Presentation (CTI)

OBJECTIVES: To address limitations of a health system perspective in evaluating the cost effectiveness (CE) of therapies for ultra-rare diseases (defined as US prevalence <10,000; no trials in populations >10,000), ICER employs a modified value framework that presents base-case CE results (health system perspective) alongside the societal perspective scenario analyses when societal costs are considered substantial relative to health care costs. The objective of this study was to assess which societal cost components are included in these ICER ultra-rare disease assessments in practice and how their inclusion influences CE outcomes.
METHODS: ICER Final Evidence Reports published between 2017-2025 using the ultra-rare disease framework were identified and included if they presented results from ≥1 assessment incorporating a societal perspective. Included societal cost components and CE results from the healthcare system and modified societal perspectives were collected. Analyses assessed the frequency of societal cost component inclusion and compared CE results across perspectives.
RESULTS: Six reports used the ultra-rare framework to assess CE of treatments for neuromuscular (n=2), retinal (n=2), and hematologic (n=2) conditions. The most frequently included societal cost components were labor market earnings lost (n=5), cost of unpaid lost productivity due to illness (n=5), unpaid caregiver-time costs (n=4), cost of patients’ time spent receiving care (n=4), and impact on caregiver health-related quality of life (n=3). No assessments included consumption, legal/criminal justice, housing, or environmental costs. Five assessments (83.3%) showed a decrease in the cost per quality-adjusted life year gained when societal costs were included.
CONCLUSIONS: In practice, ICER ultra-rare disease assessments incorporated a limited range of societal costs, largely focused on productivity and caregiving. The inclusion of any societal costs generally improved CE results, underscoring the importance of generating comprehensive evidence on societal cost impacts to support value narratives, economic evaluations, and launch planning for therapies targeting ultra-rare conditions.