Patient Preferences to Assess Value IN Gene Therapies: Protocol Development for the PAVING Study in Hemophilia

Abstract

Introduction: Gene therapies are innovative therapies that are increasingly being developed. However, health technology assessment (HTA) and payer decision making on these therapies is impeded by uncertainties, especially regarding long-term outcomes. Through measuring patient preferences regarding gene therapies, the importance of unique elements that go beyond health gain can be quantified and inform value assessments. We designed a study, namely the Patient preferences to Assess Value IN Gene therapies (PAVING) study, that can inform HTA and payers by investigating trade-offs that adult Belgian hemophilia A and B patients are willing to make when asked to choose between a standard of care and gene therapy.Methods and Analysis: An eight-step approach was taken to establish the protocol for this study: (1) stated preference method selection, (2) initial attributes identification, (3) stakeholder (HTA and payer) needs identification, (4) patient relevant attributes and information needs identification, (5) level identification and choice task construction, (6) educational tool design, (7) survey integration, and (8) piloting and pretesting. In the end, a threshold technique survey was designed using the attributes “Annual bleeding rate,” “Chance to stop prophylaxis,” “Time that side effects have been studied,” and “Quality of Life.”Ethics and Dissemination: The Medical Ethics Committee of UZ KU Leuven/Research approved the study. Results from the study will be presented to stakeholders and patients at conferences and in peer-reviewed journals. We hope that results from the PAVING study can inform decision makers on the acceptability of uncertainties and the value of gene therapies to patients.

Patient preferences for gene therapy in haemophilia: Results from the PAVING threshold technique survey

“…The TIME levels (30 years for PFRT and 10 years for gene therapy) were held constant throughout the questions. As knowledge of participants on gene therapy was expected to be limited and hypothesized to influence acceptance of gene therapy, an educational tool informed participants on gene therapy before they were asked threshold technique questions 25 . The design process was supported by stakeholder and patient involvement.…”

“…The protocol for this study has been published elsewhere 25 and described an eight‐step approach that was taken in the survey design. The threshold technique 26 was chosen as preference elicitation method.…”

“…We hypothesized that both attributes ABR and QOL could be included in the survey as PWH with the same QOL could have different ABR and vice versa, and this was proven by the results of this survey. 25 Based on the sample characteristics and population data, we believe that our sample was representative to the Belgian PWH population eligible for gene therapy treatment. Often, patient preference studies have limitations relating to recruitment through panels and patient organizations, and self-reported diagnosis.…”

Patient preferences and priorities for haemophilia gene therapy in the US: A discrete choice experiment

“…To date, patient preference studies in haemophilia have focused on the attributes of factor replacement therapies, with preferences predominantly related to efficacy, inhibitor development, and treatment administration 14–16 . Few studies have attempted to quantify patient preferences related to gene therapy attributes, and none of those exploring this emerging area have applied a discrete choice experiment design among patients with moderate or severe haemophilia A or B 17,18 …”

“… 14 , 15 , 16 Few studies have attempted to quantify patient preferences related to gene therapy attributes, and none of those exploring this emerging area have applied a discrete choice experiment design among patients with moderate or severe haemophilia A or B. 17 , 18 …”