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

ABSTRACT:

Introduction

Gene therapy has shown promise in clinical trials for patients with haemophilia, but patient preference studies have focused on factor replacement treatments.

Aim

We conducted a discrete choice experiment (DCE) to investigate the relative importance and differential preferences patients provide for gene therapy attributes.

Methods

We surveyed male adults with haemophilia in the United States recruited from patient panels including the National Hemophilia Foundation Community Voices in Research platform using an online survey over 4 months in 2020/21. Participants indicated preferences for gene therapy attributes including dosing frequency/durability, effect on annual bleeding, uncertainty related to side effects, impact on daily activities, impact on ment

SUBMITTER: Witkop M 

PROVIDER: S-EPMC9290457 | biostudies-literature | 2021 Sep

REPOSITORIES: biostudies-literature