R01 clinical trials for orphan drugs to address rare-disease unmet needs and support new labels
This funding opportunity supports clinical trials that evaluate the efficacy and/or safety of orphan products to address unmet needs in rare diseases. Trials may be conducted to support a new indication or changes in product labeling, with an emphasis on collaborative and efficient approaches that can help increase approved rare-disease treatments. R01 (clinical trials required) suggests appli…
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