NPP Case Studies2019-07-30T09:57:58+01:00

Helping patients access an EU-approved product not registered in the US


Patients in the US suffering from a particularly rare and debilitating metabolic disease have no approved treatment options available to them. To help support these patients, a US physician had, at one time, managed a treatment IND which allowed access to a non-FDA approved product. However, this physician could not maintain the program and it was terminated. Patients were left with nothing and were desperate to find a way of […]

Supplying approved treatments in new markets


A leading pharmaceutical company wanted to supply its portfolio of treatments for a rare congenital disease into new markets on an unlicensed basis. This required the company to navigate the varied regulatory landscapes across different markets and understand the range of possible distribution channels for making product available, including unlicensed commercial sales, compassionate use, and Named Patient Programs. The company wanted to find a partner with the right knowledge and […]


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