In July, President Obama signed into law the Food and Drug Administration Safety and Innovation Act of 2012 (FDASIA), which reauthorizes the Prescription Drug User Fee Act (PDUFA) and includes new Food and Drug Administration (FDA) performance goals and procedures for 2013–2017. FDASIA creates two new programs to enhance patient participation in drug development and benefit-risk assessments.
Although the FDA involves patients in the product review process, there are few venues to gather the patient perspective more broadly. There is a new acknowledgment by the FDA that people with different conditions, and different stages of the same condition, may perceive the risk associated with a treatment differently (i.e., someone with an inhibitor may be willing to accept more risk for a new therapy than someone with mild hemophilia). These new efforts seek to get more information from patients on the broad range of perspectives. NHF is excited to participate in the following new initiatives:
Patient-Focused Drug Development Initiative
In this initiative, the FDA will focus on 20 disease areas during a five-year period. One of the proposed areas is “clotting disorders (e.g., hemophilia A (factor VIII deficiency) and von Willebrand disease).” For each disease area, FDA will have a public meeting to discuss topics, such 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. These meetings will include FDA reviewers, the patient advocacy community and other interested stakeholders.
NHF will be submitting written comments to the FDA on the preliminary list of disease states. We will commend the FDA for including bleeding disorders on the list. We will also participate in meetings relevant to the bleeding disorders community. This is an excellent opportunity to ensure that the FDA better understands the perspective of individuals with bleeding disorders. We will keep you informed as we learn more about this initiative.
Patient Consultation Meetings FDA is also initiating a series of meetings with patient stakeholders to discuss process questions for patient-focused drug development. These meetings will be more general than those mentioned above and are designed to help the FDA identify other possible issues related to patient-focused drug development. At this time FDA has only released a few discussion topics, including how it can balance different disease areas and how to approach issues when patient stakeholders for the same disease have different and potentially conflicting views. NHF is looking forward to participating in the first stakeholder meeting.