n-of-1 trials and randomised withdrawal studies

Presented at PVRI's Drug Discovery & Development Symposium 2025, by Martin Wilkins, Imperial College London

Summary

The discussion discussed the limitations of relying on single reading averages from traditional clinical trials and introduces n-of-1 studies. The application of remote monitoring through implanted devices provides an opportunity to extend n-of-1 studies to pulmonary hypertension. N-of-1 studies can make an important contribution to early drug development. 

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