Table 3.
Efficacy and costs
| EPAG | ROMI (naïve trial data) | ROMI (ITC data used in model) | Watch-and- rescue (W&R) | Adjusted ITC OR (EPAG/ROMI) (95% CI) | ∆ EPAG - ROMI | ∆ EPAG – W&R | |
|---|---|---|---|---|---|---|---|
| Efficacy | |||||||
| Overall response | 74.6% (47/63) | 71.4% (30/42) | 72.3% | 20.7% (6/29) | 1.13 (0.21, 5.96) | 2.3% | 53.9% |
| Severe bleeding (WHO 3–5) | 0.8% (0/63 – Cochrane adjustment to 0.5/63) | 11.9% (5/42) | NA | 10.3% (3/29) | 0.03 (0.00,1.15) | −22.1% | −9.6% |
| Moderate bleeding only (WHO 2) | 36.5% (23/63) | 71.4% (30/42) | 52.2% | 44.8% (13/29) | 0.53 (0.12, 2.23) | −15.7% | −8.3% |
| Use of rescue medication | 19.0% (12/63) | 40.5% (17/42) | 20.9% | 24.1% (7/29) | 0.89 (0.20, 4.02) | −1.9% | −5.1% |
| Mortality (derived from severe bleeding) | 0.03% | NA | 0.82% | 0.37% | NA | 0.79% | 0.34% |
| Costs ($) | NA | NA | NA | NA | NA | NA | NA |
| Drug costs | 62,202 | NA | 84,396 | 22,024 | NA | −22,194 | 40,178 |
| Administration costs | 0 | NA | 1,955 | 889 | NA | −1,955 | −889 |
| Routine care costs | 486 | NA | 486 | 486 | NA | 0 | 0 |
| Rescue medication costs | 983 | NA | 569 | 2,184 | NA | 414 | −1,201 |
| Cost of severe bleeding (G3–5) | 354 | NA | 10,191 | 4,613 | NA | −9,837 | −4,259 |
| Cost of moderate bleeding (G2) | 802 | NA | 1,147 | 984 | NA | −345 | −183 |
| Adverse events costs | 1,709 | NA | 1,860 | 1,335 | NA | −151 | 373 |
| Mortality costs | 16 | NA | 453 | 205 | NA | −437 | −189 |
| Total costs | 66,550 | NA | 101,056 | 32,720 | NA | −34,506 | 33,830 |
Abbreviations: EPAG, eltrombopag; ROMI, romiplostim; ITC, indirect treatment comparison; NA, not applicable; W&R, watch-and-rescue.