Daily Rules, Proposed Rules, and Notices of the Federal Government
On July 9, 2012, the President signed into law the Food and Drug Administration Safety and Innovation Act of 2012 (FDASIA). Title I of FDASIA reauthorizes the Prescription Drug User Fee Act (PDUFA) that provides FDA with the necessary user fee resources to maintain a predictable and efficient review process for human drug and biologic products. The reauthorization of PDUFA includes performance goals and procedures that represent FDA's commitments during fiscal years 2013-2017. These commitments are referred to in section 101 of FDASIA and are available on the FDA Web site at
Section X of these commitments relates to enhancing benefit-risk assessments in regulatory
FDA is committed to obtaining input from patients and, as set out in the commitment letter, will conduct public meetings to consider 20 different disease areas over the 5-year authorization of the program. For each disease area, FDA will conduct a public meeting to discuss such topics as the impact of the disease on patients, the spectrum of severity for those who have the disease, the measures of benefit that matter most to patients, and the adequacy of the existing treatment options for patients. In a separate notice published elsewhere in this issue of the
FDA recognizes that there is significant interest in patient-focused drug development within the patient community. To ensure that patient stakeholders have an additional opportunity to engage in a discussion of key process considerations as this initiative moves forward in PDUFA V, FDA is convening a series of periodic consultation meetings with patient stakeholders to address key process questions for patient-focused drug development. These periodic consultation meetings will be separate from the disease-specific public meetings that are part of FDA's commitments in PDUFA V; however, the process consultation discussions may help inform the best strategies for conducting future disease-specific meetings. FDA anticipates that the periodic consultation meetings will be focused on process questions for consideration by FDA and patient stakeholders. Examples of potential process topics include the following:
1. Given the limits of FDA staff resources and time available, how to prioritize and balance different disease areas identified by different patient stakeholders.
2. How to approach issues when patient stakeholders for the same disease area have different and potentially conflicting views.
3. How to balance access to FDA for patient stakeholders who are local to FDA headquarters versus those in other locations who have less physical access.
4. How to support engagement of patients in disease areas for which no formal advocacy organizations exist. What role, if any, might already organized groups play?
Patient stakeholders provided critical input in the development of the patient-focused drug development proposal during the PDUFA V discussions. FDA expects that there will be continued interest among patient stakeholders as this PDUFA V enhancement is implemented. FDA is publishing this
If you are an individual patient stakeholder who intends to participate in periodic consultation meetings regarding FDA's implementation of the patient-focused drug development initiative, please provide notification by email to