July 11, 2018
FDA's Patient Affairs Staff Taking the Lead
By: Andrea Furia-Helms and Samir Shaikh
In December 2017, an FDA Voice blog underscored FDA's commitment to working with patients, caregivers, and advocates and to "incorporate the perspectives of these groups into the FDA regulatory decision making processes." We identified several key objectives that would help build on existing patient engagement activities led by the Patient Affairs Staff (PAS), an internal Agency team devoted to supporting cross-cutting patient engagement activities. We want to update you on the progress of several of these key objectives.
Creating and assisting with public and private collaborations and partnerships to discuss regulatory topics of interest
Members Identified for Patent Engagement Collaborative: To strengthen patient involvement, FDA announced the launch of the Patient Engagement Collaborative (PEC), comprising of external patient community stakeholders who could offer their experiences and perspectives on patient engagement in FDA's regulatory processes. Following an official call for nominations, a selection committee consisting of patient advocates, staff from the Clinical Trials Transformation Initiative, and FDA staff from multiple medical product centers, has chosen 16 individuals to serve as the first slate of PEC members. A great deal of emphasis was placed on ensuring involvement of representatives with a variety of perspectives and inclusion of patients, caregivers, and representatives from a diversity of patient organizations. Members are expected to serve for a total of 2 to 3 years, with this first slate beginning their terms as of August 2018.
Leading cross-center programs and activities that can leverage best practices and enhance patient engagement
Listening Session MOU with NORD: PAS led the signing of a Memorandum of Understanding with the National Organization for Rare Disorders (NORD) to help conduct outreach (e.g., pilot listening sessions) on ways to expand the inclusion of patient-related experience into FDA regulatory decisions on rare diseases and conditions. PAS is in the process of organizing listening sessions as part of the MOU pilot on rare diseases to help foster early and iterative engagement on key clinical and regulatory issues. The first therapeutic area identified for the listening pilot is genetic disorders.
Central Point of Entry for Inquiries and Meeting Requests: PAS has initiated the development of a centralized point of entry into FDA for inquiries and meeting requests from patients, caregivers, and patient organizations. PAS is co-developing a solution with the medical product centers to craft streamlined routing processes to ensure inquiries are received and responded to in an effective and efficient manner. The new entry point will build on existing systems situated within each of the medical product centers such as "Request a Meeting on Drugs" developed by the Professional Affairs and Stakeholder Engagement (PASE) Staff in CDER.
Enhancing FDA’s external communication platforms to expand public awareness and help patients, caregivers, and advocates navigate FDA engagement activities and regulatory processes
We have updated the PAS webpage to provide information on Staff activities and accomplishments related to the key objectives, including the following:
- For patients and patient advocates wishing to engage now, PAS has provided a table summarizing several of the agencies patient engagement initiatives.
- A glossary of standardized nomenclature and terminologies is being shared specifically for use in the context of Patient-Focused Drug Development (PFDD). The glossary will help ensure transparency and the standardized use of terminology as the Agency drafts a series of patient-focused drug development guidances, an effort resulting from the 21st Century Cures Act of 2016 and FDA commitments under the reauthorization of the Prescription Drug User Fee Act (PDUFA VI).
FDA is committed to expanding patient involvement in the regulatory work it does, and provisions in both the 21st Century Cures Act of 2016 and the Food and Drug Administration Reauthorization Act of 2017 support our new initiatives. More plans are in development, and we will continue to keep you informed as additional activities are announced. In the meantime, for more information about FDA's Patient Affairs Staff and initiative and the new Patient Engagement Collaborative, visit the PAS webpage.
Andrea-Furia Helms, Director of FDA’s Patient Affairs Staff
Samir Shaikh, Deputy Director of FDA’s Patient Affairs Staff