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Growth in the pharmaceutical industry is no longer exclusively driven by large pharmaceutical companies. The switch in the marketplace from a focus on blockbuster drugs to an emphasis on therapies that treat orphan and rare diseases has led to the rise of small and emerging pharma companies founded to advance novel medicines for specialized patient populations. These innovators require the support of contract service providers that recognize their unique requirements.

Recent successes in the development of orphan drugs coupled with productivity challenges in the classic pharma R&D model for indications with a higher prevalence have resulted in a number of major pharmaceutical companies establishing business units focused on rare diseases.