Velikanova Rimma, Wolters Sharon, Hofstra Hinko S, Postma Maarten J, Boersma Cornelis
Department of Health Sciences, University of Groningen, University Medical Center Groningen, Hanzeplein 1, 9713 GZ Groningen, The Netherlands;
Asc Academics, Hereweg 120, 9725 AK Groningen, The Netherlands;
J Mark Access Health Policy. 2024 Jul 30;12(3):181-198. doi: 10.3390/jmahp12030015. eCollection 2024 Sep.
With the increasing pipeline of cell and gene therapies (CGTs) and the expected surge in the number of approvals, understanding the market access landscape becomes crucial for timely patient access. This study evaluates the challenges Dutch stakeholders encounter in CGT market access, offering insights for improving time-to-patient access. A traditional literature review was conducted to identify market access challenges and solutions for CGTs. Based on the findings, participants for semi-structured interviews, designed using an interview guide adapted to the Dutch context, were selected to capture diverse perspectives on market access. This review included 124 relevant articles out of 2449, covering several aspects of market access of CGTs. Subsequently, interviews with 16 stakeholders from academia, patient advocacy groups, manufacturers, health insurers, payers, hospital pharmacists, healthcare practitioners, and the Association of Innovative Medicines were conducted. Stakeholders identified challenges and proposed solutions for reimbursement package management, clinical trials, health economics, payment models, and procedural and organisational aspects. Thematic analysis revealed unique country-specific challenges and solutions in the Netherlands. This research provides insights into these challenges and potential solutions, emphasising the need for collaborative efforts among stakeholders to develop practical and multidisciplinary measures to improve the market access landscape for CGTs in the country.
随着细胞和基因疗法（CGT）的研发进程不断推进，获批数量预计也将大幅增长，因此了解市场准入情况对于患者及时获得治疗至关重要。本研究评估了荷兰利益相关者在CGT市场准入方面遇到的挑战，为缩短患者获得治疗的时间提供见解。通过传统文献综述来确定CGT的市场准入挑战和解决方案。基于研究结果，选择了参与半结构化访谈的人员，访谈指南根据荷兰情况进行了调整，以获取关于市场准入的不同观点。该综述从2449篇文章中筛选出124篇相关文章，涵盖了CGT市场准入的多个方面。随后，对来自学术界、患者权益倡导组织、制造商、健康保险公司、支付方、医院药剂师、医疗从业者以及创新药物协会的16名利益相关者进行了访谈。利益相关者确定了报销套餐管理、临床试验、健康经济学、支付模式以及程序和组织方面的挑战，并提出了解决方案。主题分析揭示了荷兰特有的国家层面的挑战和解决方案。本研究提供了对这些挑战和潜在解决方案的见解，强调利益相关者需要共同努力，制定切实可行的多学科措施，以改善该国CGT的市场准入情况。
