Centre for Innovation in Regulatory Science, London, UK; Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Utrecht, The Netherlands.
Centre for Innovation in Regulatory Science, London, UK.
Value Health. 2018 Jun;21(6):707-714. doi: 10.1016/j.jval.2017.11.003. Epub 2017 Dec 9.
To evaluate the current practice of companies and agencies to assess the changes made in aligning regulatory and health technology assessment (HTA) stakeholders; to identify areas of commonality of evidentiary requirements that could occur; and to identify strategic issues and trends of regulatory and HTA synergy.
Two separate questionnaires were developed to assess stakeholders' perceptions on regulatory and HTA alignment, one for pharmaceutical companies and the other for regulatory and HTA agencies. The responses were analyzed using descriptive statistics.
Seven regulatory and 8 HTA agencies from Australia, Canada, and Europe and 19 international companies developing innovative medicine responded to the survey. This study provided a snapshot of the current regulatory and HTA landscape. Changes made over the past 5 years were reflected in three main areas: there is an increasing interaction between regulatory and HTA agencies; current conditional regulatory approvals are not always linked with flexible HTA approaches; and companies are more supportive of joint scientific advice. Four types of evidentiary requirements were identified as building blocks for better alignment: acceptable primary end points, inclusion of an active comparator, use of patient-reported outcomes, and choice and use of surrogate end point.
The study showed that the gap between regulatory and HTA requirements has narrowed over the past 5 years. All respondents supported synergy between regulatory and HTA stakeholders, and the study provided several recommendations on how to further improve evidentiary alignment including the provision of joint scientific advice, which was rated as a key strategy by both agencies and companies.
评估公司和机构在评估调整监管和卫生技术评估（HTA）利益相关者方面的现行做法；确定可能出现的证据要求共同领域；并确定监管和 HTA 协同的战略问题和趋势。
为评估制药公司和监管及 HTA 机构的利益相关者对监管和 HTA 调整的看法，分别开发了两份单独的调查问卷。使用描述性统计方法分析了答复。
来自澳大利亚、加拿大和欧洲的 7 个监管机构和 8 个 HTA 机构以及 19 家开发创新药物的国际公司对调查做出了回应。本研究提供了当前监管和 HTA 格局的快照。过去 5 年发生的变化反映在三个主要领域：监管机构和 HTA 机构之间的互动日益增加；目前的有条件监管批准并不总是与灵活的 HTA 方法相关联；以及公司更支持联合科学咨询。确定了四种证据要求类型作为更好调整的基础：可接受的主要终点、纳入活性对照、使用患者报告结果以及选择和使用替代终点。
研究表明，过去 5 年来，监管和 HTA 要求之间的差距已经缩小。所有受访者都支持监管和 HTA 利益相关者之间的协同作用，研究还就如何进一步改善证据一致性提出了一些建议，包括提供联合科学咨询，这被机构和公司都评为一项关键战略。
