K Chevreul1,2,3,4, K Berg Brigham3,4, M-C Clément3, C Poitou5, M Tauber6. 1. Sorbonne Paris Cité, Univ Paris Diderot, Paris, France. 2. Inserm, ECEVE, Paris, France. 3. URC-Eco, AP-HP, Paris, France. 4. Univ Paris-Est Créteil, Créteil, France. 5. Institute of Cardiometabolism and Nutrition, ICAN, AP-HP, Nutrition Department and French Reference Centre for Prader-Willi Syndrome, University Pierre et Marie Curie-Paris 6, Paris, France. 6. Reference Centre for Prader-Willi Syndrome, Children Hospital, Department of Endocrinology, Obesity, Bone Diseases, Genetics and Gynecology, Toulouse Hospital, Toulouse, France.
Abstract
BACKGROUND: To date, there has been no published comprehensive estimation of costs related to Prader-Willi syndrome (PWS). Our objective was therefore to provide data on the economic burden and health-related quality of life associated with PWS in France in order to raise awareness of the repercussions on individuals suffering from this syndrome and on caregivers as well as on the health and social care systems. METHOD: A retrospective cross-sectional study was carried out on 51 individuals recruited through the French PWS patient association. Data on their demographic characteristics and resource use were obtained from an online questionnaire, and costs were estimated by a bottom-up approach. The EQ-5D-5L health questionnaire was used to measure the health-related quality of life of individuals suffering from PWS and their caregivers. RESULTS: The average annual cost of PWS was estimated at €58 890 per individual, with direct healthcare accounting for €42 299, direct non-healthcare formal costs €13 865 and direct non-healthcare informal costs €8459. The main contributors to PWS costs were hospitalisations and social services. Indirect costs resulting from loss of productivity in the labour market was €32 542 for adults suffering from PWS. Mean EQ-5D utility scores were 0.4 for individuals with PWS and 0.7 for caregivers. CONCLUSIONS: Prader-Willi syndrome represents a major economic burden from a societal perspective and has a significant impact on health-related quality of life both for individuals suffering from PWS and for their caregivers in France. These results underscore the need to develop tailored policies targeted at improving care. Likewise, a larger study collecting a broader range of medical characteristics should be undertaken to achieve more precise estimations.
BACKGROUND: To date, there has been no published comprehensive estimation of costs related to Prader-Willi syndrome (PWS). Our objective was therefore to provide data on the economic burden and health-related quality of life associated with PWS in France in order to raise awareness of the repercussions on individuals suffering from this syndrome and on caregivers as well as on the health and social care systems. METHOD: A retrospective cross-sectional study was carried out on 51 individuals recruited through the French PWSpatient association. Data on their demographic characteristics and resource use were obtained from an online questionnaire, and costs were estimated by a bottom-up approach. The EQ-5D-5L health questionnaire was used to measure the health-related quality of life of individuals suffering from PWS and their caregivers. RESULTS: The average annual cost of PWS was estimated at €58 890 per individual, with direct healthcare accounting for €42 299, direct non-healthcare formal costs €13 865 and direct non-healthcare informal costs €8459. The main contributors to PWS costs were hospitalisations and social services. Indirect costs resulting from loss of productivity in the labour market was €32 542 for adults suffering from PWS. Mean EQ-5D utility scores were 0.4 for individuals with PWS and 0.7 for caregivers. CONCLUSIONS:Prader-Willi syndrome represents a major economic burden from a societal perspective and has a significant impact on health-related quality of life both for individuals suffering from PWS and for their caregivers in France. These results underscore the need to develop tailored policies targeted at improving care. Likewise, a larger study collecting a broader range of medical characteristics should be undertaken to achieve more precise estimations.
Authors: A M Russell; J L O'Dwyer; L D Bryant; A O House; J C Birtwistle; S Meer; A Wright-Hughes; R E A Walwyn; E Graham; A J Farrin; C T Hulme Journal: Pilot Feasibility Stud Date: 2018-10-29
Authors: Karlijn Pellikaan; Anna G W Rosenberg; Anja A Kattentidt-Mouravieva; Rogier Kersseboom; Anja G Bos-Roubos; José M C Veen-Roelofs; Nina van Wieringen; Franciska M E Hoekstra; Sjoerd A A van den Berg; Aart Jan van der Lely; Laura C G de Graaff Journal: J Clin Endocrinol Metab Date: 2020-12-01 Impact factor: 5.958