ADEQUATELY REFLECTING THE CLINICAL BENEFITS IN RARE DISEASE ECONOMIC MODELING USING SMA TYPE I AS A CASESTUDY
Author(s)
Zuluaga S1, Knight C1, Thompson R2, Teynor M3
1RTI Health Solutions, Manchester, UK, 2Biogen, Zug, Switzerland, 3Biogen, Cambridge, MA, USA
OBJECTIVES: Spinal muscular atrophy (SMA) is a rare, hereditary, autosomal recessive neuromuscular disorder caused by deletion of the survival motor neuron 1 gene (SMN1). Type I SMA is one of the most severe forms of SMA that affects infants between 0-6 months of age where they never develop the ability to sit and have a short life expectancy. Nusinersen is the first approved treatment for SMA, and prior management of the disease centred on the symptomatic treatment of respiratory, nutritional, and orthopaedic function decline. The objective of this study was to build a model to adequately reflect the clinical benefits of a novel treatment (nusinersen) in a rare disease which has resulted in patients achieving motor function abilities not previously observed. METHODS: A decision analytic model was developed based on the clinical trial outcome measures, registry data and clinical opinion via an advisory board. Health states were based on both motor milestones consistent with Type I SMA and for motor milestones not previously experienced by Type I SMA patients, such as sitting without support and standing with assistance due to the improvement in motor function experienced by patients treated with nusinersen. Due to paucity of data, quality of life utility values were derived from a vignette study. Data for the major clinical events such as scoliosis surgery were based on the literature. RESULTS: The resulting model structure showed that over a 40-year time horizon patients treated with nusinesen gained an average of 6.5 life-years and over 6 quality-adjusted life years (undiscounted) CONCLUSIONS: The resulting model structure is a basis on which a full economic model can be developed to support nusinersen in future Health Technology Assessments (HTAs).
Conference/Value in Health Info
2017-11, ISPOR Europe 2017, Glasgow, Scotland
Value in Health, Vol. 20, No. 9 (October 2017)
Code
PND25
Topic
Economic Evaluation
Topic Subcategory
Cost-comparison, Effectiveness, Utility, Benefit Analysis
Disease
Musculoskeletal Disorders, Neurological Disorders, Rare and Orphan Diseases