Comment in 2017

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.

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.