Hemophilia Gene Therapy-Key Components of Clinical Care
Hemophilia Gene Therapy-Key Components of Clinical Care
Gene Therapy in Hemophilia
AMA PRA Category 1 Credit(s)™
March 15, 2021
March 14, 2021
Estimated Time to Complete Activity
EDUCATIONAL ACTIVITY LEARNING OBJECTIVES
Upon completion of the activity, participants should be able to:
- Outline the current and emerging gene therapy approach for treating hemophilia, including various approaches to gene therapy
- Identify key characteristics of current clinical trials in gene therapy for hemophilia A and hemophilia B
- Recognize key concerns and unknowns related to the future of gene therapy for hemophilia
Michael Makris, MA, MB BS, MD, FRCP, FRCPath
Sheffield Hemophilia and Thrombosis Centre
Sheffield, United Kingdom
Wolfgang Miesbach, MD, PhD
Head, Department of Coagulation Disorders and the Comprehensive Care Haemophilia Center
Goethe University Hospital
Flora Peyvandi, MD, PhD
Professor of Internal Medicine at the University of Milan
Director of the Angelo Bianchi Bonomi Hemophilia and Thrombosis Centre
Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico
METHOD OF PARTICIPATION / HOW TO RECEIVE CREDIT
- There are no fees for participating in and receiving credit for this activity.
- Review the activity objectives and CME/CE information.
- Participate in the CME/CE activity.
- Complete the CME/CE evaluation form, which provides each participant with the opportunity to comment on how participating in the activity will affect their professional practice; the quality of the instructional process; the perception of enhanced professional effectiveness; the perception of commercial bias; and his/her views on future educational needs.
- Credit documentation/reporting:
- If you are requesting AMA PRA Category 1 Credit(s)™ or a certificate of participation—your CME/CE certificate will be available for download.
- If you are requesting MOC credit, your MOC points will be submitted electronically to the ACCME, which will register data and notify certifying boards.
This activity is jointly provided by The France Foundation, the International Society on Thrombosis and Haemostasis, and the European Association for Haemophilia and Allied Disorders.
This activity is intended for physicians (hematologists), nurse practitioners, physician assistants, and nurses managing patients with hemophilia. The activity is also intended for scientists with an interest in basic, translational, and clinical research in hemophilia around the world.
STATEMENT OF NEED
As development of gene therapy for hemophilia continues into Phase 3 clinical trials, and approval of this therapeutic approach is anticipated, it is essential for all members of the hemophilia care team to be knowledgeable and poised for the integration of this new therapeutic approach into clinical practice.
This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of The France Foundation, the International Society on Thrombosis and Haemostasis (ISTH), and the European Association for Haemophilia and Allied Disorders (EAHAD). The France Foundation is accredited by the ACCME to provide continuing medical education for physicians.
Physicians: The France Foundation designates this enduring activity for a maximum of 1.0 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
Nurses: Nurses who are certified by the American Nurses Credentialing Center (ANCC) may utilize activities that are certified by ACCME-accredited providers toward their requirement for certification renewal by the ANCC. A certificate of attendance will be provided by The France Foundation, an ACCME accredited provider.
In accordance with the ACCME Standards for Commercial Support, The France Foundation, ISTH, and EAHAD require that individuals in a position to control the content of an educational activity disclose all relevant financial relationships with any commercial interest. TFF, ISTH, and EAHAD resolve all conflicts of interest to ensure independence, objectivity, balance, and scientific rigor in all their educational programs. Furthermore, TFF, ISTH, and EAHAD seek to verify that all scientific research referred to, reported, or used in a CME/CE activity conforms to the generally accepted standards of experimental design, data collection, and analysis. TFF, ISTH, and EAHAD are committed to providing learners with high-quality CME/CE activities that promote improvements in health care and not those of a commercial interest.
Activity Staff Disclosures
The planners, reviewers, editors, staff, CME committee, or other members at The France Foundation who control content have no relevant financial relationships to disclose.
