Highlights the need for and discusses alternative clinical trial designs to overcome the constraints of traditional RCTs in rare diseases like pulmonary hypertension.
Alternative trial designs merit exploration in pulmonary hypertension; leaves open optimal implementation for rare-disease research.
In pulmonary hypertension, as in many other diseases, there is a need for a smarter approach to evaluating new treatments. The traditional randomized controlled trial has served medical science well, but constrains the development of treatments for rare diseases. A workshop was established to consider alternative clinical trial designs in pulmonary hypertension and here discusses their merits, limitations and challenges to implementation of novel approaches.
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Chow et al. (2013) studied this question.
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