Unfortunately, many RDs go undiagnosed (3). Pharma companies must develop strong relationships with all key Rare Disease Patient Organizations (RDPO) and research hospitals (e.g., children’s hospitals, key academic research hospitals, etc.), which puts pressure on external medical affairs teams to foster relationships with key opinion leaders (KOLs) and RD experts.Īnother key element is the diagnosis and treatment of RD. While finding appropriate patients is always a challenge for conducting clinical trials, this is especially acute for RDs, given the small populations involved. Starting from the clinical trial stage of the product/project lifecycle, the first major challenge is patient recruitment. The successful commercialization of ODs requires a range of strategic and tactical elements to be implemented by pharma companies. Finding patients and dealing with diagnosis
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