Italian Perspective on Rare Disease New Treatments and HTA: Insights From Iptacopan Multidimensional Evaluation in PNH
Author(s)
Lucrezia Bianca Ferrario, mecon1, LORENA D'ANNA, MEcon2, Emanuela Foglia, PhD1.
1HD LAB – Healthcare Datascience LAB - Carlo Cattaneo – LIUC University, Castellanza, Italy, 2NOVARTIS FARMA SPA, Milano, Italy.
1HD LAB – Healthcare Datascience LAB - Carlo Cattaneo – LIUC University, Castellanza, Italy, 2NOVARTIS FARMA SPA, Milano, Italy.
OBJECTIVES: Analyze multidimensional benefits of oral iptacopan as treatment of Paroxysmal Nocturnal Hemoglobinuria-PNH compared to current treatments in Italy (C5-inhibitors eculizumab or ravulizumab, and the C3-inhibitor pegcetacoplan). Given iptacopan’s favorable efficacy, the analysis would focus on its impact on patient quality of life, National Health Service economic and organizational sustainability.
METHODS: Health Technology Assessment, based on the EUnetHTA Core Model, was conducted from the Italian NHS, assuming implementation of iptacopan. The analysis included: i) literature review, to assess comparative efficacy and safety; ii) qualitative questionnaires administered to 14 healthcare professionals and 8 PNH patients, using a 7-item Likert scale, to investigate iptacopan’s perceived multidimensional advantages over current treatments, with inferential testing to evaluate statistically significant differences in perceived benefits; and iii) quantitative tools to estimate potential economic, organizational, and social benefits of shifting from infusion-based therapy to oral one.
RESULTS: Accounting for all direct healthcare costs (diagnosis, monitoring, adverse events management, transfusions, drug acquisition), iptacopan resulted in a 10% cost-reduction per patient versus ravulizumab, however a 5% and 47% increase versus pegcetacoplan and eculizumab (including biosimilar). Excluding drug costs, iptacopan consistently required fewer resources with per-patient cost-savings ranging from 15% to 52%. Overall, iptacopan showed a cost-effective nature versus all comparators, due to superior hematologic response. At national level, routine adoption of iptacopan would generate economic annual savings of 1.4% (-2.200.671€) for treating 452 PNH patients. Oral therapy also reduces hospital visits, productivity loss, and CO₂ emissions. Healthcare professionals reported their preference towards iptacopan, declaring better perceived safety, effectiveness, and impact on waiting lists (p-value<0.05). Patients acknowledged iptacopan’s potential to reduce fatigue and PNH-related symptoms, to improve emotional health, family and social life, and ability to perform daily activities (p-value <0.05).
CONCLUSIONS: This analysis showed iptacopan’s multidimensional benefits in optimizing PNH outcomes, generating value for the NHS, hospitals, and patients.
METHODS: Health Technology Assessment, based on the EUnetHTA Core Model, was conducted from the Italian NHS, assuming implementation of iptacopan. The analysis included: i) literature review, to assess comparative efficacy and safety; ii) qualitative questionnaires administered to 14 healthcare professionals and 8 PNH patients, using a 7-item Likert scale, to investigate iptacopan’s perceived multidimensional advantages over current treatments, with inferential testing to evaluate statistically significant differences in perceived benefits; and iii) quantitative tools to estimate potential economic, organizational, and social benefits of shifting from infusion-based therapy to oral one.
RESULTS: Accounting for all direct healthcare costs (diagnosis, monitoring, adverse events management, transfusions, drug acquisition), iptacopan resulted in a 10% cost-reduction per patient versus ravulizumab, however a 5% and 47% increase versus pegcetacoplan and eculizumab (including biosimilar). Excluding drug costs, iptacopan consistently required fewer resources with per-patient cost-savings ranging from 15% to 52%. Overall, iptacopan showed a cost-effective nature versus all comparators, due to superior hematologic response. At national level, routine adoption of iptacopan would generate economic annual savings of 1.4% (-2.200.671€) for treating 452 PNH patients. Oral therapy also reduces hospital visits, productivity loss, and CO₂ emissions. Healthcare professionals reported their preference towards iptacopan, declaring better perceived safety, effectiveness, and impact on waiting lists (p-value<0.05). Patients acknowledged iptacopan’s potential to reduce fatigue and PNH-related symptoms, to improve emotional health, family and social life, and ability to perform daily activities (p-value <0.05).
CONCLUSIONS: This analysis showed iptacopan’s multidimensional benefits in optimizing PNH outcomes, generating value for the NHS, hospitals, and patients.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA213
Topic
Economic Evaluation, Health Service Delivery & Process of Care, Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
Rare & Orphan Diseases