S23orphan drugs. Quality of life data seems to be a more critical outcome for OOD in the 3 countries. CONCLUSIONS: Despite less compelling evidence and substantial trials limitations, OODs achieve similar HTA recommendation as non-OODs in France suggesting a more flexible attitude in the deliberative process. Similar findings in Germany are driven by mandatory additional benefit for ODs. While in Canada, OODs achieve a lower positive recommendation than non-OOD suggesting a more strict deliberative process.
Sensitivity analyses eliminating T315I status from the weighting yielded similar results. ConClusions: Adjusted ponatinib efficacy outcomes in third-line CP-CML were similar to unadjusted, and efficacy was more than double that observed with bosutinib, irrespective of T315I mutation status. These results suggest that the higher efficacy observed is PACE (ponatinib) versus the bosutinib trial is not due to bias in patient selection.
progression-free survival (PFS), progressed disease (PD) and death. The disease progression and death data from the real-world study results were used as clinical effectiveness input in the model. The drug costs were retrieved from IQVIA CHPA database. Other medical expenses including adverse events costs, laboratory test cost, hospitalization cost, and end-of-life cost were collected from public literatures and local clinician interviews. The quality-adjusted life years (QALYs), direct medical costs and incremental cost-effectiveness ratios (ICERs) were reported. We used lifetime horizon and healthcare system perspective. Both QALY and costs were discounted at a rate of 3.5%. Uncertainty was assessed by one-way and probabilistic sensitivity analysis. Results: AG regimen provided an effectiveness of 1.35 QALY at an average cost of RMB 153,872 whereas FOLFIRINOX regimen brought 0.82 QALY at a cost of RMB 158,565 in a lifetime horizon. Therefore, AG regimen was dominant with an ICER of RMB -8,971 compared with FOLFIRINOX regimen. AG arm generated less 1 st -line treatment drug cost, 1 st -line medical cost, hospitalization cost and end-of life cost than FOLFIRINOX arm. Sensitivity analyses confirmed the robustness of the results. Conclusions: As the first study comparing cost-effectiveness of these two regimens, AG is likely the better option for the 1 st -line mPC treatment compared with FOLFIRINOX in China.
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