When in-house experts’ forecasts for the market potential of a treatment for an orphan disease conflicted, the management team at a large, multinational pharmaceutical company turned to Ozmosi to resolve the differences.

Addressing the central questions required a full literature review, including analysis of more than 30 individual studies released between the 1990s and 2013, as well as physician surveys, internal studies, advisory board results, and other sources. We also conducted in-depth interviews with the company’s two in-house epidemiology experts working on the project. Projections were complicated by the fact that the disease, which affects fewer than 10 people per million, had no associated diagnostic codes, or even much medical consensus around diagnostic criteria.

Ozmosi took a nonconventional approach to estimating the incidence and prevalence of the disease, combining data from insurance claims with information collected from specialty medical centers that worked with individuals who had the disease. As a result, we were able to deliver estimates that the entire team—including the individuals responsible for the earlier conflicting forecasts—could agree with.

Ozmosi’s involvement in the project extended well beyond providing a simple epidemiological analysis. We were asked to summarize and present all known information in a manner that would allow the client’s internal experts to align their forecasts. We provided detailed analysis of data on patient population estimates by country and delivered a report that supported country-level forecasts across the company’s global market. As a result, experts across organizational functions and geographic territories were able to agree on an estimate to drive strategy for the drug.

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