Abstract Number: PB0664
Meeting: ISTH 2021 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia Gene Therapy
Background: Life-long prophylactic treatment with factor replacement therapy is standard of care for patients with severe or moderately severe haemophilia to prevent bleeding episodes, especially haemarthroses. Gene therapy is an investigational treatment that has the potential to result in sustained endogenous levels of clotting factor within the normal range, thus offering potentially transformative benefits including a functional cure for haemophilia.
While conventional clinical metrics offer some insight into the benefits of gene therapy for patients, they can fail to account fully for more holistic aspects of health or evaluate what is most important to patients. Regulators and pharmaceutical appraisal bodies have thus issued guidance to incorporate patient preference data into development programs, which could better characterise what a ‘functional cure’ could mean to patients.
Aims: To summarise current research into haemophilia patient preferences related to emerging pharmaceutical modalities such as gene therapy.
Methods: Desktop research was undertaken to identify current literature on patient preferences in haemophilia, with particular attention to gene therapy. The focus of this search was patient preference studies such as discrete choice experiments (DCEs) and qualitative analyses to assess costs, benefits, and trade-offs associated with new therapies.
Results: There exist few published studies that explore haemophilia patient preferences. Previous studies in haemophilia using the DCE framework have reported that patients had a strong preference for infrequent dosing, non-invasive route of administration, and protection against bleeding. Only one publication cited aspects of gene therapy in the aims and attributes of the analysis.
Conclusions: An analysis of the literature confirms a need to conduct further research to identify outcomes that are most important to haemophilia patients and their preferences regarding gene therapy. Transformative innovations such as gene therapy may offer expansive benefits beyond the reach of traditional clinical outcome measures, and further research to understand how gene therapy may address patients’ needs is warranted.
To cite this abstract in AMA style:
Burke T, Asghar S, O'Hara J, Yee D, Kar S. The Importance of Patient Preferences and Other Patient-Centric Metrics in Developing and Evaluating New Haemophilia Treatments [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/the-importance-of-patient-preferences-and-other-patient-centric-metrics-in-developing-and-evaluating-new-haemophilia-treatments/. Accessed March 22, 2024.« Back to ISTH 2021 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/the-importance-of-patient-preferences-and-other-patient-centric-metrics-in-developing-and-evaluating-new-haemophilia-treatments/