An N-of-1 Trial in a Progressive Rare Disease: A Case Report on Design, Consent and Interpretation
DOI:
https://doi.org/10.5281/zenodo.22649050Keywords:
An N-of-1 Trial in a Progressive Rare Disease, Translational medicine; Evidence synthesis;, Clinical implementation; Health services researchAbstract
An N-of-1 Trial in a Progressive Rare Disease 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 an n-of-1 trial in a progressive
rare disease, 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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