CME Information

EDUCATIONAL ACTIVITY LEARNING OBJECTIVES
Upon completion of the activity, participants should be able to:

• Describe gene therapy correctly, including basic terms and concepts
• Discuss up-to-date information on the current and emerging approaches for treating hemophilia, including various approaches of gene therapy
• Recommend appropriate patients for ongoing and emerging trials
• Appropriately discuss pros and cons of gene therapy with patients and caregivers, including available resources for further education
• Anticipate short- and long-term monitoring requirements
• Recognize modifications to multidisciplinary care approach necessary for gene therapy
• Identify potential modifications to health care systems needed to enable successful integration of gene therapy

FACULTY
Annette von Drygalski, MD, PharmD
University of California San Diego
La Jolla, California

Guy Young, MD
Keck School of Medicine of USC
Los Angeles, California

PLANNERS
David Lillicrap, MD, FRCPC
Queen’s University
Kingston, Canada

Johnny Mahlangu, BSc, MBBCh, MMed, FCPath
Faculty of the Health Sciences–University of the Witwatersrand and National Health Laboratory Service
Johannesburg, South Africa

Wolfgang A. Miesbach, MD, PhD
Goethe University Hospital
Frankurt/Main, Germany

K. Margareth C. Ozelo, MD, PhD
University of Campinas
Hemocentro UNICAMP, Brazil
Campinas, São Paulo, Brazil

Glenn F. Pierce, MD, PhD
World Federation of Hemophilia (WFH)
United States of America

Steven W. Pipe, MD
University of Michigan
Ann Arbor, Michigan

Flora Peyvandi, MD, PhD
University of Milan
Milan, Italy

Alok Srivastava, MD, FRACP, FRCPA, FRCP
Christian Medical College
Vellore, India

Thierry VandenDriessche, PhD
Vrije University Brussels
Brussels, Belgium

METHOD OF PARTICIPATION / HOW TO RECEIVE CREDIT

1. There are no fees for participating in and receiving credit for this activity.
2. Review the activity objectives and CME/CE information.
3. Participate in the CME/CE activity.
4. 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.
5. 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.

ACCREDITED PROVIDER
This activity is jointly provided by The France Foundation and the International Society on Thrombosis and Haemostasis.

TARGET AUDIENCE
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.

ACCREDITATION STATEMENT
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 (TFF) and the International Society on Thrombosis and Haemostasis (ISTH). TFF is accredited by the ACCME to provide continuing medical education for physicians.

CREDIT DESIGNATION
Physicians: The France Foundation designates this enduring activity for a maximum of 1.5 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Nurses: Nurses 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 TFF, an ACCME accredited provider.

DISCLOSURE POLICY
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 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.

Faculty Disclosures–Planners

The faculty listed below report that they have no relevant financial relationships to disclose:

• 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, Bioverativ, CSL Behring, and Octapharma Plasma.
• Johnny Mahlangu, BSc, MBBCh, MMed, FCPath, serves on the speakers bureau and does contract research for CSL Behring, Catalyst Biosciences, Freeline Therapeutics, Novo Nordisk, Roche, Sanofi, Spark, and Takeda
• Wolfgang Miesbach, MD, receives 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 also serves on advisory boards for Bayer, BioMarin, Biotest, CSL Behring, Chugai, Freeline, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Sanofi, Takeda/Shire, and uniQure.
• Margareth Ozelo, MD, PhD, serves as consultant for BioMarin, Novo Nordisk, Pfizer, Roche, Sanofi, and Takeda. She serves on the speakers bureau for Bayer, BioMarin, Novo Nordisk, Roche, and Takeda. Dr. Ozelo also conducts contract research for BioMarin, Novo Nordisk, Pfizer, Roche, Sanofi, Spark, and Takeda.
• Flora Peyvandi, MD, PhD, serves as a consultant for Sanofi and Sobi. She serves on the speakers bureau for Bioverativ, 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, Bayer, 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 also receives honoraria from Baxalta, Shire, Takeda, Bayer, Biotest, and Pfizer.

Faculty Disclosures–Activity Faculty

The faculty listed below report that they have relevant financial relationships to disclose:

• Annette von Drygalski, MD, PharmD, serves as a consultant and speaker for Bayer, BioMarin, Bioverativ/Sanofi-Genzyme, CSL Behring, Novo Nordisk, Pfizer, Regeneron, Takeda and uniQure.
• 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 also does research for Genentech, Grifols, Spark and Takeda.

All conflicts of interest disclosures for the faculty presenter and planners were mitigated.

DISCLOSURE OF UNLABELED USE
TFF and the ISTH 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 and the 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, Spark Therapeutics, and uniQure, Inc.

DISCLAIMER
TFF and the ISTH 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. TFF, the ISTH, and the commercial supporter(s) assume no liability for the information herein.

COPYRIGHT INFORMATION
Copyright © 2021 The France Foundation. Any unauthorized use of any materials on the site may violate copyright, trademark, and other laws. You may view, copy, and download information or software ("Materials") found on the Site subject to the following terms, conditions, and exceptions:

• The materials are to be used solely for personal, noncommercial, informational and educational purposes. The materials are not to be modified. They are to be distributed in the format provided with the source clearly identified. The copyright information or other proprietary notices may not be removed, changed, or altered.
• Materials may not be published, uploaded, posted, transmitted (other than as set forth herein), without The France Foundation's prior written permission.

PRIVACY POLICY
The France Foundation protects the privacy of personal and other information regarding participants and educational collaborators. The France Foundation will not release personally identifiable information to a third party without the individual's consent, except such information as is required for reporting purposes to the ACCME.

The France Foundation maintains physical, electronic, and procedural safeguards that comply with federal regulations to protect against the loss, misuse or alteration of information that we have collected from you.

Additional information regarding The France Foundation's Privacy Policy can be viewed at www.francefoundation.com/privacy-policy .

CONTACT INFORMATION
If you have questions about this CME activity, please contact The France Foundation at 860-434-1650 or This email address is being protected from spambots. You need JavaScript enabled to view it..