Patient perspectives regarding gene therapy in haemophilia: Interviews from the PAVING study

Abstract

Introduction Exploring patient perceptions regarding gene therapies may provide insights about their acceptability to patients. Objective To investigate opinions of people with haemophilia (PWH) regarding gene therapies. Moreover, this study aimed to identify patient‐relevant attributes (treatment features) that influence PWH’s treatment choices. Methods Semi‐structured individual interviews were conducted with Belgian PWH, types A and B. A predefined interview guide included information sections and open, attribute ranking and case questions. Qualitative data were organized using NVivo 12 and analysed following framework analysis. Sum totals of scores obtained in the ranking exercise were calculated per attribute. Results In total, 20 PWH participated in the interviews. Most participants demonstrated a positive attitude towards 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 PWH 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 participants were concerned about the uncertainty regarding long‐term safety, most participants were less concerned about uncertainty regarding long‐term efficacy. Conclusions This qualitative study showed that most PWH have a positive attitude towards 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 haemophilia. Moreover, they may inform clinical trial design, pay‐for‐performance schemes and real‐world evidence studies.

An exploration of why men with severe haemophilia might not want gene therapy: The exigency study

“…Qualitative studies have begun to explore the reasons why PwH might consider gene therapy 1,8,9,10,11 . They show that while individuals are concerned about the reliability of gene expression, length of expression and safety, most remain willing to consider gene therapy a potential treatment option.…”

Patient selection for hemophilia gene therapy: Real‐life data from a single center

“…The PAVING study that was conducted in 2019 and involved a small Belgian patient sample (n = 20) demonstrated a more positive attitude of individuals with hemophilia toward gene therapy. This study, however, may have been subject to a sampling bias, and thus its participants may not be representative of all Belgian people with hemophilia 14 …”

“…This study, however, may have been subject to a sampling bias, and thus its participants may not be representative of all Belgian people with hemophilia. 14 Upon follow-up visits, at patient meetings, and as part of a comprehensive approach to hemophilia care, the hematologists should…”

Patients’ Perception of the Impact of Innovation on Hemophilia Care Management Organization: A Qualitative Study Protocol (INNOVHEMO Study)

“…These studies highlighted acceptability criteria involved in shared decision making, such as treatment ease of use, efficacy on bleeds, safety and impact on daily life. 45 , 46 Another study underlined the importance for patients to communicate with a clinical team from an expert center in hemophilia to implement a new patient-centered care approach. 47 This confirms the relevance to explore patient perceptions regarding the impact of new therapies on care organization.…”