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Case Study: Quantifying the value of clinical data for an ultra-orphan drug

Problem

A commercial stage pharmaceutical company focused on Orphan Diseases had an asset in Phase III for a very rare CNS disorder. They wanted to determine the preference share value of various clinical data sets in order to drive clinical investment decisions.

Solution

  • Medis Consulting had previously led global qualitative research on this product, so we were able to build on an understanding of the disease and it’s clinical needs.
  • We designed a 20 minute online survey collaboratively with the client that included:
    • Insights into treatment patterns and recognition of the constellation of symptoms
    • Staged reviews of multiple potential profiles
    • Conjoint analysis exercise including 5 attributes across 4 levels
  • We leveraged global recruitment partners to field the survey across three regions: US (n=125), Europe (n=125), and Latin America (n=80)

Outcome

Statistically validated results on treatment experience, stated interest, and intent to use (via profile testing), and derived preference share based on attribute levels (via conjoint).

Full analysis and discussion of strategic context for results, as well as recommendations for clinical development.