Table 2

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

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

Kenny et al. Orphanet Journal of Rare Diseases 2008 3:23   doi:10.1186/1750-1172-3-23