Transforming Drug Development for Neurological Disorders: Proceedings from a Multidisease Area Workshop

Abstract

Neurological disorders represent some of the most challenging therapeutic areas for successful drug approvals. The escalating global burden of death and disability for such diseases represents a significant worldwide public health challenge, and the rate of failure of new therapies for chronic progressive disorders of the nervous system is higher relative to other non-neurological conditions. However, progress is emerging rapidly in advancing the drug development landscape in both rare and common neurodegenerative diseases. In October 2022, the Critical Path Institute (C-Path) and the US Food and Drug Administration (FDA) organized a Neuroscience Annual Workshop convening representatives from the drug development industry, academia, the patient community, government agencies, and regulatory agencies regarding the future development of tools and therapies for neurological disorders. This workshop focused on five chronic progressive diseases: Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, Duchenne muscular dystrophy, and inherited ataxias. This special conference report reviews the key points discussed during the three-day dynamic workshop, including shared learnings, and recommendations that promise to catalyze future advancement of novel therapies and drug development tools.

Artificial Intelligence and Disease Modeling: Focus on Neurological Disorders

“…For neurodegenerative diseases, modeling techniques are recognized as critical for therapeutic innovation given their potential for integrating multiscale data such as fluid and imaging biological markers, sensors and wearable devices data, and of course, clinical outcomes 2 . Among modeling techniques, disease modeling (DM) has become an instrumental tool because it can offer a quantitative framework to characterize and better understand the progression of diseases, notwithstanding that validated markers are rare and clinical measures are complex and multidimensional.…”

Advancing precision medicine therapeutics for Parkinson’s utilizing a shared quantitative systems pharmacology model and framework

“…The above path has been successfully advanced at Critical Path Institute across many disorders including Alzheimer's disease, Huntington's disease, Duchenne Muscular Dystrophy (reviewed by Stephenson et al (2023), and Type 1 Diabetes (Podichetty et al, 2022). There has been much progress by the Parkinson community to deliver on shared clinical and biomarker databases.…”

Parkinson's disease: Still waiting for a cure

“…Subsequently I have been invited to participate in numerous drug development workshops including those sponsored by Critical Path, the FDA and industry to provide the patient lived experience. 6 I even pivoted my own career to lead patient engagement activities with a previous employer by incorporating patient perspectives in developing their select programs. I have personally witnessed Patient Focused Drug Development (PFDD) evolve from being a token afterthought implemented at late stages of clinical development to participating on panels where the patient voice is central in aiding translational research and early development decisions.…”