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Rare Disease Roundtable: How Rare Is Too Rare?

Executive Summary

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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Rare Disease Roundtable: Dealing With Pricing Pressure

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.

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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. 

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