Essais cliniques/Clinical trials
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Small Population Clinical Trials Task Force Workshop Report and Recommendations www.irdirc.org wp-content uploads 2016 11 SPCT_Report.pdf
A framework: make it useful to guide and improve practice of clinical trial design in smaller populations
Alternative designs for clinical trials in rare diseases
Does the low prevalence affect the sample size of interventional clinical trials of rare diseases? An analysis of data from the aggregate analysis of clinicaltrials.