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

SUBMITTER: van Overbeeke E 

PROVIDER: S-EPMC7985056 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature