Evaluating the Value Elements Considered in Health Technology Assessments of Paroxysmal Nocturnal Hemoglobinuria Treatments: A Targeted Review
Author(s)
Anggie Wiyani, MSc1, Misaki Okuyama, BSc (Hons)2, Eve McArthur, BSc2, Maria-Magdalena Balp, MASc, MD3.
1Novartis Pharmaceuticals UK Ltd., London, United Kingdom, 2Costello Medical, London, United Kingdom, 3Novartis Pharma AG, Basel, Switzerland.
1Novartis Pharmaceuticals UK Ltd., London, United Kingdom, 2Costello Medical, London, United Kingdom, 3Novartis Pharma AG, Basel, Switzerland.
OBJECTIVES: The ISPOR value flower includes 10 elements of value beyond quality-adjusted life years (QALYs) and costs that warrant consideration in health technology assessments (HTAs): productivity, family and scientific spillover, equity, disease severity, insurance, fear of disease, value of knowing, hope and real-option. HTA bodies’ adoption and perspective of these values remain unclear, particularly in rare diseases including paroxysmal nocturnal hemoglobinuria (PNH). This analysis assessed how value elements were considered in HTAs for PNH treatments and their impact on HTA decision-making.
METHODS: A targeted search was conducted in June 2025 for PNH treatments approved since 2020 (iptacopan, pegcetacoplan, danicopan, crovalimab) across five HTA body websites (NICE, GBA, HAS, TLV, Medicinrådet). Details of value elements and their impact on HTA bodies’ decisions were extracted from company submissions (CSs) and HTA Committee reports (CRs) into a structured grid.
RESULTS: 19 HTAs were captured from six GBA appraisals, four NICE, four HAS, three Medicinrådet, and two TLV. CSs and CRs were available for 13 and 19 HTAs, respectively. QALYs and/or costs were considered in most CRs (13/19). Several novel value elements were discussed in CSs and CRs: productivity (9/13; 2/19), real-option value (9/13; 2/19), equity (4/13; 3/19), disease severity (1/13; 3/19), family spillover (3/13; 0/19), value of knowing (1/13; 0/19). No other novel value elements were identified. Supporting data for these novel value elements were primarily qualitative, except for productivity. Some HTA Committees acknowledged the positive value effect of novel elements, while others dismissed it; the influence of these on the final HTA decision was unclear across all appraisals.
CONCLUSIONS: Novel ISPOR value elements were included in CSs and CRs with qualitative supporting data, however costs and QALYs remained the primary focus. Research and efforts from companies/HTA bodies are needed to generate supporting data and facilitate adoption of broader value elements in decision-making, particularly in rare diseases.
METHODS: A targeted search was conducted in June 2025 for PNH treatments approved since 2020 (iptacopan, pegcetacoplan, danicopan, crovalimab) across five HTA body websites (NICE, GBA, HAS, TLV, Medicinrådet). Details of value elements and their impact on HTA bodies’ decisions were extracted from company submissions (CSs) and HTA Committee reports (CRs) into a structured grid.
RESULTS: 19 HTAs were captured from six GBA appraisals, four NICE, four HAS, three Medicinrådet, and two TLV. CSs and CRs were available for 13 and 19 HTAs, respectively. QALYs and/or costs were considered in most CRs (13/19). Several novel value elements were discussed in CSs and CRs: productivity (9/13; 2/19), real-option value (9/13; 2/19), equity (4/13; 3/19), disease severity (1/13; 3/19), family spillover (3/13; 0/19), value of knowing (1/13; 0/19). No other novel value elements were identified. Supporting data for these novel value elements were primarily qualitative, except for productivity. Some HTA Committees acknowledged the positive value effect of novel elements, while others dismissed it; the influence of these on the final HTA decision was unclear across all appraisals.
CONCLUSIONS: Novel ISPOR value elements were included in CSs and CRs with qualitative supporting data, however costs and QALYs remained the primary focus. Research and efforts from companies/HTA bodies are needed to generate supporting data and facilitate adoption of broader value elements in decision-making, particularly in rare diseases.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA136
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
Rare & Orphan Diseases