Managing Orphan Drugs for Rare Diseases: Challenges, Opportunities, and Value Assessment

ACPE UAN #:   0233-0000-17-002-H04-P  

Release Date:  May 27, 2017

Expiration Date:  May 27, 2020

Activity Type:  Knowledge-based  

Credit Hours:  1.5 contact hours 

Activity Fee:  Member: $15.00  Non-Member:  $30.00  

Activity Overview: 

Managing Orphan Drugs for Rare Diseases: Challenges, Opportunities and Value Assessment. The Orphan Drug Act of 1983 established incentives for the development of drugs used to treat rare diseases—conditions that affect fewer than 200,000 people in the United States. More than 400 medications have been approved with orphan status since then. Today, orphan drugs are being approved at a record pace, and they account for an ever-increasing percentage of specialty drug approvals.   Rare diseases and orphan drugs pose unique challenges for payers, because standard population management strategies often don’t apply. In an evidence-based world, how do we assess and apply results of clinical trials that may include 100 or fewer patients? How do we evaluate efficacy in the absence of biomarkers or other objective measures of clinical outcome? How do we assess value if traditional cost-effectiveness calculations are unreliable?  This session will provide the payer perspective on managing orphan drugs for rare diseases and explore promising methods for assessing the value of treatment. The panel discussion that follows will expand the dialogue to include viewpoints from a number of key stakeholders.  

Target audience:   This program was designed for managed care pharmacists and those with an interest in the management of specialty pharmacy utilization and spend.

Learning Objectives:

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

1. Identify at least three challenges payers face when determining coverage for orphan drugs.
2. Discuss how the available evidence may best be used to assess treatments for rare diseases.
3. Describe promising methods of assessing the value of treatments for rare diseases.
4. Explain how coverage decisions for orphan drugs affect key stakeholders, including patients and clinicians.

Method and CE Requirements:   

Participants should claim continuing pharmacy education (CPE) credit for this Specialty Pharmacy Connect home study activity, only if they have not claimed credit for the live activity presented in conjunction with AMCP Annual Meeting 2017. 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 CPE Monitor account .

Pharmacists interested in obtaining CPE credit must:

  • Review the full content of the activity. 
  • Complete a Post-Test which requires a passing score of 70% or higher.  It may be taken a maximum of three times.
  • Complete a specific evaluation. 

System Technical and Internet 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. Contact US.

Faculty Relevant Financial Relationships Disclosure: 

AMCP asks everyone who is in a position to control the content of a continuing pharmacy education activity to disclose all relevant financial relationships with commercial interests. If AMCP determines that a conflict of interest exists as a result of a financial relationship you have (or that your spouse/partner has), we will need to take appropriate steps to resolve the conflict of interest.


ACPE Logo  The Academy of Managed Care Pharmacy (AMCP) is accredited by the Accreditation Council for Pharmacy Education (ACPE) as a provider of continuing pharmacy education (CPE).

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.  


Permission has been obtained to use copyrighted materials.

Statement of Commercial Support

This activity is supported by an independent educational grant from Sanofi Genzyme.  

Meet the ModeratorSusan C. Winckler's biography.

Credit Information

Activity Number Credit Amount Accreditation Period
ACPE UAN #0233-0000-17-002-H04-P 1.5 Contact Hours March 27, 2017 - March 27, 2020