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Accelerating Clinical Trials in the EU (ACT EU) is an initiative developed by the European Commission, Heads of Medicines Agencies and the European Medicines Agency to transform the clinical trials landscape in the European Union. It aims to foster regulatory, technological and process innovation to support better, faster and optimized clinical trials.
The passage of the FDA Modernization Act 2.0 in 2022 has provided additional impetus to develop new approach methods for predicting the effects of drug candidates in humans from models such as microphysiological systems based on human-derived induced pluripotent stem cells. Here, we highlight progress in the field and strategies to address various challenges, including the application of artificial intelligence tools.
New requirements for clinical trial sponsors to submit diversity action plans to the US Food and Drug Administration (FDA) are an important step to embed planning for diverse representation in clinical trial research. These must not, however, be implemented in ways that are detrimental to other countries’ health and research interests.
The European Innovation Network is working to support the European medical innovation ecosystem by facilitating early dialogue between developers of medicines and regulators, as well as providing a platform for regulators to share information, good practices and expertise.
An unprecedented number of potentially disruptive therapeutic technologies are under development. Forward-looking policies, incentives and infrastructure are needed to harness these advances to provide effective and globally equitable healthcare.
A public–private partnership managed by the FNIH aims to address biological, manufacturing and regulatory challenges to the development of gene therapies for rare diseases.
Open science initiatives that harness human genomic and genetic data could help increase the likelihood of successfully developing new drugs for neurodegenerative diseases.
Clinical trial costs seem to be inflating excessively, without a clear increase in the value of the information gained. Here, we highlight factors that could be driving this trend and discuss potential solutions.
