Comment in 2025

The full potential of pharmacogenomics to improve drug development and clinical practice has yet to be realized. Here, we present recommendations to address barriers to its implementation.

Collaborative translational research and adaptive clinical trial design are needed to accelerate the advancement of novel T cell therapies towards achieving cures in solid tumours.

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.