Cost-Effectiveness of Connected Injection Device for Pediatric Growth Hormone Deficiency in Spain: A Scenario-Based Microsimulation Analysis Using Real-World Data
Author(s)
Antonio de Arriba, PhD1, Claudia Roeder, MSc2, Emilia Nivelle, MSc3, Paula van Dommelen, PhD4, José Juan Alcón Sáez, PhD5, Cristina Latre Gorbe, PhD6, Heidi de los Santos Real, PhD7, Irene Sanchez Collado, PhD7, Falko Boehm, PhD8, Cristina Masseria, PhD9.
1Pediatric Endocrinology Unit, Hospital Miguel Servet, Zaragoza University, Zaragoza, Spain, 2AESARA, Zug, Switzerland, 3She Consulting, Brussels, Belgium, 4Department of Child Health, The Netherlands Organization for Applied Scientific Research, Leiden, Netherlands, 5Unidad de Endocrinología Pediátrica, Consorcio Hospital General Universitario de Valencia, Valencia, Spain, 6Hospital Sant Joan de Déu, Barcelona, Spain, 7Merck S.L.U, an affiliate of Merck KGaA, Madrid, Spain, 8Merck Healthcare, Darmstadt, Germany, 9Vice President Value & Evidence, Aesara, Madrid, Spain.
1Pediatric Endocrinology Unit, Hospital Miguel Servet, Zaragoza University, Zaragoza, Spain, 2AESARA, Zug, Switzerland, 3She Consulting, Brussels, Belgium, 4Department of Child Health, The Netherlands Organization for Applied Scientific Research, Leiden, Netherlands, 5Unidad de Endocrinología Pediátrica, Consorcio Hospital General Universitario de Valencia, Valencia, Spain, 6Hospital Sant Joan de Déu, Barcelona, Spain, 7Merck S.L.U, an affiliate of Merck KGaA, Madrid, Spain, 8Merck Healthcare, Darmstadt, Germany, 9Vice President Value & Evidence, Aesara, Madrid, Spain.
OBJECTIVES: To evaluate the cost-effectiveness of the connected electronic device, Easypod®, for recombinant human growth hormone, compared to non-connected devices in the treatment of pediatric growth hormone deficiency (GHD) in Spain.
METHODS: A microsimulation model of 10,000 children aged 2-13 years was developed from the perspective of the Spanish Health System, using a time horizon based on bone maturation age (17 years for boys; 15 for girls). The model combined a decision tree with a 6-month cycle Markov model (adherence states: continuous, intermittent, discontinuation). Adherence data for the connected device were sourced from Spanish real-world evidence (RWE) study[1]. For non-connected devices, estimates from the RWE1 study and KOL insights were used to assess a range of adherence scenarios. Outcomes were height gain (cm) until adulthood, utilities, and direct medical costs, discounted at 3% annually.
[1] de Arriba A et al. Front. Endocrinol 2024. 15:1450573.
RESULTS: The connected injection device achieved a superior height gain of 4.3 cm compared to non-connected devices. Treatment costs were higher due to higher adherence rates, although partially offset by reduced costs related to unnecessary dose increases and fewer clinical visits. The analysis showed a 0.1 gain in QALYs for the connected device. The incremental cost-effectiveness ratio was €28,379 per QALY gained. The cost per cm of height gained was lower for connected versus for non-connected devices (difference of €400). Results were mostly sensitive to adherence assumptions and age of treatment initiation.
CONCLUSIONS: Connected injection devices enabled early detection of non-adherence, helping clinicians optimize treatment, avoid unnecessary dose escalations, and identify non-responders. In Spain, the connected injection device has proven to be a cost-effective option for managing pediatric GHD with real-time monitoring improving outcomes and efficiency. Early initiation and sustained adherence drive effectiveness. These findings support broader adoption of innovative digital devices to optimize care.
METHODS: A microsimulation model of 10,000 children aged 2-13 years was developed from the perspective of the Spanish Health System, using a time horizon based on bone maturation age (17 years for boys; 15 for girls). The model combined a decision tree with a 6-month cycle Markov model (adherence states: continuous, intermittent, discontinuation). Adherence data for the connected device were sourced from Spanish real-world evidence (RWE) study[1]. For non-connected devices, estimates from the RWE1 study and KOL insights were used to assess a range of adherence scenarios. Outcomes were height gain (cm) until adulthood, utilities, and direct medical costs, discounted at 3% annually.
[1] de Arriba A et al. Front. Endocrinol 2024. 15:1450573.
RESULTS: The connected injection device achieved a superior height gain of 4.3 cm compared to non-connected devices. Treatment costs were higher due to higher adherence rates, although partially offset by reduced costs related to unnecessary dose increases and fewer clinical visits. The analysis showed a 0.1 gain in QALYs for the connected device. The incremental cost-effectiveness ratio was €28,379 per QALY gained. The cost per cm of height gained was lower for connected versus for non-connected devices (difference of €400). Results were mostly sensitive to adherence assumptions and age of treatment initiation.
CONCLUSIONS: Connected injection devices enabled early detection of non-adherence, helping clinicians optimize treatment, avoid unnecessary dose escalations, and identify non-responders. In Spain, the connected injection device has proven to be a cost-effective option for managing pediatric GHD with real-time monitoring improving outcomes and efficiency. Early initiation and sustained adherence drive effectiveness. These findings support broader adoption of innovative digital devices to optimize care.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE242
Topic
Clinical Outcomes, Economic Evaluation, Epidemiology & Public Health
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
Cardiovascular Disorders (including MI, Stroke, Circulatory), Diabetes/Endocrine/Metabolic Disorders (including obesity), No Additional Disease & Conditions/Specialized Treatment Areas, Pediatrics