Answers are grounded in this paper and cite their sources.
This exploration shows how master protocols improve therapeutic testing efficiency in rare neurological disorders, suggesting better future outcomes.
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.
No takes yet. Share an insight, caveat, or question.
Vermilion et al. (2026) studied this question.
Synapse has enriched 10 related papers. Consider them for comparative context: