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Table 2 Clinical outcomes used as indicators to assess the performance of medical treatment and mental health

From: Monitoring clinical quality in rare disease services – experience in England

Condition or Service Outcome measure
Alstrom syndrome Appropriate measure not clear, there are biochemical markers of quality of care.
Chorioncarcinoma Deaths
Epidermoloysis bullosa Appropriate measure not clear, there are biochemical markers of quality of care.
Intestinal failure Annual mortality
Lysosomal storage disorders Quality of life scales currently in development
Mental health service for deaf adolescents Global function score
Obsessive compulsive disorder Yale-Brown Obsessive Compulsive Score
Paediatric liver disease Heterogeneous category: five marker conditions are under consideration; Hepatoblastoma, Biliary atresia, Tyrosinaemia, Autoimmune sclerosing cholangitis, Bile acid disease. For all of these survival is the outcome indicator.
Paediatric pulmonary hypertension Survival
Persistent Hyperinsulinemic hypoglycaemia of infancy (PHHI) Survival and disability
Secure forensic mental health service: for young people Health of the Nation Outcome Scales for Children and Adolescents (HONOSCA) and; Health of the Nation Outcome Scales (HONOS) Secure plus global function