Update on Efficacy of Clinical Trials
A comprehensive educational resource designed by leading experts for the global hemophilia community to help you stay abreast of the evolving science and latest clinical advancements in gene therapy in hemophilia.
Activity Title: Update on Efficacy in Clinical Trials
Topic: Gene Therapy in Hemophilia
Accreditation Type: AMA PRA Category 1 Credit(s)™
Release Date: August 7, 2020
Expiration Date: August 8, 2021
Estimated Time to Complete Activity: 30 minutes
Update on Efficacy in Clinical Trials
Gene Therapy in Hemophilia
AMA PRA Category 1 Credit(s)™
August 7, 2020
August 8, 2021
Estimated Time to Complete Activity
Upon completion of the activity, participants should be able to:
- Outline the current and emerging gene therapy approaches for treating hemophilia Identify key characteristics of current clinical trials in gene therapy for both hemophilia A and hemophilia B
- Recommend appropriate patients for ongoing and emerging trials
- Recognize key concerns and unknowns related to the future of gene therapy for hemophilia
Guy Young, MD
Director, Hemostasis and Thrombosis Program
Professor of Pediatrics (Clinical Scholar)
Keck School of Medicine of USC
Los Angeles, California
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.
- Complete the CME/CE activity
- Complete the online posttest. A score of 100% is required to successfully complete this activity. The participant may take the test until successfully passed.
- Complete the CME/CE evaluation/attestation 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 Credits™ or a certificate of participation—your CME/CE certificate will be available for download.
This activity is jointly provided by The France Foundation and the International Society on Thrombosis and Haemostasis.
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.
TThis 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 (TFF) and the International Society on Thrombosis and Haemostasis (ISTH). 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 0.50 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, TFF and the ISTH require that individuals in a position to control the content of an educational activity disclose all relevant financial relationships with any commercial interest. TFF and the ISTH resolve all conflicts of interest to ensure independence, objectivity, balance, and scientific rigor in all their educational programs. Furthermore, TFF and the ISTH 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 and the ISTH 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 reviewers, editors, staff, CME committee, or other members at The France Foundation who control content have no relevant financial relationships to disclose.
The reviewers, editors, staff, or other members at the International Society of Thrombosis and Haemostasis who control content have no relevant financial relationships to disclose.
The faculty listed below report that they have no relevant financial relationships to disclose:
- Claire McLintock, MBChB, FRACP, FRCPA
- Alok Srivastava, MD, FRACP, FRCPA, FRCP
The faculty listed below report that they have relevant financial relationships to disclose:
- David Lillicrap, MD, FRCPC, receives honoraria for academic consulting from Bioverativ, CSL Behring, and Octapharma Plasma. He receives research funding from Bayer, BioMarin, Bioverative, CSL-Behring, and Octapharma Plasma.
- Johnny Mahlangu, BSc, MBBCh, MMed, FCPath serves on the speakers bureau and does contract research for CSL Berhing, Catalyst Biosciences, Freeline Therapeutics, Novo Nordisk, Roche, Sanofi, Spark, and Takeda.
- K. John Pasi, MChB, PhD, FRCP, FRCPath, FRCPCH, receives research funding from BioMarin GeneR8 programme, uniQure HOPE-B programme, and Sanofi–ATLAS fitusiran program. He receives honoraria from Alnylam, ApcinteX, BioMarin, Catalyst Bio, Chugai, Novo Nordisk, Octapharma Plasma, Pfizer, Roche, Sanofi, Shire, and Sobi.
- Flora Peyvandi, MD, PhD, serves as a consultant for Sanofi and Sobi. She serves on the speakers bureau for Bioverative, Grifols, Roche, Sanofi, Sobi, Spark, and Takeda.
- Glenn F. Pierce, MD, PhD, receives honoraria for academic consulting from BioMarin, Genentech/Roche, Pfizer, St. Jude, and VarmX. He holds leadership positions with Global Blood Therapeutics, NHF MASAC, and World Federation of Hemophilia.
- Steven W. Pipe, MD, serves as a consultant for Apcintex, Bauer, BioMarin, Catalyst Biosciences, CSL Behring, HEMA Biologics, Freeline, Novo Nordisk, Pfizer, Roche/Genentech, Sangamo Therapeutics, Sanofi, Takeda, Spark Therapeutics, and uniQure. He does research for Siemens.
- Thierry VandenDriessche, PhD, holds leadership positions with NHF and ISTH. He receives research funding from Pfizer and Takeda. Dr. VandenDriessche receives honoraria from Baxalta, Shire, Takeda, Bayer, Biotest, and Pfizer
Faculty Disclosures–Activity Faculty
The following faculty report that they have relevant financial relationships to disclose:
- Guy Young, MD, is a member of the Speaker’s Bureau for BioMarin and Sanofi. He serves as a consultant for Genentech, Grifols, Novo Nordisk, Sanofi, Roche, Takeda and uniQure. Dr. Young does research for Genentech, Grifols, Spark and Takeda.
DISCLOSURE OF UNLABELED USE
TFF and ISTH require CME faculty (speakers) to disclose when products or procedures being discussed are off label, unlabeled, experimental, and/or investigational, and 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 U.S. 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 and ISTH 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.
COMMERCIAL SUPPORT ACKNOWLEDGMENT
This activity is supported by educational grants from BioMarin, Freeline Therapeutics Limited, Pfizer Inc, Shire, Spark Therapeutics, and uniQure, Inc.
The France Foundation and International Society of Thrombosis and Haemostasis present this information for educational purposes only. The content is provided solely by faculty who have been selected because of 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, International Society of Thrombosis and Haemostasis, and the commercial supporter(s) assume no liability for the information herein.
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