Hemophilia Gene Therapy Shared Decision Making Tool Needed, Providers Say


Surveyed clinicians and nurses from Rady Children's Hospital San Diego identified important factors and considerations needed in a potential SMD tool.

A new paper published in Haemophilia has outlined the need for a shared decision making (SMD) tool for clinicians treating patients with gene therapy for hemophilia, and has proposed a tool to be refined and evaluated in clinical practice and real-world experiences.

“A clinician-oriented SDM tool may prepare clinicians to talk about gene therapy with persons with hemophilia and provide a systematic approach to the discussion,” study authors Jacqueline Limjoco, and Courtney D. Thornburg, MD, both from Hemophilia and Thrombosis Treatment Center, Rady Children's Hospital San Diego, wrote in the paper. “This study was designed to inform the development of a clinician SDM tool by understanding clinician experience with SDM and key gene therapy attributes to include in the tool.

The paper focused on decision making specifically for patients with hemophilia A being treated with valoctocogene roxaparvovec (val-rox; BioMarin), approved under the name Roctavian. Limjoco and Thornburg surveyed 10 clinicians and 2 nurses from the Hemophilia and Thrombosis Treatment Center that had 1 to 27 years of experience caring for patients with hemophilia. The participants had no (n = 1), slight (n = 3), moderate (n = 5) and high (n = 1) confidence in having a clinical discussion about gene therapy and all agreed that an SDM tool would be useful for clinical practice. The participants gave feedback on language and presentation, content, and implementation of a potential tool, which should provide unbiased information and have companion tools with patient-centric language.

READ MORE: FDA Accepts BLA Filing for Hemophilia B Gene Therapy Fidanacogene Elaparvovec

Regarding language and presentation, participants stressed the importance of specified definitions of terms for different types of bleeds or adverse events, which can be further simplified for patients. In terms of actual content of the tool, the participants identified the need for guidance concerning the process of gene therapy, including post-infusion follow-up, the safety, including need for immune suppression, the efficacy, including durability of response, as well as psychosocial impact, impact on family planning and alcohol consumption, and other factors including insurance coverage and other treatment options. The participants thought that the tool should be unbiased and able to be updated. Another consideration that remains to be refined is whether the SDM tool should be split into 2 parts between provider and patient or combined into 1.

“Data from this study demonstrates the need for provider education to increase confidence in discussing GT and answering questions about safety and efficacy. This is like prior findings from provider surveys,” Limjoco and Thornburg wrote. “The tool includes resources for gene therapy education available through the National Hemophilia Foundation, the International Society of Thrombosis and Hemostasis, the World Federation of Hemophilia, etc. Participants are familiar with SDM but did not identify any specific tools to guide discussion of gene therapy. With the first approvals of hemophilia gene therapy, these tools are urgently needed.”

Limjoco J, Thornburg CD. Development of a haemophilia A gene therapy shared decision-making tool for clinicians. Haemophilia. 2023; 00-00. https://doi.org/10.1111/hae.14822
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