The Promise of Gene Therapy: Access and Coverage for Innovative High-Cost Treatments

• Release Date: March 25, 2020
• Expiration Date: September 30, 2021
• Estimated Time to Complete Activity: 1.0 hour
• Compatible with all modern browsers and mobile devices

EXPERT FACULTY

Tammuella Chrisentery-Singleton, MD Director, Hemophilia Treatment Center Chief, Pediatric Hematology Mississippi Center for Advanced Medicine (MCAM) Louisiana Center for Advanced Medicine (LCAM) Click here for biography Edmund Pezalla, MD, MPH CEO Enlightenment Bioconsult, LLC Click here for biography Mari-Pat Pusey, MBA Senior Product Director OptumRx Click here for biography Brendan Hayes Director of External Affairs National Hemophilia Foundation Click here for biography

Target Audience

• This activity is designed to meet the educational needs of managed care pharmacy directors, clinical pharmacists, quality directors, medical directors, registered nurses, and other managed health care professionals.

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 
After completing this activity, the participant should be better able 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

ACCREDITATION

Physician Continuing Medical Education

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 through the joint providership of Medical Education Resources (MER), 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 designates these enduring materials for a maximum of 1.0 AMA PRA Category 1 Credit^TM. Physicians should claim only the credit commensurate with the extent of their participation in the activity. 

Credit Designation
Medical Education Resources designates these continuing education activities for 1.0 contact hour (0.10 CEU) of the Accreditation Council for Pharmacy Education.
UAN: 0816-9999-20-122-H01-P.
This activity is certified as Knowledge-based CPE.

Nursing Continuing Education

Medical Education Resources is accredited as a provider of continuing nursing education by the American Nurses Credentialing Center's Commission on Accreditation. Medical Education Resources is accredited as a provider of continuing nursing education by the California Board of Registered Nursing, Provider #CEP 12299.

Credit Designation
These educational activities for 1.0 contact hour are provided by Medical Education Resources.

DISCLOSURE OF UNLABELED USE
This educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the FDA. The planners of this activity do not recommend the use of any agent outside of the labeled indications.

The opinions expressed in the educational activity are those of the faculty and do not necessarily represent the views of the planners. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.

Disclosure of Conflicts of Interest

Medical Education Resources ensures balance, independence, objectivity, and scientific rigor in all our 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. MER is committed to providing its learners with high-quality activities that promote improvements or quality in health care and not the business interest of a commercial interest.

The faculty reported the following financial relationships with commercial interests whose products or services may be mentioned in this activity:

The content managers reported the following financial relationships with commercial interests whose products or services may be mentioned in this activity:

DISCLAIMER
The content and views presented in this educational activity are those of the authors and do not necessarily reflect those of Medical Education Resources, Impact Education, LLC, and/or BioMarin Pharmaceuticals Inc., uniQure, Spark Therapeutics, and Takeda. The authors have disclosed if there is any discussion of published and/or investigational uses of agents that are not indicated by the FDA in their presentations. Before prescribing any medicine, primary references and full prescribing information should be consulted. Any procedures, medications, or other courses of diagnosis or treatment discussed or suggested in this activity should not be used by clinicians without evaluation of their patient’s conditions and possible contraindications on dangers in use, review of any applicable manufacturer’s product information, and comparison with recommendations of other authorities. The information presented in this activity is not meant to serve as a guideline for patient management.

METHOD OF PARTICIPATION AND REQUEST FOR CREDIT
There are no fees for participating and receiving CME/CE credit for this activity. During the period March 25, 2020 through September 30, 2021, participants must 1) read the learning objectives and faculty disclosures; 2) study the educational activity; 3) successfully complete the post-test with a score of 70% or better; 4) and complete the evaluation form. Upon completion of the full CME/CE activity, your certificate will be made available immediately to download and print.

For Pharmacists: Upon successfully completing the post-test with a score of 70% or better and the activity evaluation form, you may immediately submit your CPE credit to the NABP CPE Monitor Service. This may require you to add or update the e-profile ID/date of birth information saved in your account.

MEDIA
Internet

FEE INFORMATION
There is no fee for this educational activity.