A framework: make it useful to guide and improve practice of clinical trial design in smaller populations

Overview of attention for article published in BMC Medicine, November 2016

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Qualified placebo for trials of herbal medicine treatment in rare diseases? A cross-sectional analysis

Article in Orphanet Journal of Rare Diseases (November 2023)

Evidence supporting regulatory-decision making on orphan medicinal products authorisation in Europe: methodological uncertainties

Article in Orphanet Journal of Rare Diseases (November 2018)

A systematic literature review of evidence-based clinical practice for rare diseases: what are the perceived and real barriers for improving the evidence and how can they be overcome?

Article in Trials (November 2017)

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