Abstract Number: PB0658
Meeting: ISTH 2021 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia Gene Therapy
Background: Evidence of patient preferences for novel hemophilia treatment such as gene therapies is limited. Understanding which treatment attributes are important for people with hemophilia A (PWHA) and B (PWHB) will help to inform the value of gene therapy.
Aims: To examine treatment preferences, including gene therapies, of PWH using a discrete choice experiment (DCE).
Methods: An online survey was conducted among US male adults with severe or moderate hemophilia, recruited from patient panels including the National Hemophilia Foundation Community Voices in Research platform. In the DCE, each participant completed 14 choice tasks selecting their preferred option from hypothetical pairs of treatment scenarios with varying attribute levels. A total of six attributes were developed based on a systematic literature review and semi-structured interviews with patient advocates and clinical experts. Responses were excluded for inconsistencies using a dominated choice task and completion time. Random parameters logit models (which assumes preference heterogeneity) were used to estimate the preference weights and the relative attribute importance (RAI) in PWHA and PWHB.
Results: The DCE was completed by 183 PWH with a mean (SD) age of 39.3 (13.8) years, with 120 PWHA and 63 PWHB. The two most important attributes were ‘Dose frequency and durability’ (RAI: PWHA 23%; PWHB 35%) and ‘Effect on overall annual bleeding rate (ABR)’ (RAI: PWHA 32%; PWHB 29%), the former being more important for PWHB. They were followed by ‘Uncertainty regarding short-term or long-term significant safety issues’ (RAI: PWHA and PWHB both 17%; all three attributes contributing to 70-80% of RAI). The attributes ‘Impact on activity of daily life/physical activity’, ‘Transformative/mental health impact’ and ‘Post treatment, possibility to undergo minor surgery without need for factor therapy’ contributed to the remaining 20-30% of RAI.
Conclusions: The results from this novel application of DCE informs the value that patients place on different aspects of gene therapies.
To cite this abstract in AMA style:
Witkop M, Morgan G, O'Hara J, Recht M, Buckner TW, Nugent D, Curtis R, O’Mahony B, Skinner MW, Mulhern B, Cawson M, Ali T, Sawyer E, Li N(. Preferences of People with Hemophilia A and B for Treatments Including Gene Therapies in the US: A Discrete Choice Experiment [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/preferences-of-people-with-hemophilia-a-and-b-for-treatments-including-gene-therapies-in-the-us-a-discrete-choice-experiment/. Accessed March 21, 2024.« Back to ISTH 2021 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/preferences-of-people-with-hemophilia-a-and-b-for-treatments-including-gene-therapies-in-the-us-a-discrete-choice-experiment/