Ofori-Asenso Richard, Hallgreen Christine E, De Bruin Marie Louise
Copenhagen Centre for Regulatory Science (CORS), Department of Pharmacy, Faculty of Health and Medical Sciences, University of Copenhagen, Copenhagen, Denmark.
Department of Epidemiology and Preventive Medicine, Monash University, Melbourne, VIC, Australia.
Front Med (Lausanne). 2020 Oct 16;7:582634. doi: 10.3389/fmed.2020.582634. eCollection 2020.
The need to optimize drug development and facilitate faster access for patients has ignited discussions around the importance of improving interactions between health technology assessment (HTA) bodies and regulatory agencies. In this study, we conducted a systematic review to examine processes, progress, outcomes, and challenges of harmonization/interaction initiatives between HTA bodies and regulatory agencies. MEDLINE, EMBASE, and the International Pharmaceutical Abstracts database were searched up to 21 October 2019. Searches for gray literature (working papers, commissioned reports, policy documents, etc.) were performed via Google scholar and several institutional websites. An online cross-sectional survey was also conducted among HTA ( = 22) and regulatory agencies ( = 6) across Europe to supplement the systematic review. Overall, we found that while there are areas of divergence, there has been progress over time in narrowing the gap in evidentiary requirements for HTA bodies and regulatory agencies. Most regulatory agencies (4/6; 67%) and half (11/22, 50%) of the HTA bodies reported having a formal link for "collaborating" with the other. Several mechanisms such as early tripartite dialogues, parallel submissions (reviews), adaptive licensing pathways, and postauthorization data generation have been explored as avenues for improving collaboration. A number of pilot initiatives have shown positive effects of these models to reduce the time between regulatory and HTA decisions, which may translate into faster access for patients to life-saving therapies. Thus, future approaches aimed at improving harmonization/interaction between HTA bodies and regulatory agencies should build on these existing models/mechanisms while examining their long-term impacts. Several barriers including legal, organizational, and resource-related factors were also identified, and these need to be addressed to achieve greater alignment in the current regulatory and reimbursement landscape.
优化药物研发并促进患者更快获得药物的需求引发了关于改善卫生技术评估（HTA）机构与监管机构之间互动重要性的讨论。在本研究中，我们进行了一项系统综述，以考察HTA机构与监管机构之间协调/互动举措的流程、进展、成果和挑战。检索了截至2019年10月21日的MEDLINE、EMBASE和国际药学文摘数据库。通过谷歌学术和几个机构网站搜索灰色文献（工作论文、委托报告、政策文件等）。还对欧洲各地的HTA机构（n = 22）和监管机构（n = 6）进行了在线横断面调查，以补充系统综述。总体而言，我们发现，虽然存在分歧领域，但随着时间的推移，在缩小HTA机构和监管机构证据要求差距方面已取得进展。大多数监管机构（4/6；67%）和一半（11/22，50%）的HTA机构报告称与对方有“合作”的正式联系。已探索了一些机制，如早期三方对话、平行提交（评审）、适应性许可途径和批准后数据生成，作为改善合作的途径。一些试点举措显示了这些模式在缩短监管决策与HTA决策之间时间方面的积极效果，这可能转化为患者更快获得救命疗法。因此，未来旨在改善HTA机构与监管机构之间协调/互动的方法应在这些现有模式/机制的基础上进行，并同时考察其长期影响。还确定了包括法律、组织和资源相关因素在内的一些障碍，要在当前的监管和报销格局中实现更大程度的一致，就需要解决这些障碍。
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