Comment in 2018

Investment in drug development for neurodevelopmental disorders has suffered from recent failures in clinical trials that were based on promising preclinical findings. Here, we discuss development and validation of translational biomarkers of neurodevelopmental disorders that can enable more informative clinical experiments and translational success in these diseases.

The development of oncology drugs traditionally begins by studying them in heavily pretreated patients, and then working ‘upstream’ to populations with earlier-stage disease. The recent FDA approval of an androgen receptor antagonist first in prostate cancer patients without demonstrable metastatic disease but at high-risk for metastasis, based on a novel metastasis-free survival end point developed by the FDA, could provide a template for a paradigm shift.

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.