NORD releases statement about PDUFA VI commitment letter
The U.S. Food and Drug Administration (FDA) has published its latest performance goals for the PDUFA VI, which is an agreement involving the pharmaceutical industry and the FDA. It requires those two organizations to pay user fees that will help pharmaceutical products gain more reviews. PDUFA VI must receive reauthorization approval every five years, typically including regulatory reforms.
The agreement is still waiting for approval from Congress, but it may help people who have rare diseases.
The statement discusses the Rare Diseases program (RDP) being joined with rare disease specialists, which could be helpful for reviewing rare disease drugs. Orphan therapies are also included in this new review system. Having Rare Diseases Program staff available with each review division, regulatory review and orphan drug development will speed up and better serve the rare disease community.
Reforms include several new changes. They continue the Patient-Focused Drug Development Program (PFDD), widen the Benefit/Risk Framework development process, unite product review for more rare diseases, promote public workshops and modify the biomarker qualification process, as well as speed and increase funding for breakthrough therapies.
Organizations in this story
National Organization for Rare Disorders (NORD) 55 Kenosia Avenue Danbury, CT 06810 ,