PDUFA: Looking Back and Moving Ahead


Since its initial passing in 1992, the Prescription Drug User Fee Act (PDUFA) has done wonders to improve the Federal Drug Administration's (FDA) process for analyzing and reviewing new medicines. Every renewal of PDUFA - which happens every five years - has pinpointed areas of federal policy in need of change and provided the solutions. Increased resources and staff, shortened review periods, and earlier launch dates--all PDUFA byproducts--have made it easier for the right medicines to get into the hands of the people who need them most.

Giving the FDA the chance to listen to patients and use what it learns to evaluate new treatments were two central parts of PDUFA V, and they remain in focus now. With Congress scheduled to reauthorize PDUFA in 2017, we need to revisit what makes patient input key to the review process. PDUFA V used a two-part plan to bring their opinions into the discussion, via the Patient Focused Drug Development Initiative and an improved Benefit/Risk Analysis model.

Benefit Risk Analysis

Weighing the benefits and risks of each trial medication is a crucial part of the FDA's job. However, not everyone feels the same way about the potential pros and cons of a new treatment: A patient may consider the risks of a medicine incidental in comparison to the potential benefits to their quality of life, while other stakeholders, such as regulators and drug companies, may weigh those risks differently. But who is being served if not the patients themselves? Incorporating a variety of opinions means that the FDA's final decisions best reflect patient needs and that the agency understands what people who will actually take the medicine would consider most effective and beneficial. The FDA's use of a structured benefit-risk assessment can inform decision-makers, facilitate dialogue with companies over the course of a medicine's development, and help the FDA communicate with patients when new medications are approved.

Patient-Focused Drug Development Initiative

The Patient-Focused Drug Development Initiative allows patients' insights to be heard. Periodic meetings give patient groups direct access to the people who have the final say on medicines that may improve or even save their lives. The FDA then records and catalogs what it has learned into reports that can help make its ultimate decisions on a treatment more patient-centric.

This is a great start, but we can still do more to improve the lives of Americans who need timely access to new medications. While decisions will (and should) always rest on the review of clinical data, adding patient input gives the FDA a valuable perspective it might not otherwise have. The time is now for the FDA to actively advance the science of patient input by partnering with advocacy groups, social science academics, and sponsors to establish the best practices for integrating the patient perspective.

As we move into discussions about the next version of PDUFA on July 15, we encourage the FDA to remain transparent and open about how it incorporates this perspective into its decisions, so that patient groups and medical innovators can better understand this core piece of the process. We hope to help them accomplish all of these goals with the passing of PDUFA VI.