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Bridging and Validation of a Thyroid Eye Disease (TED) Specific Quality of Life (QOL) Questionnaire to Utility Values Assessed By a Time Trade-Off (TTO) Analysis

Speaker(s)

Holt R1, Taylor S1, Barretto N1, Hirst A2, Oliver L2, Brandolini G2, Brazier JE3
1Horizon Therapeutics, Deerfield, IL, USA, 2Adelphi Values Ltd, Bollington, UK, 3University of Sheffield, Sheffield, UK

OBJECTIVES: TED, an autoimmune, inflammatory disease, is characterized by retro-orbital tissue expansion and devastating changes in facial appearance and visual function. The Graves’ Ophthalmopathy QOL (GO-QOL) questionnaire is TED specific but has not been mapped to health state utility (HSU) scores. Recently, mean score for moderate-severe TED was assessed as 0.44. To quantify QALY improvements with teprotumumab, we correlated and bridged GO-QOL values with TTO-derived utility scores.

METHODS: GO-QOL data from 2, 24-week randomized, masked, placebo-controlled teprotumumab trials (n=171) were compared with TTO values assessed from 6 health states of the trial patients based on proptosis/diplopia severity. R Project® was used for statistical assessments.

RESULTS: Baseline GO-QOL scores were consistent with HSU scores across TED severity levels indicating generally worse severity was associated with lower/worse GO-QOL. Less discrimination in GO-QOL was noted in the intermediate health states. GO-QOL scores were highly positively correlated with utility scores of the 6 health states (mean (r2=0.76) and median (r2=0.88), translating to a 1-point change in mean GO-QOL equaling a 0.013 utility change. Increasing diplopia had a major impact on worsening QOL, particularly as noted by changes in the visual GO-QOL subscale. Moving from a no diplopia state to a constant state worsened overall GO-QOL by 20.42 points. Only 1/79 (1.3%) teprotumumab patients progressed to a more severe state versus 13/78 (16.7%) for placebo. This indicates teprotumumab QALY gain over 3 years was 0.74 and 0.51 when placebo adjusted. The 3 year QALY was 2.06 for teprotumumab and 1.55 for placebo.

CONCLUSIONS: TED produces significant disutility and the clinical trial reported GO-QOL scores significantly correlated with TTO derived utility values. Declining utility values are associated with worsening GO-QOL and increasing TED severity. Teprotumumab is estimated to improve placebo adjusted QALY by 0.51 over 3 years. These bridging analyses serve as a basis for future TED QALY gain calculations.

Code

MSR67

Topic

Methodological & Statistical Research

Topic Subcategory

PRO & Related Methods, Survey Methods

Disease

Diabetes/Endocrine/Metabolic Disorders