The impact of parallel regulatory-health technology assessment scientific advice on clinical development. Assessing the uptake of regulatory and health technology assessment recommendations
The impact of parallel regulatory-health technology assessment scientific advice on clinical development. Assessing the uptake of regulatory and health technology assessment recommendations
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DOI：
10.1111/bcp.13524
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发表时间：
2018-05-01
影响因子：
3.4
通讯作者：
Vamvakas, Spiros
中科院分区：
文献类型：
--
作者：
Tafuri, Giovanni;Lucas, Ines;Vamvakas, Spiros
平行的监管-卫生技术评估科学建议（PSA）程序允许制造商同时从欧盟监管机构和卫生技术评估（HTA）机构获得关于新药开发计划的反馈。本研究的主要目的是调查PSA是否被整合到临床开发计划中。方法对2010 - 2015年监管机构和卫生管理局各流程提供的PSA内容进行分析。所有寻求PSA的临床研究的发展都使用三个不同的数据库进行跟踪。监管机构和HTA机构提供的建议的采纳率根据两个关键变量进行评估：比较国/s和主要终点。结果：在PSA的实际开发过程中，我们的分析表明，在21项研究中的12项中，制造商实施了比较剂来满足监管机构和至少一个HTA体的需求。对于主要终点，在所有纳入的研究中，制造商同时满足了监管机构和至少一个HTA机构的需求。该分析的一个关键发现是，制造商倾向于根据监管机构和HTA的建议对主要终点和比较物的选择实施开发计划的变更。这也证实了研究比较者的挑战性选择，因为制造商似乎更倾向于满足监管建议。从公共卫生的角度来看，这一领域的持续研究工作至关重要。
AimsThe parallel regulatory-health technology assessment scientific advice (PSA) procedure allows manufacturers to receive simultaneous feedback from both EU regulators and health technology assessment (HTA) bodies on development plans for new medicines. The primary objective of the present study is to investigate whether PSA is integrated in the clinical development programmes for which advice was sought.MethodsContents of PSA provided by regulators and HTA bodies for each procedure between 2010 and 2015 were analysed. The development of all clinical studies for which PSA had been sought was tracked using three different databases. The rate of uptake of the advice provided by regulators and HTA bodies was assessed on two key variables: comparator/s and primary endpoint.ResultsIn terms of uptake of comparator recommendations at the time of PSA in the actual development, our analysis showed that manufacturers implemented comparators to address both the needs of regulators and of at least one HTA body in 12 of 21 studies. For primary endpoints, in all included studies manufacturers addressed both the needs of the regulators and at least one HTA body.ConclusionsOne of the key findings of this analysis is that manufacturers tend to implement changes to the development programme based on both regulatory and HTA advice with regards to the choice of primary endpoint and comparator. It also confirms the challenging choice of the study comparator, for which manufacturers seem to be more inclined to satisfy the regulatory advice. Continuous research efforts in this area are of paramount importance from a public health perspective.