From Gaucher Disease to Tay-Sachs: Evaluating the Drug Treatment for Rare Diseases

ACPE UAN #:  0233-0000-16-065-H01-P 

Release Date:  October 6, 2016

Expiration Date:  October 6, 2019

Activity Type:    Knowledge-based

Contact Hours:  1.5

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

Target Audience: Pharmacists who practice in managed care settings.

Activity Overview

With the passage of the Orphan Drug Act in 1983, pharmaceutical manufacturers have invested in researching and developing therapies for rare diseases, resulting in  more treatments available to patients. Given the nature of a rare disease, there may be limited outcomes data available on these therapies, and common utilization management programs may not be applicable to these patients.  As payers, what rare disease are you likely to see? How are these treatments managed?

This session will focus on specific rare diseases and their treatment, and then delve into payer challenges, such as treatment access and the role of evidence and outcomes.

Learning Objectives:  

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

  1. Define “rare diseases” as outlined in the Orphan Drug Act of 1983.
  2. For specific rare diseases, identify the currently available treatment options.
  3. Summarize the cost considerations for the treatment options available for rare diseases.
  4. Discuss payer strategies used to evaluate reimbursement for the treatment options for rare diseases.
  5. Describe the challenges and possible solutions available that payers face with regards to rare diseases such as the lack of evidence and outcomes.

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. 

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 Nexus 2016.  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 web sites.  View the minimum technical and system requirements for continuing education online learning activities.

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.

Steve Boekenoogen, PharmD
Director, Clinical Account Services
MedImpact Healthcare Systems, Inc.
Lake Stevens, Washington

Credit Information

Activity Number Credit Amount Accreditation Period
ACPE UAN #0233-0000-16-065-H01-P 1.5 Contact Hours October 06, 2016 - October 06, 2019