Comment in 2024

The Open Targets consortium was founded in 2014 to translate insights from genetics and functional genomics into identifying and prioritizing therapeutic targets. We highlight key achievements and insights stemming from this partnership of academic and industry scientists.

New approaches are needed to streamline clinical trials of drugs for patients with rare diseases. Digital biomarkers offer one such approach, but several challenges must be addressed to realize their potential.

Drug discovery institutes comprised of experienced drug discovery scientists collaborating with fundamental biomedical researchers provide solutions to many of the challenges in translating biomedical research.

The AMR Accelerator is an Innovative Medicines Initiative programme integrating nine projects with the shared goal of progressing the development of new antibiotics and building antimicrobial resistance research capability. Five years in, we reflect on the programme’s value, results and key challenge: ensuring the sustainability of assets, infrastructures and expertise.

Clinical trial participant compensation is badly overdue for thoughtful reform. The first step: generating consensus across the various stakeholders, including patients, who make up the research ecosystem.

The DISRUPT-DS roundtable, which brings together data science leaders from large pharmaceutical companies, aims to be a forum for sharing experiences and networking, for shaping industry-level topics and for amplifying the role of data science across pharmaceutical R&D.

Drugs that modulate fundamental mechanisms of ageing offer the promise of substantially improving the health of ageing populations, but innovative approaches to identify and evaluate such ‘gerotherapeutics’ are needed.

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.