Table A4.
PSA assumptions.
| Parameter | Distribution | Mean | Standard error | Source |
|---|---|---|---|---|
| Change in ppFEV1 by week 16 for LUM/IVA + SC patients ⩾ 12 years | Normal, bounded by 0 | 2.80 | 0.52 | 95% CI (Ratjen et al.18) |
| Change in ppFEV1 by week 24 for LUM/IVA + SC patients 6–11 years | Normal, bounded by 0 | 2.40 | 1.00 | 95% CI (Wainwright et al.10) |
| Change in weight-for-age Z score over 2 years for LUM/IVA + SC patients | Normal, bounded by 0 | 0.066 | 0.028 | 95% CI (Konstan et al.13) |
| Change in weight-for-age Z score over 2 years for SC patients | Normal, bounded by 0 | −0.060 | 0.015 | 95% CI (Konstan et al.13) |
| Age-dependent ppFEV1 rates of decline after 24 weeks for SC 6–8 years | Normal, bounded by 0 | −1.12 | 0.22 | Assumed 20% of mean |
| Age-dependent ppFEV1 rates of decline after 24 weeks for SC 9–12 years | Normal, bounded by 0 | −2.39 | 0.48 | Assumed 20% of mean |
| Age-dependent ppFEV1 rates of decline after 24 weeks for SC 13–17 years | Normal, bounded by 0 | −2.34 | 0.47 | Assumed 20% of mean |
| Age-dependent ppFEV1 rates of decline after 24 weeks for SC 18–24 years | Normal, bounded by 0 | −1.92 | 0.38 | Assumed 20% of mean |
| Age-dependent ppFEV1 rates of decline after 24 weeks for SC 25+ years | Normal, bounded by 0 | −1.45 | 0.29 | Assumed 20% of mean |
| Long-term reduction in rate of ppFEV1 decline with LUM/IVA + SC (all ages) | Beta, bounded by 0 | 42.0% | 0.112 | 95% CI (Konstan et al.,13
VXR-HQ-88-00035) |
| LUM/IVA + SC PEx rate ratio for patients ⩾ 12 years | Log-normal, bounded by 0 | 0.440 | 0.152 | 95% CI (Wainwright et al.10) |
| Multiplier for annual PEx rate (parameter a of Goss equation), patients ⩾ 18 years | Normal, bounded by 0 | 8.594 | 1.719 | Assumed 20% of mean |
| Multiplier for annual PEx rate (parameter a of Goss equation), patients ⩾ 18 years | Normal, bounded by 0 | 3.789 | 0.758 | Assumed 20% of mean |
CI, confidence interval; LUM/IVA, lumacaftor and ivacaftor; ppFEV1, percent predicted forced expiratory volume in 1 second; PEx, pulmonary exacerbation; SC, standard care.