Volume 13 Issue 11, November 2014

Improving the science of drug development and regulation is important in fulfilling the public health mission of the US Food and Drug Administration (FDA). A decade on from the launch of the Critical Path Initiative, the FDA's Center for Drug Evaluation and Research (CDER) is now participating in more than 20 science-driven consortia to achieve this goal.

The Critical Path Institute brings scientists from regulatory agencies, industry and academia together to improve drug development and regulatory processes.

TransCelerate BioPharma was formed in 2012 as a non-profit organization with a mission to collaborate across the biopharmaceutical research and development community to identify, prioritize, design and facilitate the implementation of solutions to drive efficient, effective and high-quality delivery of new medicines.

Without a step change in the productivity of pharmaceutical research and development, it will be difficult to tackle the public health challenges facing societies worldwide. Public–private partnerships could play a key role in achieving this step change, but they need to be well designed and led.

The Biomarkers Consortium is a public–private biomedical research partnership that seeks to develop and qualify biomarkers across a broad range of diseases in order to accelerate the development of new medicines and improve patient care.

The Predictive Safety Testing Consortium and the Coalition Against Major Diseases, both launched by the Critical Path Institute, provide valuable examples of the outcomes and lessons learned by different types of consortia working on new drug development tools.

The International Serious Adverse Events Consortium is generating novel insights into the genetics and biology of drug-induced serious adverse events, and thereby improving pharmaceutical product development and decision-making.

The Clinical Trials Transformation Initiative (CTTI) identifies and promotes practices to increase the quality and efficiency of clinical trials through projects that generate empirical data on how trials are currently conducted, leading to recommendations for improvement.

Amgen's blinatumomab is setting the stage for a bispecific revival, enabled by new formats that may solve the field's long-standing problems.

An approval for a first dengue vaccine seems in sight, even if the efficacy data are not as strong as was once hoped.

Keith Blundy, Chief Executive Officer of Cancer Research Technology, discusses new developments at the non-profit Cancer Research UK.

This analysis examines new classes of drugs that are in development for dyslipidaemia — in particular PCSK9 inhibitors and CETP inhibitors — and discusses their likely impact on the dyslipidaemia market, which is currently dominated by statins.

Multifunctional and stimulus-sensitive nanoparticles can overcome issues associated with traditional drug delivery by, for example, improving targeting and bioavailability. Here, Torchilin highlights recent developments in these types of nanocarriers and how they can also include imaging contrast moieties to track their real-time biodistribution and accumulation at the target site.

Although previous attempts to therapeutically target RAS proteins — which are frequently mutationally activated in cancer — have been unsuccessful, recent advances in our understanding of these signalling proteins have led to a renewed interest in RAS-targeted therapies. Der and colleagues discuss progress with a range of established and emerging strategies to interfere with RAS function, and highlight the most promising avenues for future anticancer drug discovery efforts.

Hypoxia-inducible factors (HIFs) have important roles in ischaemic and inflammatory diseases and strategies aimed at therapeutically modulating hypoxia signalling pathways are gaining considerable attention. Here, Eltzschig and colleagues focus on a set of oxygen-sensing prolyl hydroxylases — which are responsible for marking HIFs for proteasomal degradation — and assess their emerging potential as therapeutic targets.

Launched a decade ago, the US Food and Drug Administration's Critical Path Initiative has helped catalyse the formation of many consortia focused on drug development challenges. In this Focus, representatives from some of the major stakeholder groups and consortia describe their roles, summarize progress so far and provide their perspectives on the opportunities, challenges and lessons learned for biomedical consortia in general.