Table 4.
Incremental cost-effectiveness ratio (ICER) in severe hemophilia A patients after the treatment modality switch
| Study year | |||||||||
|---|---|---|---|---|---|---|---|---|---|
| O | P1 | P2 | P3 | P4 | ∆P1 – O | ∆P2 – O | ∆P3 – O | ∆P4 – O | |
| All patients | |||||||||
| Patient number | 35 | 35 | 34 | 30 | 26 | ||||
| Average annual FVIII consumption (IU/year) | 155,394 | 219,036 | 210,182 | 212,989 | 213,536 | 63,641 | 54,788 | 57,594 | 58,142 |
| Average cost of FVIII (USD) | 117,840 | 166,100 | 159,387 | 161,515 | 161,930 | 48,261 | 41,547 | 43,675 | 44,090 |
| Average ABR (bleeds/year) | 27.2 | 6.3 | 4.1 | 3.1 | 2.5 | 20.9 | 23.1 | 24.1 | 24.7 |
| Average AjBR (bleeds/year) | 17.9 | 4.9 | 3.0 | 2.3 | 1.7 | 13.0 | 14.9 | 15.6 | 16.2 |
| USD per ABR avoided | 2304 | 1793 | 1813 | 1780 | |||||
| USD per AjBR avoided | 3721 | 2786 | 2796 | 2716 | |||||
| ABR, median (IQR) | 29.0 (13.5–38.0) | 6.0 (2.0–9.5) | 4.0 (1.0–5.0) | 2.0 (1.0–4.8) | 2.0 (1.0–3.0) | ||||
| AjBR, median (IQR) | 18.0 (6.0–26.0) | 4.0 (1.0–7.0) | 2.0 (1.0–4.0) | 2.0 (1.0–3.8) | 1.0 (1.0–2.0) | ||||
| Age ≤ 18 years | |||||||||
| Patient number | 6 | 6 | 6 | 6 | 6 | ||||
| Average annual FVIII consumption (IU/year) | 118,292 | 131,500 | 132,667 | 125,792 | 130,903 | 13,208 | 14,375 | 7500 | 12,611 |
| Average cost of FVIII (USD) | 89,704 | 99,720 | 100,605 | 95,391 | 99,267 | 10,016 | 10,901 | 5687 | 9563 |
| Average ABR (bleeds/year) | 31.8 | 8.0 | 6.0 | 4.2 | 3.2 | 23.8 | 25.8 | 27.6 | 28.6 |
| Average AjBR (bleeds/year) | 16.5 | 4.3 | 3.2 | 1.7 | 0.8 | 12.2 | 13.3 | 14.8 | 15.7 |
| USD per ABR avoided | 420 | 422 | 206 | 334 | |||||
| USD per AjBR avoided | 823 | 818 | 383 | 610 | |||||
| ABR, median (IQR) | 34.5 (34.0–37.3) | 8.5 (7.0–10.0) | 5.0 (4.3–5.8) | 4.0 (3.3–5.5) | 2.5 (1.3–3.8) | ||||
| AjBR, median (IQR) | 18.0 (17.3–19.5) | 4.0 (2.5–4.8) | 3.5 (1.3–5.0) | 1.5 (1.0–2.0) | 1.0 (1.0–1.0) | ||||
| Age > 18 years | |||||||||
| Patient number | 29 | 29 | 28 | 24 | 20 | ||||
| Average annual FVIII consumption (IU/year) | 163,071 | 237,147 | 226,793 | 234,788 | 238,326 | 74,076 | 63,722 | 71,717 | 75,255 |
| Average cost of FVIII (USD) | 123,661 | 179,834 | 171,983 | 178,046 | 180,729 | 56,174 | 48,322 | 54,385 | 57,068 |
| Average ABR (bleeds/year) | 26.3 | 5.9 | 3.6 | 2.9 | 2.3 | 20.4 | 22.7 | 23.4 | 24.0 |
| Average AjBR (bleeds/year) | 18.2 | 5.0 | 2.9 | 2.4 | 1.9 | 13.2 | 15.3 | 15.8 | 16.3 |
| USD per ABR avoided | 2761 | 2135 | 2324 | 2375 | |||||
| USD per AjBR avoided | 4276 | 3170 | 3452 | 3507 | |||||
| ABR, median (IQR) | 22.0 (13.0–38.0) | 5.0 (2.0–8.0) | 3.0 (1.0–4.3) | 2.0 (1.0–4.3) | 2.0 (1.0–3.0) | ||||
| AjBR, median (IQR) | 18.0 (5.0–30.0) | 3.0 (1.0–7.0) | 2.0 (0.8–4.0) | 2.0 (1.0–4.0) | 2.0 (1.0–2.0) | ||||
ABR annual bleeding rate, AjBR annual joint bleeding rate, FVIII factor VIII, IQR interquartile range, IU international unit, O on-demand treatment year (1 year before prophylaxis), P1 1st year of prophylactic treatment, P2 2nd year of prophylactic treatment, P3 3rd year of prophylactic treatment, P4 4th year of prophylactic treatment, USD United States dollars