Available Until 12/31/2024

Rare Disease Spotlight: Hemophilia Treatment Advances

This activity is supported by an educational grant from CSL Behring.

Course Description:

Currently, about 20,000 individuals are living with hemophilia in the United States, and about 60% of individuals with hemophilia A experience a severe form of the blood disorder. Those with hemophilia B, however, might not begin exhibiting symptoms until later in life. Development in the rare disease drug landscape is expected to increase, where one-third of the drug pipeline extending to 2024 is through the orphan drug approval pathway. The first hemophilia B gene therapy received FDA approval in November 2022 and additional novel hemophilia treatment options are expected within the next year—what managed care strategies reduce treatment gaps and address unmet needs? Faculty for this session will discuss 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 emerging durable cure treatments


Jessica Naber, FSA, MAAA (she/her/hers)

Principal and Consulting Actuary

Milliman, Inc.

Indianapolis, IN

Guy Young, MD

Director, Hemostasis and Thrombosis Center

Children's Hospital Los Angeles

Hermosa Beach, CA


Renee Rizzo Fleming, RPh, MBA


PRN Managed Care Consulting Services, LLC

East Amherst, New York


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:0233-0000-23-008-H01-P • Activity Type: Knowledge-Based
Contact Hours: 1.25
Release Date: March 23, 2023 •  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 AMCP 2023. 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 #0233-0000-23-008-H01-P 1.25 Contact Hours March 23, 2023 - December 31, 2024