• Letter or Testimony
  • January 25, 2018

CHA Letter to FDA on PREA Orphan Drug Loophole Guidance

On January 19, CHA joined allied partners in applauding FDA's recent draft guidance on the Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases. The draft guidance proposes to close the loophole where manufacturers request orphan drug designation for a drug for a common adult disease based on the disease's rare incidence in children, but subsequently avoid the pediatric studies required under the Pediatric ResearchEquity Act (PREA). The letter adds that this is an important first step, but more needs to be done — other orphan drugs will continue to be exempted from PREA and its pediatric requirements. CHA and partners have submitted similar comments against the blanket orphan drug exemption during the debate on the Food and Drug Administration Reauthorization Act of 2017.