Enabling technologies driving drug research and development

Abstract

One of the strategic goals of the European Medicines Agency (EMA) and the European Medicines Regulatory Network is to support the research and uptake of innovative methods and technologies in the development of medicines. To promote this goal, EMA drew up a list of enabling technologies (ETs), which are novel and fast-growing technologies that have the potential to enable innovation and therefore exert considerable impact on drug development. In this work, enabling technologies identified by the EMA are analysed to measure their impact on drug development by following their journey from publications through early regulatory interactions to clinical trials between 2019 and 2022. This work also reviews the current list of EMA-identified ETs by scrutinising previously unseen innovative technologies identified in EMA submissions data. The analysis shows large variations in the appearance of the various innovative technologies in the different studied data sources, which provided valuable insights into the “Journey of Innovation” that innovative technologies undergo. Several emerging technologies were identified and endorsed for inclusion in the enabling technologies list, whereas some others already on the list were proposed to be excluded due to their low appearance in regulatory interactions as well as clinical trials and publications. Overall, this analysis highlights the relevance and value of continuously scanning and monitoring enabling technologies, supporting Europe’s goal to remain a leader in research and development of innovative technologies, methods, and methodologies relevant to drug development.

Precision medicine in regulatory decision making: Biomarkers used for patient selection in European Public Assessment Reports from 2018 to 2020

“…43,44 The fact that innovative trial designs are not used for development of these medicines more often may be due to the challenges in the planning of these studies, but also in their regulatory assessment. 45 A study of Vass et al 46 showed that novel clinical trial methodologies are, however, the predominant "enabling technology" mentioned in recent EMA procedures, such as the Qualification of Novel Methodologies or Scientific Advice procedures and Innovation Task Force meetings. However, the issued discussed and insights gained during these procedures remain confidential.…”

“…According to the same study, novel biomarkers are more prevalent hits in a PubMed search as compared to a search in the selected EMA procedures or clinical trial registrations. 46 One explanation could be that novel biomarkers are being investigated widely, but have not reached the clinical stage yet. Transparent communication and collaboration between regulators and biomarker developers is essential to move these early developments forward, by sharing information and increase common understanding of evidentiary standards that govern the use of biomarkers in medicine development programs.…”

“…A study of Vass et al 46 showed that novel clinical trial methodologies are, however, the predominant “enabling technology” mentioned in recent EMA procedures, such as the Qualification of Novel Methodologies or Scientific Advice procedures and Innovation Task Force meetings. However, the issued discussed and insights gained during these procedures remain confidential.…”

“…Nevertheless, it is anticipated that this will eventually translate into an increased representation of these methodologies in future marketing authorization dossiers. According to the same study, novel biomarkers are more prevalent hits in a PubMed search as compared to a search in the selected EMA procedures or clinical trial registrations 46 . One explanation could be that novel biomarkers are being investigated widely, but have not reached the clinical stage yet.…”