Rare Disease Roundtable: How Rare Is Too Rare?
Part 1: Execs from rare disease-focused companies weighed in on investor sentiment during a roundtable discussion led by Scrip at the J.P. Morgan Healthcare Conference.
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Part 2: High costs are built into the rare disease business model. Participants at a roundtable discussion led by Scrip at the J.P. Morgan Healthcare Conference talked about how much they are feeling the pressure on pricing.
One day in the future, clinical studies may routinely be carried out in the virtual world, with patients mostly staying put at home instead of traveling to trial sites. For now, sponsors are experimenting with new models for drug development and taking it one step at a time.
Orphan drugs and priority reviews achieved historic highs in CDER's 2018 novel approval class, but share of products with breakthrough therapies took a slide.