So-called “orphan diseases” are rare conditions that affect fewer than 200,000 people per year in the U.S. At a time when pharmaceutical firms are contending with an increasingly challenging industry environment, the orphan sector offers the alluring prospect of strong growth and attractive profit margins—along with the opportunity to improve the lives of patients with often debilitating conditions. However, there are several trends that make being a player in orphan drugs a more challenging proposition than in the past.

Commercial success is not guaranteed and the regulatory and access bars are rising. Companies that aspire to become leaders or drive growth in the orphan space must recognize the environmental challenges as well as key operational and organizational differences to the traditional biopharma model. In this Executive Insights article, L.E.K. looks at the changing dynamics of the orphan market and identifies winning strategies designed to capture value within this sector.

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