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Collaborative teamwork results in success for this study involving rare indication in a pediatric / young adult population.

According to research, emerging biopharma companies (EBPs) are responsible for two-thirds of all molecules in the R&D pipeline, and many of the drugs in development are promising candidates for rare diseases. However, only a small percentage of these are resulting in regulatory approval. This is due, in part, to the limited resources, infrastructure and experience of many of these companies in the intricate phases of conducting clinical trials.

A knowledgeable and well-connected CRO can be the difference for these companies in conducting a study that can successfully run the gauntlet of regulatory, operational and scientific obstacles that today’s clinical trials can face. George Clinical was a good fit for this study due to our extensive experience, comprehensive end-to-end services and our ability to develop good working partnerships.

Organizational issues initially resulted in delays, and a temperature-sensitive investigational product required close management. As a study involving a rare indication in a pediatric/young adult population, patient safety was paramount, and recruitment was challenging.