Managing Orphan Drugs for Rare Diseases

SPC tme changeACPE UAN: 0233-0000-17-002-L04-P 

1.5 contact hours | Knowledge-based 

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

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.  

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.


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