Engagements

Understanding the natural history of an inherited rare disease

A global rare disease specialty pharmaceutical company sought to evaluate the attractiveness of different physician specialty targets.

CRA Approach

  • Conducted an extensive literature review on the rare disease to understand its natural history and collect reported prevalence of its typical clinical features
  • Conducted primary research with KOLs and specialty physicians to validate and detail understanding of Natural History and referral patterns
  • Created a prevalence based model systematically evaluating attractiveness of lead symptomatologies (single clinical features or combinations thereof) along 8-dimensional attractiveness grid

Client impact

  • Identified primary and secondary specialty targets taking into account main competitor’s actions
  • Created transparency on most current scientific literature and medical details of all lead symptomatologies and identified knowledge gaps
  • Quantified patient opportunity by lead symptomatology and specialty based on this study and studies conducted in parallel
  • Derived hypotheses on how to approach physicians to support patient finding
Related Capabilities