In the past five years, 39 orphan drugs were launched in the US across numerous therapeutic categories, including multiple myeloma, chronic myelogenous leukemia, metastatic non-small cell lung cancer, hemophilia, tuberculosis, homozygous familial hypercholesterolemia, and cystic fibrosis. In 2013 alone, nine orphan drugs were approved, the most in a single year. The growing number of commercially available orphan drugs and the high cost of various orphan treatments have led to reconsideration of payer reimbursement policies.

Top Reasons to Attend:

• Assess methods to increase orphan drug speed to market at a lower price to the patient
• Discuss the impending economic evolution in the rare disease landscape with senior executives
• Understand factors affecting reimbursement and formulary decisions
• Benchmark strategies for pricing and cost saving valuation of orphan drugs
• Discover recent findings on global orphan drug patient access issues from a Tufts Center for the Study of Drug Development Impact Report

Rare disease has long been an important field for biotech and pharmaceutical manufacturers. With few competitors in the space for specific molecules and indications, brands have historically maintained significant influence on market and pricing indicators. Recent increases in regulatory approvals, reduced development timelines, and an array of commercial incentives have enhanced the value of orphan drugs and created an explosion in the space. As more and more orphan drugs are being added and removed from formularies, market access dynamics are shifting into a greater unknown.

These forces have created the need for a renewed understanding of the commercial economics of rare disease and orphan drugs so that manufacturers can forecast and commercialize products successfully. This Summit evaluates these forces, their economic impact, and the implications for orphan drug development, patient access, market access, pricing, and reimbursement.