TABLE 4.
Payer Responses to Scenarios Varying the Relative Cost, Efficacy, and Safety of 2 Hypothetical Treatments for Hemophilia Aa
| Scenario | Cost Options | Round 1 Results (n = 20) | Round 3 Results (n = 19)b | ||||
|---|---|---|---|---|---|---|---|
| Parity | Drug A Preferred | Drug B Preferred | Parity | Drug A Preferred | Drug B Preferred | ||
| 1. Assume drug A has noninferior efficacy and noninferior safety to drug B. | Drug A cost = Drug B cost | 19 (95) | 0 (0) | 1 (5) | 19 (100) | 0 (0) | 0 (0) |
| Drug A cost < Drug B cost | 3 (15) | 17 (85) | 0 (0) | 3 (16) | 16 (84) | 0 (0) | |
| Drug A cost > Drug B cost | 2 (10) | 1 (5) | 17 (85) | 3 (16) | 0 (0) | 16 (84) | |
| 2. Assume drug A has noninferior efficacy and superior safety to drug B. | Drug A cost = Drug B cost | 12 (60) | 6 (30) | 2 (10) | 11 (58) | 7 (37) | 1 (5) |
| Drug A cost < Drug B cost | 1 (5) | 17 (85) | 2 (10) | 2 (11) | 16 (84) | 1 (5) | |
| Drug A cost > Drug B cost | 7 (35) | 5 (25) | 8 (40) | 9 (47) | 4 (21) | 6 (32) | |
| 3. Assume drug A has superior efficacy and noninferior safety to drug B. | Drug A cost = Drug B cost | 9 (45) | 11 (55) | 0 (0) | 9 (47) | 10 (53) | 0 (0) |
| Drug A cost < Drug B cost | 5 (25) | 1 (5) | 14 (70) | 5 (26) | 1 (5) | 13 (68) | |
| Drug A cost > Drug B cost | 10 (50) | 3 (15) | 7 (35) | 11 (58) | 1 (5) | 7 (37) | |
| 4. Assume drug A has inferior efficacy and noninferior safety to drug B. | Drug A cost = Drug B cost | 5 (25) | 0 (0) | 15 (75) | 5 (26) | 0 (0) | 14 (74) |
| Drug A cost < Drug B cost | 3 (15) | 17 (85) | 0 (0) | 3 (16) | 16 (84) | 0 (0) | |
| Drug A cost > Drug B cost | 7 (35) | 2 (10) | 11 (55) | 8 (42) | 2 (11) | 9 (47) | |
| 5. Assume drug A has noninferior efficacy and inferior safety to drug B. | Drug A cost = Drug B cost | 9 (45) | 5 (25) | 6 (30) | 10 (53) | 3 (16) | 6 (32) |
| Drug A cost < Drug B cost | 6 (30) | 1 (5) | 13 (65) | 6 (32) | 0 (0) | 13 (68) | |
| Drug A cost > Drug B cost | 11 (55) | 5 (25) | 4 (20) | 10 (53) | 5 (26) | 4 (21) | |
| 6. Assume drug A has inferior efficacy and inferior safety to drug B. | Drug A cost = Drug B cost | 1 (5) | 0 (0) | 19 (95) | 2 (11) | 0 (0) | 17 (89) |
| Drug A cost < Drug B cost | 5 (25) | 1 (5) | 14 (70) | 5 (26) | 0 (0) | 14 (74) | |
| Drug A cost > Drug B cost | 2 (10) | 0 (0) | 18 (90) | 2 (11) | 0 (0) | 17 (89) | |
aData are n (%); data in some rows may not add up to 100 due to rounding.
bDuring round 3, consensus was achieved (green text) when at least 74% of panelists (i.e., 14 of 19 panelists) were within 1 SD of the median group estimate.