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The Case for Master Protocols for Rare Neurological Diseases
Jennifer Vermilion1, Sabrina Paganoni2,3, Scott R Plotkin4
1Department of Neurology, University of Rochester Medical Center, Rochester, New York, USA.
Abstract:
Master protocol trials allow for simultaneous multiple hypothesis testing within a common framework and might be applicable for rare diseases. In May 2025, the Network for Excellence in Neuroscience Clinical Trials convened a multistakeholder conference to discuss master protocol trials in rare neurological disorders. In this paper, we explore how master protocol trial designs may apply to rare neurological disorders, using the neuronal ceroid lipofuscinoses as an example. Through shared protocol elements and trial infrastructure, master protocols may decrease cost and improve efficiency in testing potential therapeutics in rare disease, accelerating the delivery of urgently needed therapies to patients. ANN NEUROL 2026;100:477-486.
Insights
Master protocol trials can streamline testing for rare neurological diseases. This approach may accelerate the development of crucial therapeutics for conditions like neuronal ceroid lipofuscinoses.
Area of Science:
- Neuroscience
- Clinical Trials
- Rare Diseases
Background:
- Master protocol trials enable simultaneous multiple hypothesis testing within a unified framework.
- These designs hold potential applicability for rare diseases, including rare neurological disorders.
- A multistakeholder conference in May 2025 focused on master protocols for rare neurological conditions.
Purpose of the Study:
- To explore the application of master protocol trial designs in rare neurological disorders.
- To examine the potential benefits of master protocols for rare disease research.
- To use neuronal ceroid lipofuscinoses as a specific example for applying master protocols.
Main Methods:
- Review of master protocol trial designs.
- Application of master protocol concepts to rare neurological disorders.
- Case study using neuronal ceroid lipofuscinoses.
Main Results:
- Master protocols can facilitate simultaneous multiple hypothesis testing.
- Shared protocol elements and infrastructure can reduce costs and improve efficiency.
- This approach may accelerate therapeutic delivery for rare diseases.
Conclusions:
- Master protocol trials offer a promising framework for rare neurological disorders.
- Implementing master protocols can enhance efficiency and reduce costs in rare disease drug development.
- Accelerated therapeutic delivery is a key potential outcome for patients with rare neurological conditions.
