Improving Clinical Trials For Rare Diseases Of The Central Nervous System

The business of discovering and developing new drugs has historically focused on finding therapies which will benefit the largest patient population. But as our understanding of the genetic basis of disease has matured in recent years, the focus has shifted to using a precision medicine approach to treat patients using personalized therapies designed to work best in the unique environment of an individual.

While oncology is the quintessential example of an area that has – and continues to be – highly influenced by precision medicine, drug development in the fields of neurological and psychiatric disease is increasingly trending the same way. Traditionally hard-to-treat neurological diseases – including Alzheimer’s and Parkinson’s – which were once considered to be common, are being reclassified as a collection of rare diseases based on genetic markers.

This article shares new strategies for enrolling patients and outlines the challenge of measuring the efficacy of the experimental treatment and determining whether it’s producing the desired effect.