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Artificial intelligence tools are increasingly being applied in the development and use of medicines. Here, we provide a regulatory perspective on such applications.
The COVID-19 vaccine effort was the most rapid global response to a health crisis in history. The challenge of managing unparalleled volumes of safety information provided unique opportunities to evaluate the robustness of current pharmaceutical industry pharmacovigilance practices and test novel approaches to strengthen safety signal detection, evaluation and communication.
The Assay Guidance Manual programme, a translational science education and training initiative at the US National Center for Advancing Translational Sciences (NCATS), aims to help bridge the gap between research discoveries and the delivery of new therapies by creating and disseminating experimental protocols and standards for rigour in preclinical translational research.
The Accelerating Medicines Partnership in Heart Failure is designed to deliver tools and knowledge that enable the development of new approaches to treat heart failure with preserved ejection fraction, which is considered the largest unmet need in cardiovascular medicine.
A decade on from the launch of the pioneering autism biomarker discovery initiative EU-AIMS, here we highlight advances in biomarker discovery and validation from the follow-on AIMS-2-TRIALS programme, which aims to improve the wellbeing and outcomes of autistic people through precision healthcare approaches.
INCATE, the INCubator for Antibacterial Therapies in Europe, aims to boost the antibiotic pipeline by accelerating the translation of academic innovation into industrial R&D projects to tackle antimicrobial resistance. Founded in 2021 as a partnership of research institutions and pharma companies, INCATE provides advice, community and non-dilutive funding to start-up companies.
Complex innovative designs in clinical trials have the potential to increase efficiency and lower the cost of drug development, improving patient access to therapies. This article highlights designs and approaches based on a meeting linked to an ongoing FDA pilot program in the field.
Pharmacological probes are important tools for exploring disease biology and discovering new therapies. Often molecules of insufficient quality are used instead, leading to spurious and misleading results. The Boehringer Ingelheim open innovation portal opnMe.com addresses this deficiency by sharing extensively validated pharmacological probes with the scientific community.
Targeted protein degraders (TPDs) are emerging classes of therapeutics that have the potential to address difficult drug targets in new ways. In 2021, experts convened to discuss challenges in TPD safety evaluation, and supported supplementing individual efforts to address these challenges with collective development of novel decision frameworks, tools and a shared evidence base.
Translation Together, a transnational consortium of translational research organizations, reflects on successes and challenges in regional COVID-19 pandemic responses and proposes five priorities to improve preparedness for future global public health crises and improve the global approach to translational research and science.
The International Rare Diseases Research Consortium (IRDiRC) is a global collaborative initiative launched in 2011, aimed at tackling rare diseases through research. Here, we summarize IRDiRC’s vision and goals and highlight achievements and prospects after its first decade.
