Gene Therapy for Hemophilia: Hope for a Cure? - Annual Meeting 2018

ACPE UAN #:  0233-0000-18-006-H01-P

Release DateApril 23, 2018

Expiration Date:  April 23, 2021

Activity Type:    Knowledge-based

Contact Hours:  0.75

Activity Fee:  Member:  $15        Non-Member: $30

Target Audience: Pharmacists who practice in managed care settings.

Activity Description

The best currently available treatments for hemophilia reduce but do not eliminate the possibility of bleeding. In contrast, gene therapy— the administration of genetic material to modify or manipulate the expression of a gene product or to alter the biological properties of living cells for therapeutic use—offers the potential for a definitive cure. A licensed gene therapy product for hemophilia could be commercially available within the next decade. How would such a product change the treatment paradigm for hemophilia? This session will discuss the promise and possible limitations of hemophilia gene therapy and introduce pharmacoeconomic model payers can use to make decisions on coverage management.

Learning Objectives:  

At the completion of this activity, participants should be able to:

1. Explain how gene therapy would be used to treat hemophilia.
2. Recognize challenges associated with the use of gene therapy for hemophilia.
3. Discuss the clinical and financial impact of gene therapy for hemophilia.
4. Explain how the management of hemophilia gene therapy would affect medical and pharmacy benefits.

Schedule of Educational Activity

This activity consists of an audio recording, pre-test assessment questions (not required to pass or continue), post-test questions (required to pass with a score of 70% or higher) and a specific activity evaluation.  


The Academy of Managed Care Pharmacy (AMCP) is accredited by the Accreditation Council for Pharmacy Education as a provider of continuing pharmacy education.

As an accredited provider, AMCP strictly adheres to the ACPE Standards for Commercial Support and other relevant guidelines and standards to ensure the independence of continuing pharmacy education (CPE) activities.  It is AMCP’s intent that all CPE activities be independent, objective, balanced, evidence-based and free of any commercial bias.

CPE Requirements:   

Participants should claim continuing pharmacy education (CPE) credit for this home study activity only if they have not claimed credit for the live activity presented at Specialty Connect 2018. 

CPE credit will be processed through the AMCP Learn portal and reported directly to CPE Monitor.  Please allow 72 hours for processing and posting in your NABP account.

To receive credit for an online activity you must:

  • Review the full content of the activity.
  • Successfully complete the post-test (required to pass with a score of 70% or higher, it may be taken a maximum of three times).
  • Complete a specific activity evaluation.

Statement of Commercial Support

This educational activity is supported by unrestricted educational grants from CSL Behring LLC, Novo Nordisk Inc. and Shire.


Susan C. Winckler | RPh, Esq. | President, Leavitt Partners Consulting | Chief Risk Management Officer | Leavitt Partners.

System Technical Requirements/Viewing Requirements: System Requirements Courses and learning activities are delivered via your Web browser and Acrobat PDF. For all activities, you should have a basic comfort level using a computer and navigating websites.  View the minimum technical and system requirements for continuing education online learning activities.

Credit Information

Activity Number Credit Amount Accreditation Period
ACPE UAN #0233-0000-18-006-H01-P 0.75 Contact Hours April 23, 2018 - April 23, 2021