The Gene Therapy Transformation: A Guide for Managed Care Decision Makers Web Activity


John Petrich, RPh, MS                                     
Manager, Investigational Drug Service
Cleveland Clinic
Click here for biography

Edmund Pezalla, MD, MPH
Enlightenment Bioconsult, LLC
Click here for biography

Mari-Pat Pusey, MBA                                                   
Senior Product Director                     
Click here for biography

Target Audience

Statement of Need/Program Overview 
Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under investigation to ensure safety and efficacy. Furthermore, research in this field is particularly challenging given the inherent nature of rare diseases and genetic disorders. Approximately 30 million individuals in the United States are living with a rare disease, which by definition have a prevalence of 200,000 or fewer cases in the United States. Disease heterogeneity and geographic dispersion add to the difficulty of completing robust studies in these small populations. Given these challenges and a limited amount of resources available, gene therapy is currently in clinical trials only for diseases that have no other cures.

Genetic disorders and other rare diseases present unique challenges for health insurers in terms of requisite clinical intervention, disease management, and economic burden. The treatment landscape in US health care has already evolved significantly over the past several decades and appears to be on the precipice of its most groundbreaking innovation to date: gene therapy. This program is designed to address the educational needs for payers and managed care professionals regarding the solutions necessary to address the unique clinical and financial considerations surrounding gene therapies that will ensure quality care and appropriate patient access.

Educational Objectives 
At the conclusion of this activity, participants should be able to demonstrate improved ability to: 

  1. Explain the molecular and physiologic principles of gene therapy in the treatment of rare diseases
  2. Review outcomes measures for clinical trials in gene therapy and the pertinent clinical trial data for investigational treatments
  3. Evaluate the financial implications of gene therapy in terms of acquisition costs reconciled with the potential for improved outcomes and reduced health care service utilization
  4. Assess current and proposed payment models aligned with appropriate use for high-cost therapies


Joint Accreditation Statement

In support of improving patient care, this activity has been planned and implemented by the Postgraduate Institute for Medicine and Impact Education, LLC. Postgraduate Institute for Medicine is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.

Physician Continuing Medical Education
The Postgraduate Institute for Medicine designates this enduring material for a maximum of 1.25 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Continuing Pharmacy Education
Postgraduate Institute for Medicine designates this continuing education activity for 1.25 contact hours (0.125 CEU) of the Accreditation Council for Pharmacy Education.

Universal Activity Number: JA4008162-9999-20-2519-H01-P
Type of Activity: Knowledge

Pharmacists have up to 30 days to complete the evaluation and claim credit for participation so that information can be submitted to CPE Monitor as required.

Continuing Nursing Education

The maximum number of hours awarded for this Continuing Nursing Education activity is 1.25 contact hours.


Postgraduate Institute for Medicine (PIM) requires instructors, planners, managers and other individuals who are in a position to control the content of this activity to disclose any real or apparent conflict of interest (COI) they may have as related to the content of this activity. All identified COI are thoroughly vetted and resolved according to PIM policy.  PIM is committed to providing its learners with high quality activities and related materials that promote improvements or quality in healthcare and not a specific proprietary business interest of a commercial interest.

Name of Faculty Reported Financial Relationship
John Petrich, RPh, MS Mr. Petrich has nothing to disclose
Edmund Pezalla, MD, MPH Consulting Fees (eg. Advisory Boards): BioMarin
Mari-Pat Pusey, MBA Ms. Pusey has nothing to disclose

The PIM planners and managers have nothing to disclose.
The Impact Education, LLC® planners and managers have nothing to disclose.

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.

Participants have an implied responsibility to use the newly acquired information to enhance patient outcomes and their own professional development. The information presented in this activity is not meant to serve as a guideline for patient management. 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 and/or dangers in use, review of any applicable manufacturer’s product information, and comparison with recommendations of other authorities.

There are no fees for participating and receiving CME/CE credit for this activity. During the period of accreditation 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 75% or better; 4) and complete the evaluation form. Upon completion of the full CME/CE activity, a certificate will be made available immediately to download and print.

For Pharmacists: Upon successfully completing the post-test with a score of 75% or better and the activity evaluation, 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. Credit must be uploaded to CPE Monitor within 30 days.


There is no fee for this educational activity.