February 16, 2015
The Committee of Orphan Medicinal Products (COMP) published an article in the Orphanet Journal of Rare Diseases highlighting medicinal products that were given orphan designation and how the COMP assessed them for medical plausibility. Medical plausibility is judged on the sponsor’s ability to demonstrate the “intention to diagnose, prevent or treat” a serious and rare condition. According to the article, sponsors face several challenges during the Committee’s scientific assessment of the applications for orphan designation.
The article provides examples of where sponsors have successfully justified medical plausibility, to steer future sponsors of medicinal products in the right direction. The authors provide examples of justification based on pre-clinical data with relevant models (animals or cellular) and endpoints. They can also be based on pre-clinical data at the early stages of development, examples of which include applications defended on grounds of preliminary data. For further guidance, the authors provide examples of unsuccessful efforts to justify medical plausibility, such as linking findings to data from other products and to non-relevant models, thus increasing assumption and weakening medical plausibility. The authors emphasised that each application is assessed case by case and even if sponsors have used appropriate models and endpoints, the dossier can still be rejected if the results obtained do not demonstrate benefit.