Neurological diseases impact more than 3 billion people worldwide, and this number is projected to double over the next two decades. Despite their widespread prevalence, these conditions remain among the most challenging to diagnose and treat. Approximately 90 percent of neurology clinical trials do not lead to regulatory approval for new drug candidates.
The complexity of the brain and its disorders makes it difficult to identify clear biological targets and measure therapeutic progress. Symptoms such as developmental delays or behavioral problems can be found across different neurological diseases, making quantification a challenge.
In recent years, there has been notable progress in how regulators approach the evaluation of new neuroscience therapies. The U.S. Food and Drug Administration (FDA) has granted accelerated approvals for treatments based on surrogate endpoints such as amyloid PET reduction in Alzheimer’s disease, plasma neurofilament light chain decreases in SOD1-ALS, and tissue protein restoration in Duchenne muscular dystrophy. This reflects a growing willingness to use biomarkers that are likely to predict clinical benefit.
Other advancements include genetic testing, multi-omic technologies, and improved plasma and cerebrospinal fluid (CSF) biomarkers. These tools help researchers select patient groups that are more likely to respond to specific treatments. For example, measuring alpha-synuclein levels in blood aids diagnosis and differentiation of Parkinson’s disease subtypes. New technologies like quantitative EEGs and advanced imaging methods are also raising standards for monitoring brain activity.
However, experts emphasize that using biomarkers alone is not enough. It remains necessary to prove that changes in these measures correspond with outcomes meaningful to patients and caregivers. As stated: “The field must show that changes in these markers translate into outcomes that matter to patients and caregivers.” While certain biomarkers such as phosphorylated Tau proteins show promise for Alzheimer’s disease, their correlation with cognitive impairment is still under study.
Rigorous natural history modeling, careful selection of trial endpoints, and incorporation of patient-centered measures are recommended strategies for future studies. The aim is to link biological effects directly with improvements in daily life for patients.
As described: “By designing trials that capture patient-centered outcomes, researchers can identify therapies that provide true clinical value, even if they do not completely reverse disease.”
To further improve success rates in neuroscience research, it is suggested that insights from previous studies should inform ongoing work across similar mechanisms or related diseases.
With increasing understanding of brain biology and better assessment tools available—such as digital endpoints—opportunities are expanding for designing trials around outcomes relevant to patients’ lives.
“Neuroscience has reached an inflection point that enables sponsors to pursue and demonstrate patient-centered differentiation,” one expert noted.
Industry leaders are encouraged to recognize when clinical data indicate a therapy’s potential benefit so drug development can move beyond incremental gains toward significant improvements for those affected by neurological diseases.
