Department of Organization and Economics of Pharmacy, Faculty of Pharmacy, Medical University of Sofia, Sofia, Bulgaria.
Pharmacoeconomics Department, Faculty of Pharmacy, University of Medicine and Pharmacy of Craiova, Craiova, Romania.
Front Public Health. 2021 Aug 23;9:729847. doi: 10.3389/fpubh.2021.729847. eCollection 2021.
The significant therapeutic potential of the advanced therapies (ATs) has predetermined the increased interests in their development mainly in the context of rare diseases most of which are genetically determined. However, there are still many challenges in front of the health insurance funds related to the cost-effectiveness and budget impact issues of these therapies. Our aim was to review and analyze the potential of low- and middle-income countries for health technology assessment (HTA) of advanced therapies focusing on Bulgaria, Romania and Poland as reference countries. A literature review of the existing good practices related to HTA of advanced therapies across the world and comparison with the national reality were performed. A list of challenges and issues from the point of view of the payer institution of all analyzed countries was performed. Pilot recommendations on how to overcome the barriers were created based on the existing practices and the potential of the national system. 15 out of 80 articles identified in PubMed were found as applicable to the study scope as most of them were published in the period 2019-2021. Undoubtedly, the main challenges correspond to the high treatment costs, the uncertainty in clinical effectiveness, and poor HTA methodological approaches applicable for ATs worldwide. The issues identified for low and middle-income countries are similar having as well the lack of enough qualified health economists for the purposes of assessment and appraisal of HTA dossiers of the advanced therapies, lack of adequate existing separate financial programs for those therapies, and not preparedness of the health system and the society as a whole for such therapies. Despite the difficulties and challenges, the advanced therapies can be defined as a futuristic therapy for which great discoveries are yet to come. Therefore, each country should consider the implementation of reliable and nationally oriented programs for HTA and adequate financial coverage of these therapies.
先进治疗方法（ATs）的显著治疗潜力决定了人们对其开发的兴趣日益增加，主要集中在大多数由遗传决定的罕见病领域。然而，这些治疗方法的成本效益和预算影响问题仍然给健康保险基金带来了许多挑战。我们的目的是审查和分析保加利亚、罗马尼亚和波兰等参考国家的中低收入国家对先进治疗方法进行健康技术评估（HTA）的潜力。对全球先进治疗方法 HTA 的现有良好实践进行了文献回顾，并与国家实际情况进行了比较。从所有分析国家的支付方机构的角度列出了挑战和问题。根据现有实践和国家系统的潜力，提出了克服障碍的试点建议。在 PubMed 中确定的 80 篇文章中有 15 篇被认为适用于研究范围，因为它们大多发表在 2019-2021 年期间。毫无疑问，主要挑战对应于治疗费用高、临床疗效不确定和全球适用于 ATs 的 HTA 方法学方法不佳。为中低收入国家确定的问题相似，也缺乏足够数量的合格卫生经济学家来评估和评估先进治疗方法的 HTA 文件，缺乏针对这些疗法的适当的现有单独财务计划，以及卫生系统和整个社会对这些疗法的准备不足。尽管存在困难和挑战，但先进的治疗方法可以被定义为一种具有巨大发现潜力的未来疗法。因此，每个国家都应考虑实施可靠的、面向国家的 HTA 计划，并为这些疗法提供充足的财务覆盖。
