Best Practice™: Streamlining the Pathway to Successful Implementation of Gene Therapy in Hemophilia B – A Focus on the Hub and Spoke Model
January 30, 2023
January 30, 2024
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This activity is provided by Physicians’ Education Resource®, LLC.
This activity is supported by an educational grant from CSL Behring LLC.
- Physicians — maximum of 1.0 AMA PRA Category 1 Credits™
- Nurses — 1.0 Contact Hours
All other healthcare professionals completing this course will be issued a statement of participation.
This educational activity is directed toward hematologists, specialty nurse practitioners/physician assistants, pharmacists, and nurses who manage patients with hemophilia B.
In this online, on-demand webcast, a renowned expert in the management of patients with hemophilia B will discuss the development of gene therapy for treating this disorder. In this educational program, this expert discusses the latest data regarding clinical trials of various gene therapies and the challenges to implementing gene therapy in hemophilia treatment centers (HTCs).
Gene therapy for hemophilia B has the potential to reduce the treatment burden for patients and their care providers by eliminating the need for regular prophylaxis. Certain challenges associated with gene therapy still need to be addressed to ensure widespread therapeutic use, but this treatment offers a promising option for this patient population. HTCs have already been established to ensure multidisciplinary care for patients and to incorporate clinical specialties, emergency departments, and appropriate laboratory facilities. Overall, gene therapy for hemophilia presents a challenge to the existing structure of HTCs and requires enhanced collaboration and information sharing to maximize benefits and minimize risks. A “hub and spoke” model has been proposed to ensure time and expertise for patient counseling, informed consent, and clear demarcation of responsibility for different aspects of the procedure. This educational activity will address the evolving treatment paradigm for hemophilia B, including the role of gene therapy and ways that it may change the way in which hemophilia care is delivered.
This educational activity is an archive of the live virtual webcast held on January 25, 2023.
- Upon completion of this activity, participants should be better able to:
- Identify the role of gene therapy in the treatment paradigm for hemophilia B
- Describe models for implementation of gene therapy for hemophilia B into clinical practice
- Discuss the role of the multidisciplinary team in the management of patients receiving gene therapy for hemophilia B
Steven W. Pipe, MD
Laurence A. Boxer Research Professor of Pediatrics and Communicable Diseases
Professor of Pathology
Director, Pediatric Hemophilia and Coagulation Disorders Program
Medical Director, Special Coagulation Laboratory
University of Michigan
Ann Arbor, MI
Physicians’ Education Resource®, LLC, is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.
Physicians’ Education Resource®, LLC, designates this enduring material for a maximum of 1.0 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
Physicians’ Education Resource®, LLC, is approved by the California Board of Registered Nursing, Provider #16669, for 1.0 Contact Hours.
Faculty, Staff, and Planners’ Disclosures
The staff of Physicians’ Education Resource®, LLC, have no relevant financial relationships with ineligible entities.
- Steven W. Pipe, MD
- Consultant: ApcinteX, ASC Therapeutics, Bayer, Biomarin, CSL Behring, Freeline Therapeutics, GeneVentiv, HEMA Biologics, Novo Nordisk, Pfizer, Regeneron/Intellia, Roche/Genentech, Sanofi, Spark Therapeutics, Takeda, uniQure.
PER® mitigated all COI for faculty, staff, and planners prior to the start of this activity by using a multistep process.
Instructions for Participation and Credit
- Complete the activity (including pre- and post-activity assessments).
- Answer the evaluation questions.
- Request credit using the drop-down menu.
Course Viewing Requirements
Internet Explorer 8.0+ for Windows 2003, Vista, XP, Windows 7, Windows 8.1 and above
Google Chrome 28.0+ for Windows, Mac OS, or Linux
Mozilla Firefox 23.0+ for Windows, Mac OS, or Linux
Safari 6.0+ for Mac OSX 10.7 and above
Supported Phones & Tablets:
Android 4.0.3 and above
iPhone/iPad with iOS 6.1 or above
Off-Label Disclosure and Disclaimer
This activity may or may not discuss investigational, unapproved, or off-label use of drugs. Learners are advised to consult prescribing information for any products discussed. The information provided in this accredited activity is for continuing education purposes only and is not meant to substitute for the independent clinical judgment of a health care professional relative to diagnostic, treatment, or management options for a specific patient’s medical condition. The opinions expressed in the content are solely those of the individual faculty members and do not reflect those of PER® or any company that provided commercial support for this activity.