Table S1.
Analysis methods and the choice of priors
| Approaches and priors | Case 1 | Case 2 | Case 3 |
|---|---|---|---|
|
| |||
| Method | Single study | Meta-analysis | Multicentric cohort – no appropriate priors |
| Test data | PASS data1 (6/428) | PASS data2 (21/1,188) | PASS data3 (6/219) |
| Classical statistical analysis | Logistic model | Random-effects logistic model | Random-effects logistic model |
| Bayesian statistical analysis | Logistic model | Hierarchical (random-effects) logistic model | Hierarchical (random-effects) logistic model |
| Noninformative prior | Ok | Ok | Ok |
| Informative prior: Baxter pivotal study (1/102) | Ok | Ok | Ok |
| Informative prior: meta-analysis of OS (seven ADVATE studies) (3/569) | Ok | Ok | Ok |
| Informative prior: meta-analysis of OS (38/3,866): do you need data per study? | Ok | Ok | Ok |
| Informative prior: EUHASS study of de novo inhibitor in PUPs, ADVATE (37/141) | No | No | Ok |
| Informative prior: EUHASS study of de novo inhibitor in PUPs (108/417) | No | No | Ok |
| Informative prior: EUHASS study of inhibitors in PTPs, ADVATE (5/707) | Ok | Ok | Ok |
| Informative prior: EUHASS study of inhibitors in PTPs (all FVIII) 22/3,736 | Ok | Ok | Ok |
| Discounted prior: discounting EUHASS in PUPs, ADVATE by 75% | No | No | Ok |
| Discounted prior: discounting EUHASS in PUPs, ADVATE by 95% | No | No | Ok |
| Discounted prior: discounting EUHASS in PUPs, all by 75% | No | No | Ok |
| Discounted prior: discounting EUHASS in PUPs, all by 95% | No | No | Ok |
| Enhanced data: enhancing study data by two times – increasing number of patients | Ok | Ok | Ok |
| Enhanced data: enhancing study data by two times – increasing number of studies | No | Ok | Ok |
| Enhanced data: enhancing study data by ten times – increasing number of studies | Ok | Ok | Ok |
| Enhanced data: enhancing study data by ten times – increasing number of studies | No | Ok | Ok |
Note: “OK” means it is valid choice thus will be used in the analysis. “No” means it is no a valid choice and won’t be used in the analysis.
Abbreviations: CI, confidence interval; CrI, credible interval; EUHASS, European Hemophilia Safety Surveillance; OS, observational study; PASS, post-authorization safety studies; PTP, previously treated patient; PUP, previously untreated patient.