Comment

A specialized platform for innovative research exploration — ASPIRE — in preclinical drug discovery could help study unexplored biologically active chemical space through integrating automated synthetic chemistry, high-throughput biology and artificial intelligence technologies.

The definition and acceptability of an orphan condition is pivotal for the assessment of European orphan medicinal product designation applications, and consequently the eligibility for incentives. Here, based on the experiences of the Committee for Orphan Medicinal Products, we discuss how to define orphan conditions in the context of the European regulatory framework.

'Adaptive' approaches to bringing drugs to market have been widely discussed in recent years. Here, we describe the evolution of the adaptive approach and of concepts developed by ADAPT SMART, a multi-stakeholder consortium funded by the European Innovative Medicines Initiative.

Clinical trials for Alzheimer disease drugs have an exceptionally high failure rate, discouraging investment in the field despite the unmet medical need. Drug developers need to more effectively harness existing and emerging data and digital technologies to improve the likelihood of success.

The NIH has launched the Somatic Cell Genome Editing (SCGE) programme to accelerate the development of safer and more effective genome-editing therapeutics.

Drug repurposing has been proposed as a strategy to develop new therapies that has fewer risks, lower costs and shorter timelines than developing completely new drugs. However, the potential of this strategy has not been as widely realized as hoped, in part owing to legal and regulatory barriers. Here, we highlight these barriers and consider how they could be overcome.

Since its launch a decade ago, the European Innovative Medicines Initiative (IMI), a leading public–private partnership in life sciences, has catalysed scientific breakthroughs and the development of strategies to address complex challenges in various biomedical fields. Here, we discuss the progress of IMI projects related to vaccine research and development.

The term 'translation' has emerged as a dominant concept in biomedical science over the last decade, but confusion around what the term means, and how it differs from translational research and translational science, is common. This article aims to help address this issue by clarifying the distinctions.

Information Exchange and Data Transformation (INFORMED), a multidisciplinary initiative anchored in the FDA Oncology Center of Excellence, is a decentralized science and technology incubator designed to harness the power of big data and advanced analytics to improve disease outcomes.

Recent progress in the discovery, development and evaluation of new drugs and combination regimens for drug-resistant tuberculosis through greater collaboration between industry, donors and academia provides renewed hope for overcoming the challenges in tuberculosis treatment.

Much biomedical research continues to focus on a small proportion of the human genome that has already been studied intensively. The Illuminating the Druggable Genome programme, initiated as a pilot project by the US National Institutes of Health Common Fund in 2014, is now being implemented to accelerate the investigation of subsets of understudied proteins that have potential therapeutic relevance.

The uptake of a new medicine represents a balance between benefit–risk assessment and value considerations. In the case of products approved via accelerated pathways, the increased uncertainty adds to the challenge. Here, we suggest solutions so that regulators, companies, payers and patients can align around management of the uncertainties and expectations.

Numerous challenges arise when developing targeted therapies for diseases comprising low-frequency molecular subtypes. In this article, we describe a pragmatic, science-based regulatory policy for the development and approval of targeted therapies in such cases.

The development of anticancer therapies is evolving with the advent of novel targeted drugs. In this Comment, I summarize some of the key changes and discuss the various choices for the optimal development of a new generation of cancer therapeutics.

The past 5 years have seen an unprecedented rate of discovery of genes that cause rare diseases and with it a commensurate increase in the number of diagnosable but nevertheless untreatable disorders. Here, we discuss the increasing opportunity for diagnosis and therapy of rare diseases and how to tackle the associated challenges.

The availability of targeted anticancer drugs and the relative affordability of genomic analyses has led to a growing expectation among patients with cancer that they can receive personalized treatment based on the genomic signature of their tumour. Here, we discuss some of the challenges and steps needed to bring such approaches into routine practice.

The sharing of legacy preclinical safety data among pharmaceutical companies and its integration with other information sources offers unprecedented opportunities to improve the early assessment of drug safety. Here, we discuss the experience of the eTOX project, which was established through the Innovative Medicines Initiative to explore this possibility.

Scientific advances, in combination with government incentives and commercial opportunity, have fuelled strong investment in orphan drugs, resulting in many innovative therapies. Here, we discuss the approach of the FDA to a range of issues that remain crucial to maintaining this momentum, such as the use of the totality of evidence in evaluating orphan drugs.

Commercializing innovations in academic environments is notoriously challenging. Here, we describe the progress of the NIH Centers for Accelerated Innovations program — initiated in 2013 to address these challenges — which we believe could help set a new standard for the early-stage commercialization of biomedical innovations in academic environments.

New opportunities to develop innovative — and often complex — products that combine drugs, devices and/or biological components are rapidly emerging, raising questions about how such products should be regulated. Here, we discuss the ongoing efforts of the FDA to develop a modern, transparent, flexible and consistent science-based regulatory approach for combination products.