Drugs, Diseases and the Managed Care Impact

(EDU-D5) Rare Disease Spotlight: Hemophilia Treatment Advances on the Horizon

Thursday, October 13, 2022

2:45 PM – 4:00 PM ET

Location: Potomac D

ACPE UAN:0233-0000-22-043-L01-P | Contact Hours: 1.25 | Activity Type: Knowledge-based

Faculty(s)

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

Moderator(s)

JG

James Gartner, BS, MBA

President
PerformRx

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

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:

Review the incidence, current treatment options, and pipeline for hemophilia, a rare blood disorder.
Identify managed care considerations for developing payment models for hemophilia.
Discuss innovative methods to address access to care for the rare blood disease hemophilia and durable cure treatments in the pipeline.