Iorio A, Keepanasseril A, Ibrahim Q, Iserman E, Blaser H. Self-reflections, lessons learned and suggestions for data quality assurance from a retrospective data analysis from the Canadian Bleeding Disorders Registry. (2024), POSTER ABSTRACT (PP-026). (2024), Issue Information. Haemophilia, 30: 1-2. https://doi.org/10.1111/hae.15016
Introduction
Real-world evidence (RWE) is used to complement primary evidence from clinical trials on safety and efficacy and to generate clinical effectiveness data. Theoretically, RWE is not subject to ‘on trial effect’, and it shows the true impact of intervention in unselected populations. Barriers to generating RWE are mostly stemming from the incomplete availability of relevant data points in the real-world setting. Aim: Analyse strengths and limitations of the ‘Real-World Effectiveness of PEGylated, recombinant Antihaemophilic Factor (Rurioctocog alfa pegol) Prophylaxis in Patients with Haemophilia A in Canada: A Retrospective, Intra-Patient Comparison with a Before-AfterDesign’.
Methods
A retrospective study, before-after design, comparing PK, clinical and PR outcomes for patient switched from SHL-FVIII or EHL-FVIII products to Rurioctocog alfa pegol. Outcomes: individual PK parameters (half-life, time spent above factor levels of0.01, 0.03 and 0.05 IU/mL), factor utilisation, bleed rates, QoL (PROBE)and joint function (HJHS). Pre-specified subgroups: previous treatment with SHL-FVIII versus EHL-FVIII, history of inhibitors versus no inhibitors, and age ≤12 versus >12 years. Strengths and limitations of the approach, including the amount/quality of data available for the analysis of the different outcomes, were analysed.
Results
Ninety-eight severe haemophilia A inhibitor-free patients qualified for the analysis. Thirty-seven patients had available PK data. There were four patients with history of inhibitors, six <12 years and eight patients formerly treated with SHL; four had HJHS and four had PROBE data. Clinical, PK and utilisation results have been presented elsewhere.
Conclusions
PROBE data shows a good cross-section of people with Haemophilia A, B and carriers. Brazil has free Selection bias is anticipated to be lower in retrospective design versus post-marketing surveillance (PMS) studies, which also threaten national registries data integrity. Included (n = 37) and excluded (n = 61) patients were similar in terms of age distributions, BMI, and number of surgery or traumatic bleedings, confirming that the retrospective analysis did not identify a selected population. Lim-ited availability of HJHS and PROBE data suggests that routine clinical practice does not yet include standardised recording of patient centred/reported outcomes, hampering the measurement of health care interventions’ value. There is a significant opportunity to improve the breadth/quality of data generation in the future to demonstrate the true impact of factor replacement in routine clinical practice.
Disclosures
Alfonso Iorio: Bayer, CSL, Pfizer, Roche, Sanofi/Sobi, Takeda; Heiko Blaser: Takeda.
View Poster: PP_26_WFH2024
