Slide 1: Perceptions, needs and expectations of patients with hemophilia toward gene therapy
Perceptions, needs and expectations of patients with hemophilia toward gene therapy Laurent Frenzel1, Céline Chenuel2, Amandine Celli3, Nathalie Grinda4, Rémi Varin5, Nicolas Giraud6, Geneviève Pietu6, Therese Backus7, Christian Schepperle8, Angelo Lupi9, Kirian Legendre10, Sanja Njegic10 1Hôpital N
Slide 2: Disclosures
Disclosures Laurent Frenzel ▪ Consulting or advisory role: BioMarin, CSL Behring, Pfizer, Roche 2
Slide 3: Gene therapy for hemophilia
Gene therapy for hemophilia ▪ GT has recently been approved for treatment Mechanism of gene therapy for hemophilia6 of adults with severe hemophilia A and B1,2 ▪ GT for hemophilia uses an AAV vector to deliver a functional copy of F8 (hemophilia A) or F9 (hemophilia B) to hepatocytes, via a single i
Slide 4: Methodology and sample composition
Methodology and sample composition A quantitative online survey concerning Sweden hemophilia management and GT was conducted 36 patients Fieldwork conducted from ▪ Participants (n=139) were males (aged ≥18 years) 23 Nov 2022 to 10 Mar 2023 diagnosed with severe hemophilia ▪ The survey was distribute
Slide 5: Study objectives
Study objectives To understand the perceptions and expectations of patients with severe hemophilia towards GT To analyze patient reactions to gene therapy and understand triggers and barriers to use To determine the necessary support required for shared decision making on GT treatment GT, gene thera
Slide 6: Participant demographics
Variable N=139 Participant Age, n (%) 18–35 years 36–65 years 49 (35) 83 (60) demographics ≥66 years France 8 (6) 61 (45) Germany 33 (24) Country, n (%) Italy 9 (7) Sweden 36 (27) Urban area 67 (48) Suburban area 14 (10) Living environment, n (%) Small town area 26 (19) Rural area 32 (23) <10 km 32
Slide 7: Almost all patients managed well or very well with their hemophilia
Almost all patients n=139 n=61 n=33 n=9 n=36 managed well 1% 6% 3% 3% 6% or very well with 12% their hemophilia I always feel overwhelmed by 31% 33% 31% my hemophilia daily 35% I often feel overwhelmed by my hemophilia 48% Most of the time I manage my hemophilia well but sometimes I feel overwhelmed
Slide 8: Over the fortnight prior to survey completion, most patients felt positive
Over the fortnight n=139 n=61 n=33 n=9 n=36 prior to survey Positive feelings 78% 79% 76% 89% 78% completion, Negative feelings 37% 41% 36% 11% 39% most patients felt positive about Feeling good about myself 60% 62% 64% 33% 58% their condition Optimistic 30% 25% 36% 33% 33% Healthy 27% 30% 24% 44% 2
Slide 9: Joint pain, joint constraints, decreasing mobility had the greatest burden
Joint pain, n=139 n=61 n=33 n=9 n=36 joint constraints, Joint pain 72% 74% 76% 78% 64% decreasing Difficulties in making movement and certain moves (joint constraints) 68% 67% 70% 56% 69% mobility difficulties Decreasing of movement amplitude 62% 74% 39% 89% 56% had the greatest Difficulty to walk /
Slide 10: Hematologists, online medical articles and patient associations preferred sources
Hematologists, n=33 n=13 n=9 n=8 n=3 online medical During consultations 88% 100% 89% 75% 67% articles and patient with hematologist associations were On the internet, articles from physicians or on CT 79% 85% 89% 50% 100% the preferred Patient associations (websites or in person) 70% 69% 44% 88% 10
Slide 11: Approximately 40% of patients would accept a new treatment on recommendation
Approximately 40% n=139 n=61 n=33 n=9 n=36 of patients would Attitude toward a new treatment have accepted a I always accept 11% new treatment when my 24% hematologist based on their recommends it 39% 39% 51% hematologist’s I prefer to do some research first 12% 44% recommendation or talk about it w
Slide 12: Despite high GT awareness, a significant unmet need for education
Despite a high level n=139 n=61 n=33 n=9 n=36 of GT awareness, Awareness of gene therapy a significant unmet need for patient Yes 97% 97% 94% 100% 100% education was No 3% 3% 6% identified I know enough about gene therapy to feel Self-evaluation of knowledge about gene therapy comfortable to talk ab
Slide 13: Hematologists were the main source of support for treatment decisions
Hematologists were n=139 n=61 n=33 n=9 n=36 the main source My hematologist of support to during consultations 83% 88% 74% 67% 86% aid treatment Patient Associations 36% 34% 39% 67% 31% decision-making Other patients when 27% 31% 26% 22% 25% discussing face to face My relatives 24% 34% 19% 0% 19% Th
Slide 14: Most patients would use GT even if frequent hospital follow-ups required
Most patients would Likelihood to use gene therapy considering it might require frequent hospital follow-up Certainly Probably Not sure Probably Certainly use GT for would not use (<10% chances) would not use (10–30% chances) whether would use (31–50% chances) would use (51–80% chances) would use (>
Slide 15: Safety and tolerability information were top priorities for patients
Information on n=139 n=61 n=33 n=9 n=36 safety and More information on 64% 59% 76% 56% 64% tolerability were safety (long-term risks) More information on top priorities for tolerability (importance of SE on your body) 61% 61% 67% 56% 58% patients considering More information on duration of efficacy
Slide 16: Conclusions
Conclusions This study provides While most were open-minded to GT, Most patients believed valuable insights into an unmet need for additional information on the benefits of GT for the perspectives this innovative therapy was identified hemophilia outweigh and expectations of the inconvenience of adu
Slide 17: Acknowledgements
Acknowledgements ▪ The authors would like to thank the individuals who participated in the study Download this presentation This study was sponsored by BioMarin Pharmaceutical Inc. Medical writing support in the form of preparation and revision of the presentation was provided by Ben Drever and Elea