Improving and accelerating drug development for nervous system disorders

Abstract

Advances in the neurosciences have placed the field in the position where it is poised to significantly reduce the burden of nervous system disorders. However, drug discovery, development, and translation for nervous system disorders still pose many unique challenges. The key scientific challenges can be summarized as follows: mechanisms of disease, target identification and validation, predictive models, biomarkers for patient stratification and as endpoints for clinical trials, clear regulatory pathways, reliability and reproducibility of published data, and data sharing and collaboration. To accelerate nervous system drug development, the Institute of Medicine's Forum on Neuroscience and Nervous System Disorders has hosted a series of public workshops that brought together representatives of industry, government (including both research funding and regulatory agencies), academia, and patient groups to discuss these challenges and offer potential strategies to improve the translational neuroscience.

Figure 1. Improving and Accelerating Therapeutic Development for Nervous System Disorders

Although there is a high burden associated with nervous system disorders, development of new therapeutics remains stagnant. Over the last decade, fewer new drugs for nervous system disorders have garnered approval in comparision to other therapeutic areas. Current data suggest that drug development, from the start of a discovery program to regulatory approval, can take an average of 12 to 15 years. This familiar statistic prompts an equally familiar question: Can the therapeutic development timeline be improved and accelerated by addressing challenges and developing opportunities? The challenges and opportunities presented in this graphic are not related and are presented in no certain order. The opportunities listed should not be interpreted as solutions to the challenges identified. This graphic illustrates common challenges and opportunities suggested by individual participants in workshops hosted by the IOM Forum on Neuroscience and Nervous System Disorders (Accelerating Therapeutic Development for Nervous System Disorders Towards First-in-Human Trials [April 8–9, 2013] and improving Translation of Animal Models for Nervous System Disorders (March 28–29, 2012]). Statements, recommendations, and opinions expressed are those of the individual participants and are not necessarily endorsed or verified by the IOM, and should not be construed as reflecting any group consensus. IND, investigational new drug; IP, intellectual property. Adapted and reprinted with permission from the Institute of Medicine.