The Promise of Gene Therapy: Access and Coverage for Innovative, High-Cost Treatments
Reserve your spot today for the Satellite Dinner Symposium
The Promise of Gene Therapy: Access and Coverage for Innovative, High-Cost Treatments

Jointly provided by the National Hemophilia Foundation, Impact Education, LLC and Medical Education Resources, Inc.
Impact Education, LLC     Impact Education, LLC     Medical Education Resources
This activity is supported by BioMarin Pharmaceutical Inc., uniQure, Spark Therapeutics, Inc. and Takeda.
Tuesday, October 29, 2019
6:00 PM - 8:00 PM
Gaylord National Resort & Convention Center
Room: National Harbor 4-5
National Harbor, MD

Please note: Registration for AMCP Nexus 2019 is not required to attend this symposium.

Click here to download the slides from this symposium.

To complete an evaluation, claim credit, and submit credit to CPE Monitor visit and use the code provided at the symposium.
  Learn About
  • Unique clinical and financial considerations and potential solutions for gene therapies
  • How to ensure quality care and appropriate patient access to gene therapies
  Expert Faculty Presenters
Mari-Pat Pusey, MBA
Senior Product Director
Edmund Pezalla, MD, MPH
Enlightenment Bioconsult, LLC
Tammuella Chrisentery-Singleton, MD
Director, Hemophilia Treatment Center
Chief, Pediatric Hematology
Mississippi Center for Advanced Medicine (MCAM)
Louisiana Center for Advanced Medicine (LCAM)
Brendan Hayes
Director of External Affairs
National Hemophilia Foundation
  Who Should Attend?

This activity is designed to meet the educational needs of managed care pharmacy directors, clinical pharmacists, quality directors, medical directors, members of the AMCP, and other managed health care professionals attending the AMCP Nexus 2019 Meeting.

  Statement of Need/Program Overview

The treatment of hemophilia continues to advance and emerging products have demonstrated the potential for improved management, increased patient adherence, and enhanced outcomes. The most recent of these advancements, gene therapy, has demonstrated tremendous promise in terms of offering at least temporarily curative potential.
While the cost of gene therapy for hemophilia is anticipated to be sizably greater than that of currently prescribed agents, many opinion leaders anticipate it to be a “once in a lifetime treatment.” As a result, the anticipated high cost of gene therapy will likely be offset by the avoidance of continued factor replacement therapy and other components of health care resource utilization. Still, the prospect of a significantly larger, albeit potentially one-time drug acquisition cost presents a challenge for payers and the standard models used to cover and pay for prescription drugs. Therefore, innovators, health insurers, and policymakers have been discussing alternative payment arrangements, such as a large upfront payment followed by installments “akin to a mortgage” and outcomes-based contracting and payments. The latter approach has already been applied in other disease states with high-cost therapies for rare diseases. A key element in these discussions is the consideration of health insurance subscriber retention.

  Educational Objectives

At the conclusion of this activity, participants should be able to demonstrate improved ability to:
  • Describe the molecular and physiologic principles of gene therapy in the treatment of hemophilia
  • Review outcomes measures for clinical trials in hemophilia gene therapy and the pertinent clinical trial data for investigational treatments
  • Characterize the financial implications of gene therapy in terms of acquisition costs reconciled with the potential for improved outcomes and reduced health care service utilization
  • Outline current and proposed payment models aligned with appropriate use for high-cost therapies

Date and Time
Tuesday, October 29, 2019
6:00 PM - 8:00 PM
Gaylord National Resort & Convention Center
Room: National Harbor 4-5
National Harbor, MD

6:00 PM Pre-Activity Learning Assessment and Opening Comments/Overview
Edmund Pezalla, MD, MPH
6:05 PM Molecular and Physiologic Principles of Gene Therapy in the Treatment of Hemophilia
Tammuella Chrisentery-Singleton, MD
6:35 PM Financial Implications of Gene Therapy and the Potential for Improved Outcomes and Reduced Health Care Service Utilization
Edmund Pezalla, MD, MPH
7:00 PM Proposed Payment Models Aligned with Appropriate Use for Potentially Eligible for Hemophilia Gene Therapy
Mari-Pat Pusey, MBA
7:25 PM Patient Perspective from the National Hemophilia Foundation
Brendan Hayes
7:35 PM Audience Q&A Session
7:55 PM Key Takeaways and Closing Comments; Post-Activity Assessment and Evaluation
8:00 PM Adjournment

  Accreditation Statements

Physician Credit
This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education through the joint providership of Medical Education Resources, Inc. (MER), the National Hemophilia Foundation and Impact Education, LLC. MER is accredited by the ACCME to provide continuing medical education for physicians.

Credit Designation
Medical Education Resources, Inc. designates this live activity for a maximum of 2.0 AMA PRA Category 1 Credits. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Pharmacy Credit
Medical Education Resources, Inc. (MER) is accredited by the Accreditation Council for Pharmacy Education as a provider of continuing pharmacy education. MER designates this continuing education activity for 2.0 contact hours (0.20 CEUs) of the Accreditation Council for Pharmacy Education.

Universal Activity Number: 0816-9999-19-174-L01-P
This activity is certified as Knowledge-based CPE.

Disclosure of Conflicts of Interest
It is the policy of Medical Education Resources, Inc. to ensure balance, independence, objectivity, and scientific rigor in all of its educational activities. In accordance with this policy, MER identifies conflicts of interest with its instructors, content managers, and other individuals who are in a position to control the content of an activity. Conflicts are resolved by MER to ensure that all scientific research referred to, reported, or used in a continuing education activity conforms to the generally accepted standards of experimental design, data collection, and analysis.

Americans with Disabilities Act
♿ Event staff will be glad to assist you with any special needs (ie, physical, dietary, etc). Please contact Impact Education, LLC, prior to the live event at (215) 619-8812.

Fee Information
No additional fee is required to attend this CME/CPE activity.
Registration for AMCP Nexus 2019 is not required to attend this symposium.

Pre-registration will close at 12:00 pm on October 28, 2019.
Walk-in registration will be available at the time of the program.
If you have any questions, please call (215) 619-8812 or email