法所面临的挑战的信息有限。本研究的目的是从医疗保健专业人员 （HCP） 的角度探讨获得血友病治疗的挑战。使用横断面研究设计。向澳大利亚、加拿大、法国、意大利、新西兰、爱尔兰共和国、土耳其、美国和英国的血友病治疗中心分发了一项经过试点测试的在线调查。问卷涵盖有关产品获取、经济考虑、卫生技术评估要求和患者组织参与的问题。使用 SPSS 对结果进行描述性分析。共有 154 名 HCP 完成了问卷。国家、区域和中心在 HCP 获得最近开发的新型治疗方法的知识方面存在异质性。据报道，可及性存在显著限制，例如根据患者年龄和产品类型、经济考虑以及 HTA 机构日益增长的影响力而存在的可及性差异。许多国家都有一个血友病患者组织，该组织在决策桌上没有投票权。有必要使 HCP 能够更好地了解导致访问受限的国家医疗保健结构和决策。必须了解 HTA 机构的要求，以优化设计临床研究和治疗方案的价值生成。这可能会加强血友病治疗中心的声音，共同授权与关键相关个人（例如付款人和政治家）进行交流，以提供最佳治疗。