Available Until 12/31/2023

Rare Disease Spotlight: Hemophilia Treatment Advances on the Horizon

This activity is supported by an educational grant from BioMarin Pharmaceutical Inc.

Course Description:

Diagnosis of rare diseases affecting the blood are increasing in incidence due to sophistication in diagnostics and molecular genetic discoveries and are particularly concerning because of the life-threatening complications. Hemophilia describes a duo of genetically inherited rare blood disorders, hemophilia A and hemophilia B, which lead to excessive bleeding episodes in sufferers. New FDA approved treatment options are expected within the next year, introducing novel mechanisms of action, gene-directed therapies, or alternate administration options to reduce treatment gaps and address unmet needs, also bringing complex managed care considerations to access to care and coverage design for single-investment durable cures.

During this session, faculty will focus on hemophilia treatment advances, identify considerations for developing payment models, and discuss innovative methods to address access to care.

Learning Objectives:

At the completion of this activity, participants should be able to:
  1. Review the incidence, current treatment options, and pipeline for hemophilia, a rare blood disorder.
  2. Identify managed care considerations for developing payment models for hemophilia.
  3. Discuss innovative methods to address access to care for the rare blood disease hemophilia and durable cure treatments in the pipeline.


Anne Jackson, FSA, MAAA

Principal and Consulting Actuary

Milliman, Inc.

Indianapolis, IN

Steven W. Pipe, MD

Professor of Pediatrics and Pathology

University of Michigan

Ann Arbor, MI


James Gartner, BS, MBA



Pittsburgh, PA


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

ACPE UAN:0233-0000-22-043-H01-P • Activity Type: Knowledge-Based
Contact Hours: 1.25
Release Date: October 13, 2022 •  Expiration Date: December 31, 2023
Activity Fee: Free for Members and Non-Members

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 Nexus 2022. 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.

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 #0233-0000-22-043-H01-P 1.25 Contact Hours October 13, 2022 - December 31, 2023