van Overbeeke E, Michelsen S, Hauber B, Peerlinck K, Hermans C, Lambert C, Goldman M, Simoens S, Huys I. Patient perspectives regarding gene therapy in haemophilia: interviews from the PAVING study. Haemophilia. 2020 Nov 7. doi: 10.1111/hae.14190.

INTRODUCTION: Exploring patient perceptions regarding gene therapies may provide insights about their acceptability to patients.

OBJECTIVES: To investigate the opinions of hemophilia patients regarding gene therapies. Moreover, this study aimed to identify patient-relevant attributes (treatment features) that influence patients’ treatment choices.

METHODS: Semi-structured individual interviews were conducted with Belgian hemophilia A and B patients. A predefined interview guide included information sections and open, attribute ranking and case questions. Interview transcripts were analyzed using NVivo 12 following framework analysis. Sum totals of scores obtained in the ranking exercise were calculated per attribute.

RESULTS: In total 20 hemophilia patients participated in the interviews. Most patients demonstrated a positive attitude toward gene therapy and were very willing (40%; n=8) or willing (35%; n=7) to receive this treatment. The following five attributes were identified as most important to patients in making their choice: annual bleeding rate, factor level, uncertainty of long-term risks, impact on daily life, and probability that prophylaxis can be stopped. While patients were concerned about the uncertainty regarding long-term safety, most patients were less concerned about uncertainty regarding long-term efficacy.

CONCLUSIONS: This qualitative study showed that most hemophilia patients have a positive attitude toward gene therapy and that besides efficacy, safety and the related uncertainties, also impact on daily life is important to patients. The identified patient-relevant attributes may be used by regulators, health technology assessment bodies and payers in their evaluation of gene therapies for hemophilia. Moreover, they may inform clinical trial design, pay-for-performance schemes and real-world evidence studies.

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