C-Path Consortium Aims To Speed, De-Risk Duchenne R&D
This article was originally published in Scrip
While some firms in recent years have found great success with rare disease medicines – with orphan drugs turning out to be the new blockbusters in return on investment – it's nonetheless, generally been a rough road for those products getting to the marketplace, given the difficulty in enrolling patients and proving efficacy in small populations.
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Repeated trial failures in this rare disease space have given birth to new alliances – one backed by Critical Path and the other by the CureDuchenne advocacy group – that aim to model disease progression much more accurately through data sharing and rigorous analysis.
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