Abstract

Background:Gene therapy has the potential to fundamentally alter care for patients living with hemophilia. To educate and prepare clinicians for this potential paradigm shift, the National Hemophilia Foundation (NHF), European Haemophilia Consortium (EHC), the World Federation of Hemophilia (WFH), and Medscape Education established a multinational collaboration to develop an online continuing medical education (CME) curriculum.Aims:The current study assessed the ability of online CME to improve clinicians’ knowledge regarding the latest data from ongoing trials for gene therapy in hemophilia.Methods:A 15‐minute, CME‐certified, Expert Video Commentary activity was developed and launched online on 09/25/2018. Educational effectiveness was assessed with a repeated‐pairs pre‐/post‐assessment study design, with each individual serving as his/her own control. Responses to 3 multiple‐choice, knowledge questions and 1 self‐efficacy confidence question were analyzed. A chi‐squared test assessed changes pre‐ to post‐assessment. P values <0.05 are statistically significant. Effect sizes were evaluated using Cramer's V (<0.05 modest; 0.06‐0.15 noticeable effect; 0.16‐0.26 considerable effect; >0.26 extensive effect).Results:To date, 2,747 clinicians, including 2,229 physicians, have participated in this educational activity. This analysis comprises data from the subset of hematologists/oncologists (n = 84; hem/oncs) and pediatric hematologists (n = 223) who answered all pre‐/post‐assessment questions during initial analysis period of 09/25/18 thru 11/09/18; data collection is ongoing.Both hem/oncs and pediatric hematologists exhibited significant improvements overall and in specific areas of assessment (Figure). A notable proportion of providers – 17% of hem/oncs and 35% of pediatric hematologists – expressed increased confidence with regard to their understanding of the latest safety and efficacy data for gene therapy. In addition to the positive impact of education, these findings also uncovered educational gaps, such as the need for additional education regarding vector‐related molecular biology, design, and dosing considerations. These topics may be the focus of future CME programs.Summary/Conclusion:Hematologists/oncologists and pediatric hematologists who participated in this online, Expert Video Commentary activity demonostrated significantly improved knowledge of efficacy and safety data from clinical trials in hemophilia.image

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