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Predicting patients with an ultra-rare disease for treatment launch planning

A pharmaceutical company needed to understand the total addressable market for an ultra-rare disease to help inform new treatment launch planning. Using the analytic capabilities of Passport Planning and Performance, the company identified 4,400 patients as highly likely to have the ultra-rare disease. This information was critical in helping the company create a successful go-to-market strategy.

Key result


patients predicted to have the disease enabled the company to confidently proceed with the launch planning

The challenge

Discovering the right patients to guide launch planning due to no identifiable ICD-10 code

A pharmaceutical company planned to launch a first-in-class treatment for an ultra-rare disease that recently became medically identifiable as a unique illness. However, because there was no ICD-10 code available it was challenging to estimate the eligible patient universe, identify care sites and physicians to call on at launch, and develop a forward-looking prediction on the emergence of new patients.

The solution

Leveraging the predictive analytics capabilities of Passport Planning & Performance to identify patients with the ultra-rare disease

Using technology within Passport Planning & Performance and medical expertise to analyze claims data and EMR clinical notes revealed speed to insights in identifying patients. The results helped the company predict the annual universe of eligible patients with varying degrees of disease (i.e. high likelihood, likely, unlikely) and help with prelaunch planning.

The impact

By assigning the predictions of the 4,400 patients the company confidently moved forward toward the launch of the new treatment

The ability to predict patients with the ultra-rare disease allowed the company to identify approximately 4,400 patients as highly likely to have the disease. This company used the intelligence to create accurate forecasts and identify physicians to target with product information and disease education at launch.