PB0515 - Societal Perspective in Measuring the Economic Burden of Haemophilia: A Scoping Review

Background: The high cost of haemophilia treatment and its long-term complications contribute to a significant humanistic and economic burden. Economic analyses in haemophilia primarily adopt a payer perspective, overlooking direct non-medical and indirect costs. Adopting a societal perspective adds valuable insights for healthcare policy and reimbursement decisions.

Aims: This scoping review aims to map economic analyses conducted in haemophilia and to identify those that adopted a societal perspective.

Methods: We systematically searched eight databases, grey literature, and performed manual searches on the 5th of January 2024 to identify relevant publications. Inclusion criteria were full-text English publications of economic studies in haemophilia, conducted from a societal perspective, with no date limits.

Results: Out of the 3,341 retrieved records, 2,978 were excluded after screening and examination. We identified 363 economic studies, of which 51 (14%) adopted a societal perspective and were included in this review (Figure 1). From these studies, 26 (51%) were based on economic models, while 25 (49%) utilised prospective and retrospective data from actual cohorts. In terms of economic analysis, 23 (45%) were cost-of-illness analyses, 14 (27%) were cost-utility analyses, and 7 (14%) were comparative and non-comparative analyses (Figure 2). Regarding interventions, 19 (37%) studies evaluated the cost of routine care, 14 (27%) estimated the cost of prophylaxis compared to episodic treatment, 6 (12%) compared the cost of Emicizumab to other treatment modalities, 5 (10%) estimated the cost of prophylaxis, and 4 (8%) evaluated the cost of medical and surgical interventions.

Conclusion(s): Despite the comprehensiveness of analyses conducted from a societal perspective, only a small fraction of the total number of haemophilia economic studies have adopted it. Additionally, most of the studies conducted a cost-of-illness analysis, which does not account for effectiveness or comparativeness between different interventions. This could lead to undermining the true economic and humanistic burden of haemophilia.