The planners, reviewers, editors, staff, CME committee, or other members at the ISTH who control content have no relevant financial relationships to disclose.
The planners, reviewers, editors, staff, CME committee, or other members at EAHAD who control content have no relevant financial relationships to disclose.
Faculty Disclosures–Activity Faculty
The faculty listed below report that they have relevant financial relationships to disclose:
- Michael Makris, MA, MB BS, MD, FRCP, FRCPath, serves as a consultant for Grifols, Freeline Therapeutics, NovoNordisk, Spark, and Takeda
- Wolfgang Miesbach, MD, PhD, received grant research support from Bayer, Biotest, CSL Behring, LFB, Novo Nordisk, Octapharma, Pfizer, and Takeda/Shire. He serves on the speakers bureau for Bayer, Biomarin, Biotest, CSL Behring, Chugai, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, and Takeda/Shire. Dr. Miesbach serves on advisory boards for Bayer, BioMarin, Biotest, CSL Behring, Chugai, Freeline, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Sanofi, Takeda/Shire, and uniQure.
- Flora Peyvandi, MD, PhD, serves as a consultant for Sanofi and Sobi. She serves on the speakers bureau for Roche, Sanofi, Sobi, and Takeda.
DISCLOSURE OF UNLABELED USE
TFF, ISTH, and EAHAD require CME faculty (speakers) to disclose when products or procedures being discussed are off label, unlabeled, experimental, and/or investigational. This includes any limitations on the information that is presented, such as data that are preliminary, or that represent ongoing research, interim analyses, and/or unsupported opinion. Faculty in this activity may discuss information about pharmaceutical agents that is outside of US Food and Drug Administration-approved labeling. This information is intended solely for continuing medical education and is not intended to promote off-label use of these medications. TFF, ISTH, and EAHAD do not recommend the use of any agent outside of the labeled indications. If you have questions, contact the Medical Affairs Department of the manufacturer for the most recent prescribing information.
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COMMERCIAL SUPPORT ACKNOWLEDGMENT
This activity is supported by educational grants from BioMarin, Spark Therapeutics, and uniQure, Inc.
TFF, ISTH, and EAHAD present this information for educational purposes only. The content is provided solely by faculty who have been selected for their recognized expertise in their field. Participants have the professional responsibility to ensure that products are prescribed and used appropriately on the basis of their own clinical judgment and accepted standards of care. The France Foundation, ISTH, EAHAD, and commercial supporter(s) assume no liability for the information herein.
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Key Components of Clinical Care
Gene therapy intends to reduce — or perhaps eliminate — the need for a regular dosage of hemophilia treatment. However, the methodology behind gene therapy is complex.
The video Hemophilia Gene Therapy - Key Components of Clinical Care is a seminar that features gene therapy researchers and education specialists in the field of hemophilia. Working with the education initiative offered by the International Society on Thrombosis and Haemostasis (ISTH), these specialists discuss the key components of clinical care associated with gene therapy for hemophilia A & B and other critical hemophilia treatment topics.
The video runs for just over 50 minutes and uses slides and commentary to discuss and explain the basics of gene therapy, including:
- The use of viral vectors
- The potential benefits of the single-dose treatment over traditional therapies
- The safety and importance of clinical trials in gene therapy
Hemophilia Gene Therapy - Key Components of Clinical Care is a tailored educational study. Well-known hematologists Professor Flora Peyvandi, Professor Michael Makris, and Professor Wolfgang Miesbach of EAHAD discuss recent updates in the clinical trial process and gene therapy models for hemophilia.
This video serves as a useful preparation tool for healthcare professionals interested in earning CME credits by learning more about the potential of gene therapy as a treatment for hemophilia.
After the lecture section of the video, the team answers questions provided during the initial session. ISTH, working with global hemophilia experts, continues to develop educational resources to improve awareness of the benefits of gene therapy and the clinical application in the treatment of hemophilia. These resources provide fundamental information regarding the future of gene therapy and the latest clinical advances in the treatment of hemophilia.