Real-World Experience Managing Orphan Drugs Across Pharmacy and Medical Benefits

ACPE UAN #:  0233-0000-18-014-H04-P

Release DateApril 24, 2018

Expiration Date:  April 24, 2021

Activity Type:    Knowledge-based

Contact Hours1.25

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

Target Audience: Pharmacists who practice in managed care settings.

Activity Description

Recently approved drugs for orphan diseases have annual costs ranging from $90,000 to over $1 million, while also having variable value propositions. Individual orphan diseases represent small populations (the total proportion of the population with a rare disease is estimated to be at least 5%), but orphan drugs consist of 9% of the total drug spend. 

This session will discuss formulary and utilization management strategies across pharmacy and medical benefits for recent treatment advances in orphan diseases, including Duchene Muscular Dystrophy (DMD), Cystic Fibrosis, Spinal Muscular Atrophy (SMA) and Huntington’s disease. Faculty also will compare modeled budget impacts with the actuarial experience of pharmacy benefit manager (PBM) and health plan perspectives with these treatments.

Learning Objectives:  

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

  1. Describe how the clinical data and value proposition for specialty drugs treating rare diseases may impact coverage.
  2. Identify the differences between the modeled United States’ budget impact versus the health plan actuarial experience thus far with high-cost specialty drugs for rare diseases.
  3. Discuss management strategies for high-cost specialty treatments of rare diseases on 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 AMCP Annual Meeting 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.


Marti Groeneweg, PharmD, BCPS | Drug Education Coordinator | Kaiser Permanente | Portland, Oregon.

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 #0233-0000-18-014-H04-P 1.25 Contact Hours April 24, 2018 - April 24, 2021