Strategies for postmarketing surveillance of drugs for rare diseases.


Rare diseases are an important part of the public health, affecting 6-8% of the population, and drugs intended for rare diseases comprise the fastest growing subcategory of new drug approvals in the United States. However, clinical study of therapeutics in these populations is limited by the low prevalence of these diseases, and the natural history or… (More)
DOI: 10.1038/clpt.2013.218


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