Centre for Innovation in Regulatory Science (CIRS), 160 Blackfriars Road, London, SE1 8EZ, United Kingdom.
Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Utrecht, The Netherlands.
Ther Innov Regul Sci. 2020 Jan;54(1):55-68. doi: 10.1007/s43441-019-00029-x. Epub 2020 Jan 6.
As regulatory agencies come under increased pressure to review medicines of critical importance through efficient regulatory systems to provide equitable access, the benefits of using expedited review pathways are being explored. These facilitated regulatory pathways (FRPs) provide a variety of review strategies that can also expedite assessments. Stringent regulatory authorities (SRAs) use primary FRPs to accelerate development or to shorten review time. Some emerging national regulatory authorities can implement primary FRPs but are more likely to use secondary FRPs that rely on or recognize an SRA or reference agency decision, the World Health Organization Collaborative Prequalification of Medicines Programme, "altruistic" reviews, or collaborative work sharing. Despite their availability, there are no formal guidelines or consensus for the definition, basic elements, or best practices for FRPs.
Herein, we present a 4-step pragmatic approach to a framework designed to help agencies determine how best to use FRPs. Each step is based on characteristics identified through research, surveys, literature assessments, regulatory capacity categorization analyses, and practical experience.
Step 1 assesses 4 domains of the environment preparedness, step 2 offers process criteria that should be in place to effectively use an FRP, step 3 tiers agencies through a self-assessment of readiness and capacity, and step 4 provides a pathway for agencies to determine the most relevant FRP for their use. Target timelines are proposed for FRPs.
This framework represents the first endeavor to holistically address the multifaceted aspects that should be considered for the effective use of an FRP.
随着监管机构面临越来越大的压力，需要通过高效的监管系统来审查具有重要意义的药物，以公平地提供药物，因此正在探索使用加速审查途径的好处。这些便利的监管途径（FRP）提供了各种审查策略，也可以加速评估。严格的监管机构（SRA）使用主要 FRP 来加速开发或缩短审查时间。一些新兴的国家监管机构可以实施主要 FRP，但更有可能使用次要 FRP，这些 FRP 依赖或承认 SRA 或参考机构的决定、世界卫生组织合作药品预认证计划、“利他主义”审查或合作工作共享。尽管这些途径可用，但对于 FRP 的定义、基本要素或最佳实践，没有正式的准则或共识。
本文提出了一个四步实用方法，旨在帮助机构确定如何最好地使用 FRP。每个步骤都基于通过研究、调查、文献评估、监管能力分类分析和实践经验确定的特征。
第 1 步评估了环境准备情况的 4 个领域，第 2 步提供了有效使用 FRP 应具备的流程标准，第 3 步通过自我评估机构的准备情况和能力对机构进行分层，第 4 步为机构确定最相关的 FRP 提供了途径。还为 FRP 提出了目标时间表。
该框架是首次全面解决有效使用 FRP 应考虑的多方面问题的努力。
