Louis S Matza, Katie D Stewart, Marie Fournier, Donna Rowen, Robin Lachmann, Maurizio Scarpa, Eugen Mengel, Travis Obermeyer, Evren Ayik, Fernando Laredo, Ruth Pulikottil-Jacob
INTRODUCTION: Acid sphingomyelinase deficiency (ASMD) type B is a rare genetic disorder leading to enlargement of the spleen and liver, pulmonary dysfunction, and other symptoms. Cost-utility analyses are often conducted to quantify the value of new treatments, and these analyses require health state utilities. Therefore, the purpose of this study was to estimate utilities associated with varying levels of severity of adult and pediatric ASMD type B. METHODS: Seven adult and seven child health state vignettes describing ASMD were developed based on published literature, clinical trial results, and interviews with clinicians, patients with ASMD, and parents of children with ASMD...
February 27, 2024: European Journal of Health Economics: HEPAC: Health Economics in Prevention and Care