What you need to know about PDUFA VI

The PDUFA VI performance goals letter is good for patients, medical innovation and the FDA.

Andrew PowalenyJuly 27, 2016

What you need to know about PDUFA VI.


The U.S. Food and Drug Administration (FDA) recently released its performance goals letter for the sixth installment of the Prescription Drug User Fee Act (PDUFA). The letter comes after months of technical negotiations between the agency and biopharmaceutical companies, and parallel meetings with patients’ advocacy groups, academia and other stakeholders.

First enacted in 1992, PDUFA authorizes the FDA to collect fees from biopharmaceutical companies to support the agency in its review of new drugs and biologics. Since its inception, PDUFA has helped make FDA review of new medicines more consistent, predictable and efficient while strengthening FDA’s already high safety and efficacy standards.

PDUFA VI builds on the success of previous agreements with continued focus on ensuring patient safety, maintaining the agency’s high standards for regulatory review and promoting timely access to safe and effective innovative medicines for patients. Additionally, there are a number of key areas that will receive increased focus under the new agreement, including:

  • Patient-focused drug development: Strengthens efforts to incorporate patient perspectives into the drug development and review process, including the use of patient-reported outcomes (PROs).
  • Rare diseases and breakthrough therapies: Continues to advance review for medicines to treat rare diseases. Additionally, the agency will continue to prioritize development of breakthrough medicines for patients with serious and life-threatening illnesses.
  • Drug safety surveillance initiative: Increases funding for FDA’s Sentinel postmarket safety monitoring system to continue to strengthen its focus on ensuring patient safety.
  • Real-world evidence (RWE): Explores the use real-world evidence for regulatory decision-making. This has the potential to further our understanding on the use, benefits and risks of drugs and biologics based on information gathered from the “real-world” use of these therapies in the practice of medicine.
  • Staffing at FDA: Helps ensure that FDA can hire and retain a strong scientific and medical workforce to advance its public health mission.

The U.S. leads the world in delivering new medicines to patients, thanks in part to PDUFA. Since 1993, the program has provided more timely access to more than 1,500 new drugs and biologics, including treatments for cancer, rare diseases, cardiovascular, neurological and infectious diseases.  It is critical that Congress reauthorizes the PDUFA program before its expiration in September 2017. 

To learn more about how PDUFA VI will benefit patients, advance medical innovation and build a more effective FDA, view a fact sheet here. View more information on PDUFA at PhRMA.org/PDUFA


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