The impact of clinical trials in neurology

Overview Fueled by the aging global population and economic growth of developing countries, the demand for new, safe, and effective therapeutics for neurological conditions in the US and globally will increase dramatically over the next generation. Scientific discovery and clinical investigation are critical for developing and evaluating new treatments and can have substantial public health benefits. However, several challenges confront the development of new therapies. Some of these are generic (e.g., rising costs of drug development, misaligned incentives, recruitment of research participants) and some are specific to neurological conditions (e.g., slow course of neurodegenerative conditions, limited availability of biomarkers). Along with these challenges are potential advances that could accelerate development, including scientific progress in the platforms that support discovery and development (e.g., in genetics and biotechnology) and in the more active participation of patients and advocacy groups that can help fuel the development of new treatments, even for the rarest of disorders. Beyond drugs for neurological conditions, clinical trials will examine other promising therapeutic interventions, including devices and procedures. Meeting the great need for effective therapeutics will not only require continued scientific discovery but also modifications in commercial incentives, improvements in the conduct of clinical trials, and advocacy and participation by the growing number of individuals affected by neurological conditions.