The challenges of orphan drug development require different clinical trial approaches, such as crossover studies and randomized withdrawal studies, officials of the FDA's Center for Drug Evaluation and Research said. They also stressed the importance of natural history studies and other preclinical preparation.
Sign up for BIO SmartBrief
Biotechnology industry news
Get the intelligence you need: news and information that is changing your industry today, hand-curated by our professional editors from thousands of sources and delivered straight to your inbox.