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The drug development process for rare diseases is known to present both opportunities and challenges. Because of the small patient pool available in these indications, there are challenges in designing and conducting clinical trials to generate the appropriate evidence, analyse and interpret the data, and ultimately meet the regulators’ expectations. The Clinical Trials Research Needs Recommendations, put together by the the Rare Disease Moonshot partners, aim to inform funders, researchers, patients, industry and other stakeholders on where public-private partnerships (PPPs) would bring most value in optimising clinical trials in small and very small populations and points to consider when setting up such collaborations.
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