Comment

As biopharmaceutical companies face mounting pressure to accelerate development timelines and improve the speed and quality of regulatory submissions, many are actively exploring the potential of generative artificial intelligence (GenAI) to transform how regulatory work gets done. This article examines how leading organizations are beginning to apply GenAI to automate content creation, analyse complex data and streamline core regulatory activities — and the critical success factors for driving adoption at scale.

Multiple consortia have been established in the past two decades with the aim of tackling roadblocks in the development of medical products, often by developing new tools to support decision making by regulatory agencies. Here, we highlight lessons learned from these efforts that could help maximize the regulatory impact of consortium-based projects.

Innovative Medicines Initiative projects focused on translational safety have developed a range of tools to improve preclinical assessments of potential drug toxicities, and demonstrated the value of integrating data from multiple companies.

New approach methodologies (NAMs) have the potential to progressively transform medicines development by reducing reliance on animal testing while increasing the relevance of nonclinical data to patients. However, achieving regulatory acceptance of NAMs demands enhanced collaboration, clear guidance and continuous, science-based adaptation of the regulatory environment to accommodate emerging innovation.

Engaging sufficient numbers of patients with cancers that have particular molecular characteristics is a key challenge in building evidence to support precision medicine in oncology. These challenges could be addressed by converging pragmatic clinical trials and hospital cancer informatics infrastructures to integrate cancer care and research.

Clear and harmonized regulatory guidelines are needed to realize the potential of new approach methodologies for improving the predictivity of nonclinical drug candidate assessment.

Launching in 2025, the European Platform for Regulatory Science Research will bring together academia, regulators and other stakeholders to accelerate collaborative regulatory science research solutions.

Oncology innovations, obesity medicines, an uptick for mergers and acquisitions, regulation for artificial intelligence applications and growing consumerization are expected to be key topics.

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.