Issue link: https://www.e-digitaleditions.com/i/1467950
28 BioPharm International May 2022 eBook www.biopharminternational.com Fostering Orphan Drugs CDMOs offer expertise and customization options for sponsors of orphan drugs. T o foster the development of treatments for u nd e r t r e ate d a nd r a r e c ond it ion s , F DA g ra nts or pha n d r ug desig nat ion to d r ug s and biologics that treat, prevent, or diagnose d is e a s e s t h at a f fe c t fe w er t h a n 2 0 0 , 0 0 0 people in the United States or drugs that meet certain cost recover y prov isions (1). T his desig nation comes with incentives such as tax credits, user fee exemptions, and ma rket exc lusiv it y. To receive such desig nation, sponsor s must subm it reque sts a nd pre sent d ata to support the request. BioPharm Inte r national asked Jud ith Jones, FR SC, D i r e c t o r R e g u l a t o r y A f f a i r s , G l o b a l R e g u l a t o r y A ffa irs, Cata lent, and Mat thew Mol lan, R Ph, PhD, Regional Head of Operations, Early Phase Development Nor t h A mer ic a , Cat a lent , a b out t he s p e c i f ic w ay s contract development and manufacturing organizations (CDMOs) can assist sponsors in developing, applying for, and manufacturing orphan drugs. REGULATORY REQUIREMENTS BioPharm: What are the criteria for receiving orphan drug designation 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 insuf- ficient 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. BioPharm: How are the processes different in the US versus the EU to receive orphan drug designation? Jones: While the process of orphan drug designation requirements for FDA and the EMA [European Medicines SUSAN HAIGNEY Partnerships for Outsourcing Quality/Regulations natali_mis - STOCK.ADOBE.COM