Estimated population means 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 | ||
| Mean (95% PI) | Mean (95% PI) | % reduction (95% PI) | Mean (95% PI) | % reduction (95% PI) | |
| (A) Using approach 1 for imposing treatment effects | |||||
| Hospital-IV-days | |||||
| Full cohort* | 10.6 (10.1, 11.1) | 10.1 (9.6, 10.6) | 4.8 (–1.2, 10.1) | 8.0 (7.6, 8.5) | 20.0 (14.3, 25.2) |
| TEZ/IVA+ELEX/TEZ/IVA† | 11.9 (11.2, 12.8) | 11.0 (10.4, 11.7) | 7.7 (0.5, 14.4) | 8.7 (8.0, 9.4) | 20.8 (13.5, 27.4) |
| ELEX/TEZ/IVA‡ | 11.7 (11.1, 12.3) | 11.1 (10.5, 11.7) | 5.3 (–0.8, 1.2) | 8.5 (8.0, 9.1) | 22.7 (16.7, 28.5) |
| Home-IV-days | |||||
| Full cohort | 7.4 (7.0, 7.8) | 7.2 (6.8, 7.6) | 2.5 (–4.2, 8.3) | 6.4 (6.1, 6.9) | 10.2 (3.7, 16.4) |
| TEZ/IVA+ELEX/TEZ/IVA | 8.7 (8.1, 9.2) | 8.3 (7.7, 9.0) | 3.9 (–4.5, 11.7) | 7.5 (6.9, 8.1) | 10.2 (2.0, 8.1) |
| ELEX/TEZ/IVA | 8.3 (7.8, 8.8) | 8.0 (7.6, 8.6) | 2.7 (–4.5, 9.3) | 7.1 (6.7, 7.6) | 11.4 (4.3, 17.9) |
| Combined-IV-days | |||||
| Full cohort | 18.0 (17.3, 18.6) | 17.3 (16.7, 17.9) | 3.8 (–0.4, 8.0) | 14.5 (14.5, 14.5) | 16.1 (13.0, 19.2) |
| TEZ/IVA+ELEX/TEZ/IVA | 20.6 (19.7, 21.6) | 19.3 (18.4, 20.3) | 6.1 (0.7, 11.4) | 16.2 (16.2, 16.2) | 16.4 (12.2, 20.3) |
| ELEX/TEZ/IVA | 20.0 (19.2, 20.8) | 19.1 (18.4, 19.9) | 4.3 (–0.4, 8.6) | 15.7 (15.7, 15.7) | 18.1 (14.9, 21.4) |
| (B) Using approach 2 for imposing treatment effects | |||||
| Hospital-IV-days | |||||
| Full cohort* | 10.6 (10.1, 11.1) | 8.4 (7.9, 8.8) | 21.0 (20.0, 21.9) | 4.7 (4.5, 5.0) | 43.4 (42.2, 44.6) |
| TEZ/IVA+ELEX/TEZ/IVA† | 11.9 (11.3, 12.7) | 7.8 (7.4, 8.4) | 34.3 (33.5, 35.1) | 4.5 (4.2, 4.8) | 42.6 (41.4, 43.6) |
| ELEX/TEZ/IVA‡ | 11.7 (11.1, 12.3) | 8.9 (8.5, 9.4) | 23.7 (22.7, 24.6) | 4.4 (4.2, 4.7) | 50.6 (49.7, 51.5) |
| Home-IV-days | |||||
| Full cohort | 7.4 (7.0, 7.8) | 5.8 (5.5, 6.1) | 21.8 (20.8, 22.9) | 3.2 (3.0, 3.4) | 44.0 (42.6, 45.3) |
| TEZ/IVA+ELEX/TEZ/IVA | 8.7 (8.1, 9.3) | 5.7 (5.3, 6.1) | 34.3 (33.3, 35.2) | 3.3 (3.0, 3.5) | 42.6 (41.2, 43.8) |
| ELEX/TEZ/IVA | 8.3 (7.8, 8.8) | 6.3 (5.9, 6.7) | 24.2 (23.1, 25.2) | 3.1 (2.9, 3.3) | 50.2 (49.2, 51.3) |
| Combined-IV-days | |||||
| Full cohort | 18.0 (17.3, 18.6) | 14.1 (13.6, 14.7) | 21.3 (20.6, 22.0) | 8.0 (7.6, 8.3) | 43.6 (42.7, 44.6) |
| TEZ/IVA+ELEX/TEZ/IVA | 20.6 (19.7, 21.6) | 13.5 (12.9, 14.2) | 34.3 (33.6, 34.9) | 7.8 (7.4, 8.2) | 42.6 (41.7, 43.5) |
| ELEX/TEZ/IVA | 20.0 (19.2, 20.8) | 15.2 (14.6, 15.8) | 23.9 (23.1, 24.6) | 7.5 (7.2, 7.9) | 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, ivacaft; TEZ, tezacaftor.