Sizing the drug-treatable population for rare diseases

The Challenge

A bio-pharmaceutical company, preparing to launch a new treatment, needed to get reliable population estimates for a (very) rare condition.

No estimates of the age group of interest were available in the literature, and after many attempts at sizing the population from an internal team, they had reached a dead end and could see no way forward.

The team needed a defensible solution for use in an orphan drug designation submission, and for dissemination across multiple country and regional teams. The available medical literature afforded no estimates, and an internal team’s attempts at forecasting remained fruitless. Finding themselves at an impasse, the company engaged Clarivate’s epidemiology team.

The Approach

Our expert analysts and data scientists used a combination of medical claims data and traditional epidemiology data in order to build a robust picture of the therapeutic landscape surrounding this rare indication, and modeled the patient flow. Weekly touch points kept the pharma brand team in the loop.

Key analyses:

  • RWD claims and EHR assessment.
  • Patient flow/progression within the disease population.
  • Market size and patient characteristics, including demographics and comorbidities.
  • Model estimates where direct data were not available.