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Few biomarkers progress from discovery to become validated tools or diagnostics. To bridge this gap, three European biomedical research infrastructures — EATRIS-ERIC (focused on translational medicine), BBMRI-ERIC (focused on biobanking) and ELIXIR (focused on data sharing) — are paving the way to developing and sharing best practices for biomarker validation.

Tumour evolution, which results in the existence of multiple distinct populations of cancer cells within the same tumour and the same patient, is increasingly appreciated to have a key role in drug resistance. In this article, we discuss the implications for drug development, including approaches to reduce the likelihood of the emergence of drug resistance.

Despite a decade of intensive preclinical research, the translation of cancer nanomedicine to the clinic has been slow. Here, we discuss how recent lessons learned from the successes with immuno-oncology therapies could be applied to cancer nanomedicine and how this may help to overcome some of the key technical challenges in this field.

Growing access to diverse 'real-world' data sources is enabling new approaches to close persistent evidence gaps about the optimal use of medical products in real-world practice. Here, we argue that contrary to widespread impressions, existing FDA regulations embody sufficient flexibility to accommodate the emerging tools and methods needed to achieve this goal.

It has been argued that patents impede the development and access of medicines for tropical diseases such as malaria. However, we believe that intellectual property can be a key tool to enable timely progression of drug development projects involving multiple partners and to ensure equitable access to successful products.

Poor adherence to medicines in clinical trials can undermine the value of the trials; for example, by compromising estimates of the benefits and risks of a medicine. In this article, we highlight such consequences and also discuss approaches to tackle this problem.

Many organizations are attempting to harness emerging digital technologies and the surge in the amount of health-related data to drive advances in the development and use of medicines. Focusing on just a few well-proven and readily available strategies could enable such organizations to quickly realize greater value from data and digital technologies.

Despite the vast amount of research on schizophrenia and depression in the past two decades, there have been few innovative drugs to treat these disorders. Precompetitive research collaborations between companies and academic groups can help tackle this innovation deficit, as illustrated by the achievements of the IMI-NEWMEDS consortium.

The growth of precision medicine presents challenges for the regulators of medicines, related to aspects that include the basis of evidence generation, patient involvement in the regulatory process, cost of new medicines and the need for new regulatory models. It also raises questions about the tolerance of risk, especially with early interventions for life-threatening diseases.

There is a growing consensus that drug discovery thrives in an open environment. Here, we describe how the malaria community has embraced four levels of open data — open science, open innovation, open access and open source — to catalyse the development of new medicines, and consider principles that could enable open data approaches to be applied to other disease areas.

A global response to the chronic shortfall in antibiotic innovation is urgently needed to combat antimicrobial resistance. Here, we introduce CARB-X, a new global public–private partnership that will invest more than US$350 million in the next 5 years to accelerate the progression of a diverse portfolio of innovative antibacterial products into clinical trials.

Rare disease drug development could benefit substantially from increased patient engagement and input to enhance understanding of the key aspects of disease impact, ways to measure these impacts and patients' perspectives on the benefit–risk profile of potential therapies.

Drug candidates may fail in clinical trials for many reasons. Biomarker-guided clinical trial design can mitigate the risk of failure and enable more informative clinical experiments regardless of outcomes.

While the pharmaceutical industry overall has faced huge challenges in drug R&D in recent years, some major companies have consistently been much more productive than average. Here, we highlight 'organizational effectiveness' as a key factor underlying this superior performance and explain how it can be enhanced.

Integrating a wide range of biomedical data such as that rapidly emerging from the use of next-generation sequencing is expected to have a key role in identifying and qualifying new biomarkers to support precision medicine. Here, we highlight some of the challenges for biomedical data integration and approaches to address them.

Japanese clinical research and regulatory frameworks have evolved considerably in the past two decades to reduce the delay in the introduction of new drugs in Japan compared with other major markets. However, recently introduced changes related to access to unapproved drugs might have the opposite effect and might not benefit all patients.

Global collaboration in translational science promises to accelerate the discovery, development and dissemination of new medical interventions. Here, we introduce a new international collaboration of translational science organizations and highlight our initial strategy to reduce or remove bottlenecks in translation.

Understanding the basis of variability in the response of patients to the dose of a drug and a willingness to vary the dose regimen as well as the choice of drug should be one of the key pillars of precision medicine.

What could be the benefits of moving the treatment paradigm for serious neuropsychiatric disorders towards earlier intervention, and what is needed to achieve this?

Technological advances coupled with novel collaborative strategies for compound sourcing and management are poised to transform the utility of high-throughput screening.