Biosimilar Orphan Drugs : The Next Regulatory Challenge 2016

Biosimilar Orphan Drugs : The Next Regulatory Challenge is organized by Biopractice and would be held Oct 21, 2016.

Short Abstract M :
Global orphan drugs market is expected to reach $176 billion mark by the end of 2020 (Report August 2016). A 10.5% compound annual growth rate will see the prescription sales in the orphan sector grow at twice the rate of the overall prescription market which is set to grow at just 5.3% annually between 2014 and 2020. This growth is partially due to the high number of biological orphan drugs, which are currently less susceptible to generic (biosimilar) erosion as compared to small molecules. Meaning, their high economic value continues beyond the end of patent expiration. While an orphan drug would appear, at first glance, to be an attractive candidate for biosimilar drug development there are a number of significant challenges that may deter some developers from undertaking such product development programmes. Both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have guidance for orphan drug development. However, there are no specific guidance for the development of a biosimilar orphan drug. If we are to apply the overarching biosimilar guidances’ in place for a biosimilar orphan drug development, the challenges are enormous and could hinder the development of such drugs.

This presentation aims to address some of the challenges and to encourage debate in the wider community :
• Regulatory Pathway
• Historical data
• Effect size
• Sample size
• Recruitment
• Comparator sourcing and cost