Patient Input at the FDA: Who, What, Why & When?


Today's post is authored by Dr. Rob Metcalf, Vice President of Global Regulatory Affairs at Eli Lilly and Company and a member of the FasterCures Benefit Risk Advisory Council. You can read the first two posts in his series on PDUFA here and here.

The most recent reauthorization of the Prescription Drug User Fee Act (PDUFA) highlighted new efforts by the Food and Drug Administration (FDA) to enable patients and patient groups to become actively involved in FDA evaluations of new medicines. Two programs in particular--the Patient-Focused Drug Development (PFDD) Initiative and Benefit/Risk Assessment--work in tandem to inform the FDA's decisions on whether or not to approve a medicine for patient use. For example, the FDA plans to weigh patient input collected at PFDD meetings in their assessment of the benefits and risks of a potential treatment.

As these programs have commenced, questions remain regarding how the FDA plans to include patient input collected at PFDD meetings in its official assessment of the benefits and risks of a potential treatment. To help understand the path forward for the PFDD and benefit/risk assessment programs, let's take a look at how they work together:

Who's involved?
Initially, the FDA set up meetings with patient groups representing twenty different diseases. While this is an excellent beginning, we need to start exploring how to provide a broader number of interested groups the ability to tell their story to the agency as well. This will provide the FDA with even more diverse input on a number of disease states.

What do we need?
The feedback often collected during PFDD meetings is clearly beneficial to the FDA. Patient stories illuminate the day-to-day difficulties of living with a disease, and offer insights into what patients really think about the benefits and tradeoffs of a potential treatment. In addition, the FDA -- being a science-driven agency -- needs further meaningful, scientific data on which to act. Some patient groups have taken initiatives such as commissioning larger and more representative surveys that can provide the FDA broader, data-driven research to evaluate. More patient advocacy organizations may want to think through how to collect scientifically-significant data that broadly represents their disease state, further supporting patient perspectives on their disease. Additionally, both industry and the FDA should be prepared to utilize this data in the development and review of new medicines.

Why do we need to know more about how the FDA uses patient input?
There's no question that the patient feedback gained through PFDD meetings and other avenues does provide the FDA with a deeper understanding of how patients weigh the pros and cons of a potential medicine. However, benefit/risk assessment is incredibly complex and relies on many factors in addition to patient input. It's important that the FDA continue to be transparent regarding how patient input is being used along with data from clinical studies and expert assessments in understanding the benefit-risk profile of new therapies.

When will regulatory change continue to happen?
The last iteration of PDUFA, PDUFA V, was reauthorized as part of the Food and Drug Administration Safety and Innovation Act in 2012, meaning that Congress will reevaluate PDUFA in 2017. However, planning for the next PDUFA reauthorization is already underway - negotiations on PDUFA VI could start as early as next summer.

Because PDUFA is reauthorized every five years, PDUFA VI offers all stakeholders a stellar opportunity to review the results of these patient-focused initiatives so far, and to clarify and improve how the information is being used to inform FDA decisions about new medicines and treatments.

If you represent a patient advocacy group and are interested in learning more about how patient input can help strengthen and further inform benefit/risk assessments, you might want to attend the FasterCures Benefit/Risk Boot Camp next week in Washington, DC. More information - including an agenda and registration details -- can be found at