Whereas Phase III cancer trials are currently randomized and involve hundreds of patients, some experts say small trials with from 20 to 50 patients make more sense for targeted drugs in ovarian cancer, considering the genetic diversity of this already uncommon disease.

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.

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.