COST-EFFECTIVENESS ANALYSIS OF TEPROTUMOMAB VERSUS BEST SUPPORTIVE CARE FOR ACTIVE MODERATE -TO-SEVERE THYROID EYE DISEASE IN FRANCE
Author(s)
Benjamin Mazaleyrat, PharmD1, Apolline Grangeon, PharmD2, Mélina Gilberg, PhD1, Cléa SAMBUC, PhD2.
1Amgen SAS, Courbevoie, France, 2Vyoo Agency, Paris, France.
1Amgen SAS, Courbevoie, France, 2Vyoo Agency, Paris, France.
OBJECTIVES: The OPTIC phase 3 trial assessed the efficacy and safety of teprotumumab in adult patients with active, moderate-to-severe thyroid eye disease (TED), a relatively rare complication of Graves’ disease. This study assessed the cost-effectiveness of teprotumumab versus best supportive care (BSC) in France, specifically in patients presenting with diplopia and/or proptosis, and the necessity for surgical intervention.
METHODS: A nine-state Markov model was developed. Six distinct health states were used to describe dipoplia and proptosis evolution pairings at active TED. Additional health states were surgery, post-surgery and death. Since TED is not associated with increasing mortality, cost per time spent in the most favorable health state (small proptosis, no diplopia) was used instead of cost per life years (LY). A complementary cost-utility analysis was proposed using a health state vignette study using a Time Trade-Off Approach. Costs were estimated from a payer perspective using comprehensive French claims databases. Uncertainty was explored through deterministic, probabilistic, and scenario sensitivity analyses
RESULTS: Compared with BSC, teprotumumab increased the time spent in the most favorable health state by 2.3 years (6.98 vs. 4.68 years) and generated an incremental cost of €131,984 (€148,783 vs. €16,799) over a 20-year time horizon. The incremental cost efficacy ratio was € 57 341 per year in the most favorable health state. The complementary cost-utility analysis leads to 0.82 additional quality adjusted life years (QALYs) compared to BSC (7.98 vs 7.17). The incremental cost-utility ratio was €161,988 /QALY. Patients treated with teprotumumab were also less likely to transition through the surgery health state (0.35 versus 0.63 years) or the post‑surgery health state (2.31 versus 4.21 years).
CONCLUSIONS: This cost-effectiveness analysis shows that data paucity in a rare disease setting may be overcome using alternative criteria, specific to disease progression. Economic value of teprotumumab needs to be confirmed after final pricing negotiation in France.
METHODS: A nine-state Markov model was developed. Six distinct health states were used to describe dipoplia and proptosis evolution pairings at active TED. Additional health states were surgery, post-surgery and death. Since TED is not associated with increasing mortality, cost per time spent in the most favorable health state (small proptosis, no diplopia) was used instead of cost per life years (LY). A complementary cost-utility analysis was proposed using a health state vignette study using a Time Trade-Off Approach. Costs were estimated from a payer perspective using comprehensive French claims databases. Uncertainty was explored through deterministic, probabilistic, and scenario sensitivity analyses
RESULTS: Compared with BSC, teprotumumab increased the time spent in the most favorable health state by 2.3 years (6.98 vs. 4.68 years) and generated an incremental cost of €131,984 (€148,783 vs. €16,799) over a 20-year time horizon. The incremental cost efficacy ratio was € 57 341 per year in the most favorable health state. The complementary cost-utility analysis leads to 0.82 additional quality adjusted life years (QALYs) compared to BSC (7.98 vs 7.17). The incremental cost-utility ratio was €161,988 /QALY. Patients treated with teprotumumab were also less likely to transition through the surgery health state (0.35 versus 0.63 years) or the post‑surgery health state (2.31 versus 4.21 years).
CONCLUSIONS: This cost-effectiveness analysis shows that data paucity in a rare disease setting may be overcome using alternative criteria, specific to disease progression. Economic value of teprotumumab needs to be confirmed after final pricing negotiation in France.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE261
Topic
Economic Evaluation, Health Technology Assessment
Disease
Rare & Orphan Diseases, Sensory System Disorders (Ear, Eye, Dental, Skin), Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)