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Published in 2019 at "Journal of Biopharmaceutical Statistics"
DOI: 10.1080/10543406.2019.1657441
Abstract: ABSTRACT One of the most challenges for rare disease clinical trials is probably the availability of a small patient population. It is then a great concern on how to conduct clinical trials with a small…
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Keywords:
statistical considerations;
rare diseases;
rare disease;
drug ... See more keywords