Embracing the Long-Term in Disruptive Health Innovations: A Strategic Necessity or a Necessary Evil? Cost Utility of Langerhans Islet Grafting in Unstable Type 1 Diabetes
Author(s)
Sandra David-Tchouda, MD, PhD1, Rosy Fares, PhD1, Hanane Kebaili, PharmD1, Dimitri Sourd, MS1, Thierry Berney, MD, PhD2, Laurence Kessler, MD, PhD3, Lionel Badet, MD, PhD4, Jean-Pierre Riveline, PhD, MD5, François Pattou, MD, PhD6, Sandrine Lablanche, MD, PhD1, Pierre-Yves Benhamou, MD, PhD1.
1CHU GRENOBLE, GRENOBLE, France, 2Hopital Universitaire de Geneve, GENEVA, Switzerland, 3CHRU STRASBOURG, STRASBOURG, France, 4CHU SAINT-ETIENNE, SAINT-ETIENNE, France, 5Assistance publique – Hôpitaux de Paris, PARIS, France, 6CHU Lille, LILLE, France.
1CHU GRENOBLE, GRENOBLE, France, 2Hopital Universitaire de Geneve, GENEVA, Switzerland, 3CHRU STRASBOURG, STRASBOURG, France, 4CHU SAINT-ETIENNE, SAINT-ETIENNE, France, 5Assistance publique – Hôpitaux de Paris, PARIS, France, 6CHU Lille, LILLE, France.
OBJECTIVES: Langerhans Islet Grafting (LIG) is an innovative treatment for patients with unstable type 1 diabetes (UT1D) experiencing severe glycaemic variability despite optimized intensive insulin treatment (IIT). While LIG shows clinical benefits in terms of morbidity, survival, and quality of life, economic evaluations remain scarce. The French STABILOT study aimed to assess the cost-utility of LIG versus IIT from the healthcare system’s perspective over both the short term (1 year) and long term (10 years).
METHODS: STABILOT estimated incremental cost-utility ratios (ICURs) for UT1D patients treated with LIG versus IIT. The short-term ICUR was based on 1-year follow-up data from the STABILOT clinical trial (NCT02854696). The long-term ICUR used additional data from the TRIMECO cohort to build a Markov model. Time-dependent transition probabilities (0-5 years; 6-10 years) and health states were defined. In the LIG arm: islet graft, insulin independence, insulin dependence, IIT, diabetes-related complications and cancer (DRCC), and death; in the IIT arm: IIT, DRCC, and death. Transition probabilities and utilities were derived from the literature.
RESULTS: At one-year follow-up, LIG costed an additional €218,268 and allowed a gain in quality of life of 0.104 QALYs compared with optimized IIT. The ICUR was €2,101,594/QALY [IC95%: €986,026; €34,357,588]. The 10-year results showed that LIG generates an additional cost of €140,274 and a gain in quality of life of 1.809 QALYs compared with control group resulting in ICUR of €77,530/QALY [IC95%: €25,476; €168,266].
CONCLUSIONS: Although associated with high upfront costs, LIG provides sustained improvements in long-term quality of life. In the short term, it exceeds France’s informal efficiency threshold (€50,000-100,000/QALY), but proves cost-effective over 10 years. These findings underscore the importance of long-term evaluation horizons when assessing disruptive innovations with delayed benefits. Short-term analysis alone may underestimate their true value for healthcare decision-makers.
METHODS: STABILOT estimated incremental cost-utility ratios (ICURs) for UT1D patients treated with LIG versus IIT. The short-term ICUR was based on 1-year follow-up data from the STABILOT clinical trial (NCT02854696). The long-term ICUR used additional data from the TRIMECO cohort to build a Markov model. Time-dependent transition probabilities (0-5 years; 6-10 years) and health states were defined. In the LIG arm: islet graft, insulin independence, insulin dependence, IIT, diabetes-related complications and cancer (DRCC), and death; in the IIT arm: IIT, DRCC, and death. Transition probabilities and utilities were derived from the literature.
RESULTS: At one-year follow-up, LIG costed an additional €218,268 and allowed a gain in quality of life of 0.104 QALYs compared with optimized IIT. The ICUR was €2,101,594/QALY [IC95%: €986,026; €34,357,588]. The 10-year results showed that LIG generates an additional cost of €140,274 and a gain in quality of life of 1.809 QALYs compared with control group resulting in ICUR of €77,530/QALY [IC95%: €25,476; €168,266].
CONCLUSIONS: Although associated with high upfront costs, LIG provides sustained improvements in long-term quality of life. In the short term, it exceeds France’s informal efficiency threshold (€50,000-100,000/QALY), but proves cost-effective over 10 years. These findings underscore the importance of long-term evaluation horizons when assessing disruptive innovations with delayed benefits. Short-term analysis alone may underestimate their true value for healthcare decision-makers.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE408
Topic
Economic Evaluation, Health Technology Assessment, Patient-Centered Research
Topic Subcategory
Trial-Based Economic Evaluation
Disease
Diabetes/Endocrine/Metabolic Disorders (including obesity)