Estimated population totals for each outcome in 1 year following the 2018 visit, presented as N/1000 (95% prediction interval (95% PI)), and % reductions: with no treatment effects applied, with the effect of TEZ/IVA imposed and with the effect of ELEX/TEZ/IVA imposed
| Eligibility group | No treatment effects | With TEZ/IVA effect applied to eligible individuals | With ELEX/TEZ/IVA effect applied to eligible individuals, including those assumed to switch from TEZ/IVA | ||
| N/1000 (95% PI) | N/1000 (95% PI) | % reduction (95% PI) | N/1000 (95% PI) | % reduction (95% PI) | |
| (A) Using approach 1 for imposing treatment effects | |||||
| Hospital-IV-days | |||||
| Full cohort* | 67.7 (64.7, 71.3) | 64.5 (61.3, 67.7) | 4.8 (–1.2, 10.1) | 51.6 (48.5, 54.7) | 20.0 (14.3, 25.2) |
| TEZ/IVA+ELEX/TEZ/IVA† | 41.5 (39.1, 44.4) | 38.3 (36.0, 40.8) | 7.7 (0.5, 14.4) | 30.3 (28.0, 32.7) | 20.8 (13.5, 27.4) |
| ELEX/TEZ/IVA‡ | 60.2 (57.3, 63.6) | 57.0 (54.0, 60.1) | 5.3 (–0.8, 11.2) | 44.0 (41.0, 47.0) | 22.7 (16.7, 28.5) |
| Home-IV-days | |||||
| Full cohort | 47.3 (44.8, 49.9) | 46.1 (43.7, 48.9) | 2.5 (–4.2, 8.3) | 41.3 (38.9, 44.1) | 10.2 (3.7, 16.4) |
| TEZ/IVA+ELEX/TEZ/IVA | 30.1 (28.2, 32.2) | 29.0 (27.0, 31.3) | 3.9 (–4.5, 11.7) | 25.9 (24.1, 28.1) | 10.2 (2.0, 18.1) |
| ELEX/TEZ/IVA | 42.7 (40.4, 45.1) | 41.5 (39.1, 44.2) | 2.7 (–4.5, 9.3) | 36.7 (34.4, 39.3) | 11.4 (4.3, 17.9) |
| Combined-IV-days | |||||
| Full cohort | 115.0 (110.9, 119.5) | 110.6 (106.7, 114.9) | 3.8 (–0.4, 8.0) | 92.8 (92.8, 92.8) | 16.1 (13.0, 19.2) |
| TEZ/IVA+ELEX/TEZ/IVA | 71.7 (68.5, 75.2) | 67.3 (64.1, 70.6) | 6.1 (0.7, 11.4) | 56.3 (56.3, 56.3) | 16.4 (12.2, 20.3) |
| ELEX/TEZ/IVA | 102.9 (99.0, 107.3) | 98.5 (94.9, 102.7) | 4.3 (–0.4, 8.6) | 80.7 (80.7, 80.7) | 18.1 (14.9, 21.4) |
| (B) Using approach 2 for imposing treatment effects | |||||
| Hospital-IV-days | |||||
| Full cohort* | 67.7 (64.6, 71.2) | 53.5 (50.9, 56.3) | 21.0 (20.0, 21.9) | 30.3 (28.7, 32.0) | 43.4 (42.2, 44.6) |
| TEZ/IVA+ELEX/TEZ/IVA† | 41.5 (39.2, 44.3) | 27.3 (25.7, 29.2) | 34.3 (33.5, 35.1) | 15.7 (14.7, 16.7) | 42.6 (41.4, 43.6) |
| ELEX/TEZ/IVA‡ | 60.2 (57.1, 63.5) | 46.0 (43.6, 48.6) | 23.7 (22.7, 24.6) | 22.7 (21.5, 24.0) | 50.6 (49.7, 51.5) |
| Home-IV-days | |||||
| Full cohort | 47.3 (44.9, 49.9) | 36.9 (35.1, 39.1) | 21.8 (20.8, 22.9) | 20.7 (19.5, 22.0) | 44.0 (42.6, 45.3) |
| TEZ/IVA+ELEX/TEZ/IVA | 30.1 (28.2, 32.2) | 19.8 (18.4, 21.3) | 34.3 (33.3, 35.2) | 11.4 (10.6, 12.3) | 42.6 (41.2, 43.8) |
| ELEX/TEZ/IVA | 42.7 (40.3, 45.2) | 32.4 (30.5, 34.3) | 24.2 (23.1, 25.2) | 16.1 (15.2, 17.1) | 50.2 (49.2, 51.3) |
| Combined-IV-days | |||||
| Full cohort | 115.0 (111.0, 119.4) | 90.5 (87.2, 94.0) | 21.3 (20.6, 22.0) | 51.0 (49.0, 53.1) | 43.6 (42.7, 44.6) |
| TEZ/IVA+ELEX/TEZ/IVA | 71.7 (68.6, 75.1) | 47.1 (45.0, 49.5) | 34.3 (33.6, 34.9) | 27.1 (25.8, 28.5) | 42.6 (41.7, 43.5) |
| ELEX/TEZ/IVA | 102.9 (98.8, 107.3) | 78.3 (75.2, 81.6) | 23.9 (23.1, 24.6) | 38.8 (37.3, 40.5) | 50.4 (49.7, 51.1) |
For the TEZ/IVA effect results, the % reduction is relative to the situation with no treatment effects. For the ELEX/TEZ/IVA results, the % reduction is relative to the TEZ/IVA results.
*The full cohort of N=6407 individuals.
†The subset eligible for both TEZ/IVA and ELEX/TEZ/IVA (n=3481).
‡The whole set of individuals eligible for ELEX/TEZ/IVA, including those assumed to switch from TEZ/IVA (n=5155).
ELEX, elexacaftor; IVA, ivacaftor; TEZ, tezacaftor.