THE UTILITY OF DIFFERENT APPROACHS TO DEVELOPING HEALTH UTILITIES DATA IN CHILDHOOD RARE DISEASES – A CASE STUDY IN SPINAL MUSCULAR ATROPHY (SMA)
Author(s)
Thompson R1, Vaidya S1, Teynor M2
1Biogen, Zug, Switzerland, 2Biogen, Cambridge, MA, USA
Presentation Documents
OBJECTIVES: SMA is a rare, hereditary, autosomal recessive neuromuscular disorder that in its most severe forms impacts infants and young children. Capturing health utilities in infants and young children is often challenging and unadvisable in clinical trial settings since most Qol or utilities instruments are not designed for such age groups. However, the development of cost effectiveness models, required in many countries for reimbursement, necessitates generating health utilities. The objective of this work is to develop health utilities for infants and young children with SMA utilizing different methodologies. METHODS: Three methodologies were undertaken to develop health utilities for input into CE models for nusinersen, the first approved therapy for treatment of SMA. A cross-sectional study of individuals with SMA in select European countries collected parent-proxy assessed Qol using the EQ-5D-3L. A case vignette study assessed physician rated Qol using EQ-5D-5L and the PedsQol for defined motor function health states in the nusinersen economic models. Lastly, the CHERISH trial PedsQol data was mapped to EQ-5D using a published algorithm. RESULTS: The three methodologies showed differences in health utilities for defined nusinersen health states. The cross-sectional study parent-proxy Qol assessment did not provide sufficient detail on patient health to determine with any amount of certainty an individual’s state of health based on the model health states. Physicians on average differentiated between Qol in different health states in a manner consistent with disease severity, and generally ranked Qol lower than observed by parents. Parent-proxy assessments of Qol in the nusinersen CHERISH trial showed little differentiation between lower and higher motor function health states, and in general parents rated Qol high, which is consistent with studies in other paediatric diseases. CONCLUSIONS: Our findings show that different methodologies yield distinct and sometimes equivocal results with parents rating Qol higher than physicians in individuals with SMA.
Conference/Value in Health Info
2017-11, ISPOR Europe 2017, Glasgow, Scotland
Value in Health, Vol. 20, No. 9 (October 2017)
Code
PND48
Topic
Patient-Centered Research
Topic Subcategory
Health State Utilities, Patient-reported Outcomes & Quality of Life Outcomes
Disease
Neurological Disorders, Rare and Orphan Diseases