Hogan Lovells | 2024 Life Sciences and Health Care Horizons | Cross-jurisdictional75Possibly significant changes to U.S. and EU orphan drugregulation may be loomingEvents over the past year suggest 2024 will bringheightened attention – and perhaps meaningfulchanges – to the orphan drug regulatory schemesin the U.S. and the EU. Authorities seem to beseeking to adjust the balance between creatingincentives for orphan drug development andallowing competing products to come to market.Recent events that may signal upcomingsignificant changes include:U.S.• in December 2023, FDA finalized a guidancedocument providing additional flexibility inthe regulatory standards for drugs intendedfor subsets of rare diseases, including withregard to nonclinical data and programs forexpediting development.• a recent FDA decision – being challenged incourt – suggests the agency is changing thestandard for breaking a competitor’s orphanexclusivity and awarding a new exclusivityperiod by a finding of clinical superiority byway of a major contribution to patient care. Anumber of core FDA and administrative lawissues are at play here.• in denying a rare pediatric disease priorityreview voucher for a gene therapy product,FDA took an approach to defining samenessof active ingredient that seems difficult toreconcile with views on the topic expressed ina relatively recent guidance.• a provision in the Inflation Reduction Act(IRA) that exempts certain orphan productsfrom being subject to price negotiations isHein van den BosPartnerAmsterdamleading some companies to rethink strategiesfor product development. The provision, whichwould seem intended to create incentivesfor orphan drug development, may actuallybe leading companies to stop investigatingproducts for second orphan uses.EU• The European Commission publishedproposed legislation that would reduce theavailability and benefits of orphan marketexclusivity. Proposed changes include:– reducing the standard orphan exclusivityperiod to nine years (from 10, which wouldbe reserved for productsaddressing a high unmet need).– limiting applicants to a maximum oftwo one-year extensions of exclusivityfor subsequent orphan approvals(rather than the full additional periodof exclusivity currently available witheach such approval).– allowing competitors to apply for marketingauthorization during the exclusivity period(rather than waiting until the end ofexclusivity before submission).– dropping the two-year extension ofexclusivity for completing pediatric studies.Especially because the nature, extent, andtiming of any changes are uncertain, the strategicimplications can be important for products in allstages of development, and merit attention.Komal Karnik NigamCounselWashington, D.C.Yetunde FadahunsiSenior AssociateWashington, D.C.
It seems that your browser's pop-up blocker has prevented us from opening a new window/tab. Please click the button below to open the link manually.
