Unwavering Priorities: Global Physician Alignment on Gene Therapy for Hemophilia
Authors: Loo Tze Yen , Saripuddin Zulisa , Cheng Boey Yee , Kyle Aine , Levent Ayse 
Published: December 2024

Background:
Gene therapy represents a novel approach to managing hemophilia, offering the potential for transformative improvements in patient outcomes. As this innovative treatment modality gains momentum, understanding the factors physicians prioritize when considering gene therapy is crucial for its successful implementation and widespread adoption. Insight into physician preferences can guide the development and refinement of gene therapy products, ensuring they align with the needs and expectations of the medical community.
Aims:
This study aimed to identify and compare the attributes physicians deem most important when selecting gene therapy as a treatment option for patients with hemophilia A and B. By elucidating these priorities across various physician subgroups, we sought to uncover any potential differences or consensus in decision-making criteria.
Methods:
The Ipsos Hemophilia Therapy Monitor, a multi-country quantitative online survey, was conducted among 259 physicians specialized in treating patients with hemophilia A and B across France (n=30), Germany (n=40), Italy (n=40), Spain (n=40), and the UK (n=26), US (n=70), and Canada (n=13). Data were collected between November 2022 and February 2023. Physicians were screened for duration of practice in their specialty and hemophilia patient caseload.
Physicians were asked to rank a list of attributes related to efficacy, safety, convenience, and access in the selection of gene therapy. We compared the top-ranked attributes across different physician subgroups to provide a nuanced analysis of the physician priorities across diverse demographics.
Results:
Our analysis revealed a consistent prioritization of efficacy and safety-related attributes in gene therapy selection among sampled physicians, regardless of region, years of experience treating hemophilia (3-10 years, 11-20 years, 21-35 years), recent clinical trial involvement in the past 6 months, and patient caseloads.
37% of the physicians - at an aggregate level - identified ‘durable factor expression’ as their top priority, while 29% emphasized the importance of ‘predictability in gene therapy outcomes’. 23% of the physicians considered ‘long-term safety data’ the most important factor in making a gene therapy decision.
Summary/Conclusion:
This study demonstrates a consensus among physicians in the regions surveyed, regarding the key attributes considered when selecting gene therapy for hemophilia. The selection of durability, predictability, and safety considerations underscore the importance of these factors in the decision-making process. These findings highlight the importance of prioritizing these attributes in gene therapy development and clinical trials to ensure the needs of both physicians and patients are met. It is also worth noting that despite a consensus being shown, proportions of sampled physicians ranking these as top attributes are consistently below 40% - work may be needed to enhance familiarity with gene therapy and its attributes. Further research using comparator data is warranted.