The PAVING II Interview Study: Assessing Evolving Patient Preferences Regarding Gene Therapy for Hemophilia Patients
Author(s)
Jolien Broekmans, MSc1, Quentin Van Thillo, MD, PhD2, Brett Hauber, PhD3, Peter Sinnaeve, MD, PhD2, Tom Adriaenssens, MD, PhD2, Peter Verhamme, MD, PhD2, Isabelle Huys, PharmD, PhD1.
1Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium, 2Department of Cardiovascular Diseases, University Hospitals Leuven, Leuven, Belgium, 3Pfizer, New York, NY, USA.
1Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium, 2Department of Cardiovascular Diseases, University Hospitals Leuven, Leuven, Belgium, 3Pfizer, New York, NY, USA.
Presentation Documents
OBJECTIVES: This patient preference study (PPS) aims to assess the evolution of patient preferences regarding gene therapy (GT) since European Medicines Agency (EMA) approval. Current preferences are compared to those from the 2020 PAVING I.
METHODS: An interview study was conducted between January and July 2025 with Belgian haemophilia patients, some of whom had already received GT. A semi-structured interview guide was used with questions on patient opinions and expectations, a rating exercise with twenty attributes, and case discussions. Interviews were thematically analysed and rating scores were calculated by summing participants’ ratings per attribute.
RESULTS: Haemophilia patients’ opinion in this study was that GT is a promising treatment option. However, many expressed hesitation due to uncertainty regarding long-term outcomes, which was reinforced by past experiences with liver infections and satisfaction with current prophylactic treatment. Some felt that GT may be especially beneficial for younger patients, primarily due to its potential to prevent joint damage. Compared to the PAVING I study, a smaller proportion of patients in the current study were very willing, and a larger proportion of patients were willing or neutral to undergo GT. In the rating exercise the top five attributes were annual bleeding rate, demonstrated duration of effect, normal factor level, probability to stop prophylaxis, and impact on daily life. Notably, demonstrated duration of effect was not among the top ten attributes in the PAVING I study, indicating a shift in priorities. In the current study, attributes like route of administration and mechanism of action were rated lower, while others, such as participation in physical activity, were rated higher than in the PAVING I study.
CONCLUSIONS: These preliminary findings reflect a shift in patients’ priorities for attributes of GT since its market authorisation. The result of this study will inform the development of attributes and levels for a quantitative PPS.
METHODS: An interview study was conducted between January and July 2025 with Belgian haemophilia patients, some of whom had already received GT. A semi-structured interview guide was used with questions on patient opinions and expectations, a rating exercise with twenty attributes, and case discussions. Interviews were thematically analysed and rating scores were calculated by summing participants’ ratings per attribute.
RESULTS: Haemophilia patients’ opinion in this study was that GT is a promising treatment option. However, many expressed hesitation due to uncertainty regarding long-term outcomes, which was reinforced by past experiences with liver infections and satisfaction with current prophylactic treatment. Some felt that GT may be especially beneficial for younger patients, primarily due to its potential to prevent joint damage. Compared to the PAVING I study, a smaller proportion of patients in the current study were very willing, and a larger proportion of patients were willing or neutral to undergo GT. In the rating exercise the top five attributes were annual bleeding rate, demonstrated duration of effect, normal factor level, probability to stop prophylaxis, and impact on daily life. Notably, demonstrated duration of effect was not among the top ten attributes in the PAVING I study, indicating a shift in priorities. In the current study, attributes like route of administration and mechanism of action were rated lower, while others, such as participation in physical activity, were rated higher than in the PAVING I study.
CONCLUSIONS: These preliminary findings reflect a shift in patients’ priorities for attributes of GT since its market authorisation. The result of this study will inform the development of attributes and levels for a quantitative PPS.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR238
Topic
Patient-Centered Research
Disease
Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)