Objective:
To investigate trends in FDA regulatory approvals and development decisions for neurology and related therapies, analyzing Center for Drug Evaluation and Research (CDER) data.
Background:
There is still high unmet need for novel therapies in debilitating neurological disorders. Recent advances in neuroscience research and a seemingly growing FDA openness regarding surrogate endpoints for accelerated approvals could point to a positive regulatory shift and increased clinical development activity.
Design/Methods:
Analysis of CDER data, active FDA-regulated industry clinical trial programs by therapeutic area, regulated clinical program starts (includes small molecules, monoclonal antibodies, therapeutic proteins), as measured by commercial (industry) IND submissions during 2009-20023 and systematic review of recent FDA approvals.
Results:
- CDER data indicates that neurology novel approvals outnumbered cancer novel approvals in the first half of 2023.
- Active neuroscience trials show continued growth, with an increase of 16% from 2020-2022. This includes an increase in neurodegenerative/ movement/ neuromuscular trials by 24.1% and an increase in psychiatry trials by 17.6% from 2020-2022.
- Although there was a slight pause in 2022 for neuroscience clinical trial starts, this followed a surge in activity during 2021, with neurodegenerative/movement/neuromuscular starts soaring by 75%.
- Across approval and development decisions in difficult conditions such as Alzheimer’s, ALS, Duchenne, and limb-girdle muscular, there is evidence of a new FDA willingness toward accelerated approval and surrogate endpoints.
Conclusions:
Although the field has faced great setbacks in the past, our analysis of CDER data and approval and development decisions across high unmet need disorders, such as Alzheimer’s, Amyotrophic Lateral Sclerosis, Duchenne Muscular Dystrophy, Friedreich’s ataxia and limb-girdle muscular dystrophy signal a changing regulatory and clinical development landscape. The evolving environment now requires more tailored development strategies, patient-focused mindset, and proactive engagement with regulators.