[eLD2] Managed Care and Health System Collaboration to Deliver Value and Equitable Access to Gene and Cell Therapy for Rare Diseases

August 29, 2023

Pricing: Free for Members and $50 for Non-Members

This activity is supported by an independent medical education grant from Pfizer, Inc.

Program Description

Gene and gene-modified cell therapy innovations are evolving, and novel treatment approaches for genetically inherited diseases are emerging to meet medical needs for rare diseases. Approximately 7,000 identified rare diseases exist, yet only a few hundred treatments are approved.1-3 Gene and cell therapy is particularly relevant to patients with a rare disease, as more than 80 percent of rare diseases have a known single-gene cause.1-2

Faculty for this session will provide an overview of gene and cell therapy innovation for rare diseases, details of the new and pipeline clinical indications, and host a discussion between managed care and health systems on collaborating strategies to bridge access and utilization of these therapies.

  1. AMCP Partnership Forum: Preparing for and managing rare diseases. J Manag Care Spec Pharm. 2021 Mar;27(3):415-420. doi: 10.18553/jmcp.2021.20546. Epub 2021 Feb 4. PMID: 33538235.
  2. Haendel M, Vasilevsky N, Unni D, Bologa C, Harris N, Rehm H, Hamosh A, Baynam G, Groza T, McMurry J, Dawkins H, Rath A, Thaxon C, Bocci G, Joachimiak MP, Köhler S, Robinson PN, Mungall C, Oprea TI. How many rare diseases are there? Nat Rev Drug Discov. 2020 Feb;19(2):77-78. doi: 10.1038/d41573-019-00180-y.
  3. U.S. Food and Drug Administration. FDA News Room. Rare Diesease Day 2020: FDA continues important work on treatments for rare diseases. Accessed January 14, 2021. https://www.fda.gov/news-events/fda-voices/rare-disease-day-2020-fda-continuesimportant-work-treatments-rare-diseases



Target Audience

This knowledge-based activity has been designed to meet the educational needs of pharmacists in managed care settings. 

Learning Objectives

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

  1. Define how rare diseases are classified in the United States.
  2. Discuss new and emerging advances in using gene and cell therapy in rare diseases based on efficacy, safety, durability, policy, and clinical utilization.
  3. Identify health plan and health system collaborations that contribute to multidisciplinary efforts of integrating gene therapy into practice.
Course summary
Available credit: 
  • 1.00 ACPE
    ACPE The Academy of Managed Care Pharmacy is accredited by the Accreditation Council for Pharmacy Education as a provider of continuing pharmacy education.
Course opens: 
Course expires: 
Event starts: 
08/29/2023 - 1:00pm EDT
Event ends: 
08/29/2023 - 2:00pm EDT


Andreas Klein, MD
Director, Transplant and Cellular Therapy Program
Tufts Medical Center

Kasey Raetz, PharmD
VP, Pharma Contracting & Strategy
Express Scripts

Marshall Summar, MD
Director, Rare Disease Institute Laboratory
Children's National Hospital
Uncommon Cures, LLC


Alex Dong, PharmD
Senior Manager, Specialty Product Development - Gene Therapy
CVS Health


Financial Relationship Disclosures:

Faculty/Reviewer/PlannerReported Relevant Financial Relationships
Elizabeth Saltzman
Kasey Raetz
Marshall Summar
Andreas Klein
Pfizer, DSMB Chair, LOXO/Lilly, Takeda, DSMB Member
  • AMCP Staff have disclosed no relevant financial relationships.
  • If applicable, relevant financial relationships have been mitigated and documented.
  • Content has undergone a peer review to ensure content validity.

Available Credit

  • 1.00 ACPE
    ACPE The Academy of Managed Care Pharmacy is accredited by the Accreditation Council for Pharmacy Education as a provider of continuing pharmacy education.


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