Physician Perception of Patient Unmet Needs and Optimism Towards Gene Therapy: Results From a Multinational Real-World Survey

OBJECTIVES: This study explores physician perceived unmet needs among their consulting patients and their optimism towards gene therapy.

METHODS: Data were drawn from the Adelphi Haemophilia III Disease Specific Programme™, a cross-sectional survey with retrospective data collection of physicians of male haemophilia A and B patients in France, Germany, Italy, Spain, and the United Kingdom from September 2023 to April 2024. Physicians provided data on attitudes towards gene therapy, patient demographics, patient risk of mortality, and quality of life. Analyses were descriptive.

RESULTS: Overall, 234 physicians provided attitudinal data, 74% were looking forward to offering gene therapy (LF) to patients and 24% were not looking forward to offering gene therapy (NLF). 1078 haemophilia patients were enrolled, 77% haemophilia A and 23% haemophilia B. Mean (standard deviation) age was 27.91 (15.53) years (LF: 28.26 [14.58], NLF: 27.15 [17.43]), of those prescribed treatment, 69% were from physicians LF and 31% physicians NLF.

Physicians LF reported 50% of consulting patients were suitable candidates for gene therapy, compared to 39% from physicians NLF. Physicians LF and NLF reported 9% and 2% of their patients respectively requested gene therapy. Physicians LF reported 75% of their patients had increased risk of mortality due to haemophilia, compared to 41% reported from physicians NLF. Physicians LF reported 43% of patients had difficulty with activities due to haemophilia, in comparison to 28% of patients from physicians NLF. Physicians LF and NLF reported 30% and 20% patients respectively had difficulty taking part in sports/hobbies due to haemophilia.

CONCLUSIONS: More physicians LF to offering gene therapy indicated a higher proportion of their patients are impacted by their haemophilia. Physician perception of unmet need may contribute towards optimism for prescribing gene therapy. Recognition of patients’ unmet needs may help foster productive shared-decision making, facilitating identification of appropriate gene therapy candidates.