Impact of the Societal Perspective on HTA Decision Making in Rare Diseases
Author(s)
Joana Anjo, PharmD1, Alexander Wilke, PhD2, Rupa Briah, MSc3, Jianni Singh-Landa, MSc4, Will Wright, PhD4, Megan Barnes, PhD4.
1Norgine, Lisbon, Portugal, 2Norgine, Wettenberg, Germany, 3Norgine, Birmingham, United Kingdom, 4FIECON, London, United Kingdom.
1Norgine, Lisbon, Portugal, 2Norgine, Wettenberg, Germany, 3Norgine, Birmingham, United Kingdom, 4FIECON, London, United Kingdom.
OBJECTIVES: While many European HTA bodies consider a societal perspective in decision-making, the relative influence is unknown, particularly in rare diseases. In light of recent Dutch guidelines placing greater emphasis on a societal perspective in HTA assessments, this research sought to assess the role of societal perspective in European HTA decision-making in rare diseases.
METHODS: A targeted literature review of HTA appraisals since 2022 was conducted for select countries that consider cost-effectiveness in decision-making (UK [NICE], Netherlands [ZIN], Finland [FIMEA], and Nordic countries [JNHB]).
RESULTS: From 01/01/2022 to 20/05/2025, 110 appraisals were identified (NICE: 61, ZIN: 37, FIMEA: 8, JNHB: 4). Societal impact was discussed in 66% of all identified appraisals, with a particular focus on the impact on employment of patients/caregivers (38%), and patient education (24%). Country-wise, societal impact was discussed in more appraisals by ZIN (84%), followed by NICE (62%), the Nordics (25%) and Finland (13%). Whereas new ZIN guidance recommends including societal perspective in the base case economic model, NICE does not consider societal perspective in the base case and only 20% of NICE appraisals for rare diseases included a societal scenario. On average, the societal perspective decreased the ICER by 23.5% (range: 16.3-37.8%) compared with the base case. In a separate example, the inclusion of a societal perspective, as scenario analysis, in the recent NICE appraisal of anhydrous sodium thiosulfate for prevention of cisplatin-induced ototoxicity in children (TA1034) decreased the ICER by 65% versus the healthcare perspective, reflecting a significant impact on the economic evaluation. The impact on school performance and psychosocial functioning were also considered as uncaptured health benefits
CONCLUSIONS: Given the value of societal impact in rare disease appraisals, and its implications on the ICER, there should be greater emphasis on considering a societal perspective, as base case, in these appraisals.
METHODS: A targeted literature review of HTA appraisals since 2022 was conducted for select countries that consider cost-effectiveness in decision-making (UK [NICE], Netherlands [ZIN], Finland [FIMEA], and Nordic countries [JNHB]).
RESULTS: From 01/01/2022 to 20/05/2025, 110 appraisals were identified (NICE: 61, ZIN: 37, FIMEA: 8, JNHB: 4). Societal impact was discussed in 66% of all identified appraisals, with a particular focus on the impact on employment of patients/caregivers (38%), and patient education (24%). Country-wise, societal impact was discussed in more appraisals by ZIN (84%), followed by NICE (62%), the Nordics (25%) and Finland (13%). Whereas new ZIN guidance recommends including societal perspective in the base case economic model, NICE does not consider societal perspective in the base case and only 20% of NICE appraisals for rare diseases included a societal scenario. On average, the societal perspective decreased the ICER by 23.5% (range: 16.3-37.8%) compared with the base case. In a separate example, the inclusion of a societal perspective, as scenario analysis, in the recent NICE appraisal of anhydrous sodium thiosulfate for prevention of cisplatin-induced ototoxicity in children (TA1034) decreased the ICER by 65% versus the healthcare perspective, reflecting a significant impact on the economic evaluation. The impact on school performance and psychosocial functioning were also considered as uncaptured health benefits
CONCLUSIONS: Given the value of societal impact in rare disease appraisals, and its implications on the ICER, there should be greater emphasis on considering a societal perspective, as base case, in these appraisals.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA195
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
Rare & Orphan Diseases