Work on medicines that can change life

ORPHAN MEDICINES

The right to health must be guaranteed to all, even to patients suffering from rare diseases: nobody must be excluded from the progress of science. Doing research and marketing orphan medicines, however, is not convenient for pharmaceutical companies: in view of considerable investments in research and development, revenues are very low. Thus, the authorities have adopted a lot of incentives for supporting the transformation of scientific results into medicines and therapies for all patients of the world.

At the same time, rare disease patients and their families need to be constantly updated on the scientific progress related to their diseases. The Gianni Benzi Foundation works on this field by creating EuOrphan, an online database collecting regulatory and scientific information on drugs for rare diseases: active substances designated or approved as ‘orphan’, designation date, orphan indication, the holder of the designation and the Market Authorisation, ATC (Anatomical Therapeutic Chemical Classification System), trade name, therapeutic indication and approved ages, date of approval, genetic disease and if it affects children.

Thanks to the information available on EuOrphan, it is also possible to access the general statistics, the comparative analysis between Europe and the United States on designations and approvals, and the analysis of the failures found in the approval of designated orphan medicines.

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