Business Development

Evaluating an Orphan Drug In-Licensing Opportunity

Sep 1, 2022

What our client needed

Our client was evaluating a business development opportunity and needed to understand the market potential for a novel treatment for a pediatric disease. The asset had the potential to be a breakthrough therapy for patients who did not respond to, or had developed resistance to, existing treatments.​

Cofactor solution

Cofactor prepared a market landscape assessment, patient segment sizing and forecast model to support valuation of the asset. The assessment included an in-depth review of the clinical literature to understand how the market was defined, how the disease was diagnosed, and which patient types that would be appropriate for the new treatment. ​ Our team then conducted primary research with a small sample of KOLs and practicing physicians to develop deeper insights into the real-world considerations in clinical practice. ​Last, we built a patient-flow model based on data from clinical literature to reflect the true size of the targetable patient population.

Project outcome

The analysis identified two specific patient populations that were ideal targets for treatment initiation. The client used our findings to refine their thinking about the nature of the commercial opportunity, the requirements for the clinical program, and ultimately the asset valuation.​ The insights we generated were key to understanding the potential for orphan status and pricing, as well as determining how to structure a potential deal with the asset owner.​ The client successfully acquired the asset.

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