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Table 1 Glossary used in this paper

From: Use of biomarkers in the context of orphan medicines designation in the European Union

Biomarker: A characteristic that is objectively measured and evaluated as an indicator of normal biologic processes, pathogenic processes, or pharmacologic responses to a therapeutic intervention [5].
Surrogate endpoints: A biomarker intended to substitute for a clinical endpoint. A surrogate endpoint is expected to predict clinical benefit (or harm, or lack of benefit) based on epidemiologic, therapeutic, pathophysiologic or other scientific evidence [5].
Condition: Any deviation(s) from the normal structure or function of the body, as manifested by a characteristic set of signs and symptoms (typically recognized distinct disease or a syndrome) (guideline ENTR/6283/00 Rev3) [2].
Orphan Condition: the condition as defined above that meets the criteria defined in Art.3 of Regulation (EC) No 141/2000 (guideline ENTR/6283/00 Rev3) [2].
Personalised Medicine: A medicine targeted to individual patients, based on their genetic characteristics [6].