EMEA: COMP Recommendation on elements required to support the medical plausibility and the assumption of significant benefit for an orphan designation

EMEA:
Committee for Orhphan Medicinal Products Recommendation on elements required to support the medical plausibility and the assumption of significant benefit for an orphan designation (draft). This Discussion Paper has two aims. Firstly, to outline the level of evidence normally required to support the medical plausibility of using the product in the applied condition, and secondly, the level of evidence required to support the assumption of significant benefit.