Endpoint Selection in Rare Disease Trials: Composite Measures and the Loss of Interpretability
DOI:
https://doi.org/10.5281/zenodo.22649538Keywords:
Endpoint Selection in Rare Disease Trials;, Translational medicine; Evidence synthesis;, Clinical implementation; Health services researchAbstract
Endpoint Selection in Rare Disease Trials is an area of active development in translational medicine, with evidence that has
accumulated unevenly across its component questions. We review the current evidence base for endpoint selection in rare disease
trials, spanning analytical, clinical, implementation, governance, and economic dimensions, focusing on where practice has
converged and where it continues to vary, and set out the practical considerations relevant to a service considering its adoption.
We conclude that selective, context-specific use is best supported by the evidence currently available, rather than uniform
adoption or avoidance.
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