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34 Pharmaceutical Technology Europe BIO/PHARMA OUTSOURCING INNOVATION eBOOK 2022 P h a r mTe c h . c o m Quality/Regulations T o foster the development of treatments for under- treated and rare conditions, the US Food and Drug Administration (FDA) grants orphan drug desig- nation to drugs and biologics that treat, prevent, or diagnose diseases that affect fewer than 200,000 people in the United States or drugs that meet certain cost recovery provisions (1). This designation comes with incentives such as tax credits, user fee exemptions, and market exclusivity. To receive such designation, sponsors must submit requests and present data to support the request. Pharmaceutical Technology Europe asked Judith Jones, FRSC, Director Regulatory Affairs, Global Regulatory Affairs, Catalent, and Matthew Mollan, RPh, PhD, Regional Head of Operations, Early Phase Development North America, Catalent, about the specific ways contract development and manufacturing organizations (CDMOs) can assist sponsors in developing, applying for, and manufacturing orphan drugs. Regulatory requirements PTE: What are the criteria for receiving orphan drug desig- nation in the US compared to the European Union? Jones: In the US, to be designated as an orphan drug, sponsors need to show that the disease or condition that the drug is intended to treat has a prevalence of 7.5 or less for every 10,000 individuals, as opposed to five in every 10,000 individuals in the European Union. If this criterion is not met, orphan drug designation could still be granted in both the US and EU if sponsors can demonstrate that there would be insufficient return on investment to cover the cost of development when commercializing the drug in the respective markets. Another additional criterion in the EU is that there is no satisfactory method of diagnosis authorized in the EU for the prevention or treatment of the disease or condition in question. PTE: How are the processes different in the US versus the EU to receive orphan drug designation? Jones: While the process of orphan drug designation re- quirements for FDA and the EMA [European Medicines Agency] do not fundamentally differ, they do vary in terms NATALI_MIS - STOCK.ADOBE.COM Fostering Orphan Drugs Susan Haigney CDMOs offer expertise and customization options for sponsors of orphan drugs.