Clinicians and nurses who treat patients with hemophilia with a gene therapy identified important factors and considerations needed in a potential shared decision making tool.
The article was originally published on CGTLive®.
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, RN, 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.
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 one.
“Data from this study demonstrates the need for provider education to increase confidence in discussing [gene therapy] 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.”
Reference
Limjoco J, Thornburg CD. Development of a haemophilia A gene therapy shared decision-making tool for clinicians. Haemophilia. 2023; 00-00. doi:10.1111/hae.14822
Joanne Mizell: Lifestyle Modification Programs Take Holistic Aim at Metabolic Disease
May 1st 2024Joanne Mizell shares insurer strategies in addressing the escalating rates of metabolic diseases, highlighting the importance of holistic treatment methods like lifestyle modification programs, which integrate nutrition, physical activity, and community engagement.
Read More
Navigating Health Policy in an Election Year: Insights From Dr Dennis Scanlon
April 2nd 2024On this episode of Managed Care Cast, we're talking with Dennis Scanlon, PhD, the editor in chief of The American Journal of Accountable Care®, about prior authorization, price transparency, the impact of health policy on the upcoming election, and more.
Listen
BRCA-Like Classification May Be a Useful Biomarker for Olaparib Response in Ovarian Cancer
May 1st 2024Adding olaparib to maintenance therapy with bevacizumab was associated with significantly longer survival for patients with ovarian cancer whose tumors have a BRCA-like genomic profile, but not among those with non-BRCA-like tumors, a study found.
Read More
Exploring Medicare Advantage Prior Authorization Variations
March 26th 2024On this episode of Managed Care Cast, we're talking with the authors of a study published in the March 2024 issue of The American Journal of Managed Care® about their findings on variations in prior authorization use across Medicare Advantage plans.
Listen
AUA to Focus on Inclusive Care, Robotic Surgeries, and Future of Urology at 2024 Annual Meeting
May 1st 2024The American Urological Association (AUA) 2024 Annual Meeting will highlight the latest innovations and future trends in urology, featuring dynamic plenary sessions, educational opportunities for providers, and discussions on cutting-edge treatments and global health initiatives.
Read More
OptumRx’s Jon Mahrt Discusses “Irresponsible” Drug Pricing for Products With Multiple Indications
April 30th 2024When the same product comes to market with additional indications, irrational pricing decisions result in ever increasing prices instead of volume translating to lower costs, said Jon Mahrt, MBA, of OptumRX.
Read More
Joanne Mizell: Lifestyle Modification Programs Take Holistic Aim at Metabolic Disease
May 1st 2024Joanne Mizell shares insurer strategies in addressing the escalating rates of metabolic diseases, highlighting the importance of holistic treatment methods like lifestyle modification programs, which integrate nutrition, physical activity, and community engagement.
Read More
Navigating Health Policy in an Election Year: Insights From Dr Dennis Scanlon
April 2nd 2024On this episode of Managed Care Cast, we're talking with Dennis Scanlon, PhD, the editor in chief of The American Journal of Accountable Care®, about prior authorization, price transparency, the impact of health policy on the upcoming election, and more.
Listen
BRCA-Like Classification May Be a Useful Biomarker for Olaparib Response in Ovarian Cancer
May 1st 2024Adding olaparib to maintenance therapy with bevacizumab was associated with significantly longer survival for patients with ovarian cancer whose tumors have a BRCA-like genomic profile, but not among those with non-BRCA-like tumors, a study found.
Read More
Exploring Medicare Advantage Prior Authorization Variations
March 26th 2024On this episode of Managed Care Cast, we're talking with the authors of a study published in the March 2024 issue of The American Journal of Managed Care® about their findings on variations in prior authorization use across Medicare Advantage plans.
Listen
AUA to Focus on Inclusive Care, Robotic Surgeries, and Future of Urology at 2024 Annual Meeting
May 1st 2024The American Urological Association (AUA) 2024 Annual Meeting will highlight the latest innovations and future trends in urology, featuring dynamic plenary sessions, educational opportunities for providers, and discussions on cutting-edge treatments and global health initiatives.
Read More
OptumRx’s Jon Mahrt Discusses “Irresponsible” Drug Pricing for Products With Multiple Indications
April 30th 2024When the same product comes to market with additional indications, irrational pricing decisions result in ever increasing prices instead of volume translating to lower costs, said Jon Mahrt, MBA, of OptumRX.
Read More
2 Commerce Drive
Cranbury, NJ 08512