From Theory to Therapy: Evaluating Canadian Hemophilia Treatment Centres' Readiness for Gene Therapy Implementation

Introduction: Gene therapies (GT) present a transformative promise for persons with hemophilia, potentially offering a long-term solution to improve patient care and experiences. However, in publicly funded systems, GT's promise will fall short if stakeholders' preparedness, capacity, and perceptions are not proactively examined to address barriers that might delay and limit its equitable access. Therefore, this study aims to examine healthcare professionals' (HCPs) perspectives on their readiness, motivation, and capacity to implement and use GT within Canadian Hemophilia Treatment Centres (HTCs).

Methods: We conducted a cross-sectional online survey of HCPs across all Canadian HTCs, developed using the R=MC² framework for organizational change. In this framework, organizational readiness is described as a function of the motivation and collective capacity to adopt and sustain an innovation, which in this context refers to hemophilia GT. We conducted cognitive pretesting and pilot testing to examine validity. We distributed the survey through each Canadian professional organizations involved in hemophilia care. Data were analyzed using descriptive statistics for quantitative data and coding into categories for qualitative data.

Results:

A total of 45 HCPs completed the survey from 80.1% (21/26) of Canadian HTCs, with a median experience in hemophilia care of 8 years (interquartile range [IQR] 5-15). Respondents included nurses/nurse practitioners (47.7%), physicians (37.8%), physiotherapists (8.9%), social workers (4.4%), and pharmacists (2.2%). All provinces were represented with the highest participation from Ontario (40%).

Perceived Need, Benefits and Acceptance: Most respondents (76%) supported the integration of GT into the Canadian healthcare system, with a more favourable outlook for hemophilia B. Respondents identified a greater perceived need for GT in hemophilia B (76%) compared to hemophilia A (64%) and expressed higher confidence in the sufficiency of clinical evidence for hemophilia B GT (76%) versus hemophilia A (47%). Additionally, patient acceptance was perceived to be higher for hemophilia B given the current treatment landscape.

Preparedness and Resources for GT Adoption: Despite their support, respondents raised concerns about HTCs' readiness, including insufficient human resources, patient education and support mechanisms, and clinical capacity for intensive post-GT monitoring. Only 20% felt their HTC had sufficient human resources, stressing needs for more nursing and psychosocial support. Respondents felt unprepared for GT implementation, highlighting low confidence in their HTC expertise and considerable educational gaps and needs, particularly from non-physicians.

Careful Candidate Selection for GT: Suitable candidates were characterized by adherence to monitoring and treatment, minimal comorbidities specific to the risks of hepatotoxicity and/or corticosteroids related toxicities, and strong psychosocial stability or support system. Conversely, candidates with significant liver disease or metabolic health issues, poor adherence, psychological distress, and logistical barriers-such as difficulty in traveling to HTC or frequent clinic visits-were deemed less or not suitable.

HTCs Coordination and Collaboration: Traditional hub-and-spoke models might not fit Canadian context. Varying opinions were noted regarding shared and individual responsibilities between HTCs. To enhance collaboration and workflow processes, respondents highlighted the need for national guidelines on patient selection, monitoring, and adverse event management. Establishing a national GT advisory group was supported by 67% of the 14 HTC directors, reflecting the need for centralized expertise and guidance.

Conclusion: The successful integration of GT into hemophilia care in Canada requires addressing significant logistical, educational, and system-level barriers, enhancing support mechanisms and HTC resources, and ensuring careful candidate selection and coordination. Enhanced training for HCPs, clear clinical guidelines and protocols, and improved patient support mechanisms are essential to fully realize the potential of GT. Establishing a national GT advisory group and leveraging patient registries for long-term monitoring could facilitate the effective and equitable delivery of GT across Canada.

Khalife:Bayer: Honoraria; Sanofi: Honoraria; Takeda: Honoraria; Novo Nordisk: Honoraria; Roche: Honoraria; CSL Behring: Consultancy, Honoraria; Pfizer: Honoraria. Bourck:CSL Behring: Consultancy, Honoraria; Pfizer: Consultancy, Honoraria; Sanofi: Consultancy, Honoraria; Roche: Consultancy. Matino:Sobi: Honoraria, Other: Scientific Advisory Board, Speakers Bureau; Roche: Research Funding; Octapharma: Honoraria, Other: Scientific Advisory Board, Research Funding, Speakers Bureau; Spark: Research Funding; Sanofi: Honoraria, Other: Scientific Advisory Board, Research Funding, Speakers Bureau; Novo Nordisk: Honoraria, Other: Scientific Advisory Board, Research Funding, Speakers Bureau; Pfizer: Consultancy, Honoraria, Other: Scientific Advisory Board, Research Funding, Speakers Bureau; Bayer: Consultancy, Honoraria, Other: Scientific Advisory Board, Research Funding, Speakers Bureau. Decker:Takeda: Consultancy, Honoraria; Roche: Consultancy, Honoraria; Sanofi: Consultancy, Honoraria; Novo Nordisk: Consultancy, Honoraria; Pfizer: Consultancy, Honoraria; Bayer: Consultancy, Honoraria. Strike:Roche: Consultancy, Honoraria; Bayer: Consultancy, Honoraria; Sanofi: Consultancy, Honoraria; Pfizer: Consultancy, Honoraria; Novo Nordisk: Consultancy, Honoraria. Pardy:Novo Nordisk: Honoraria; Pfizer: Honoraria; Octapharma: Honoraria; Novartis: Honoraria; Bayer: Honoraria. Teitel:Pfizer: Consultancy, Other: Clinical trial investigator; Spark: Consultancy, Other: Clinical trial investigator; Bayer: Consultancy; Octopharma: Consultancy; Sanofi: Consultancy; Biomarin: Consultancy, Other: Scientific Advisory Board/Data Safety Monitoring Board; Vega Therapeutics: Consultancy, Other: Scientific Advisory Board/Data Safety Monitoring Board. Sun:CSL Behring; Pfizer; Roche; Sobi: Consultancy.