It seems obvious that an agency mandated to protect the public health of the United States would rely on patients to share their disease experiences and expectations for treatment.  However, active involvement of patients in FDA regulatory policy and decision-making only began with the HIV/AIDS crisis in the late 1980’s.   During the AIDS crisis in the 1980s, advocacy organizations such as ACT UP and Project Inform directly engaged FDA to increase access to life-saving therapies.  HIV/AIDS advocates challenged the notion that patients should not have a voice in whether they are exposed to certain risks.  Activists also became heavily involved in the conduct of clinical trials, including the use of community-based trials, which were easier to implement, less expensive, and ensured that doctors familiar with HIV/AIDS patients were involved in their treatment and care.  

One of the most important accomplishments of the HIV/AIDS activist movement was its intimate engagement with FDA and subsequent influence on regulatory policy.  Members of ACT UP and Project Inform came to FDA prepared with the knowledge necessary to engage meaningfully with agency experts to accomplish their disease-specific policy goals.  They came to the agency focused on very specific issues, solutions, and opportunities for change.   Their efforts resulted in a partnership with the agency that changed FDA regulatory policy and led to significant regulatory and legislative changes. 

Patient engagement at FDA has subsequently changed a lot over the last 30 years.  In the 1990’s, the Patient Representative Program provided an opportunity for patients, patients’ families, and caregivers to provide the patient perspective as participants on FDA Advisory Committees.   Patients and patient advocacy groups have also participated in open public hearings, public policy and town hall meetings, and provided written comments to advisory committees and in response to proposed FDA rules in the Federal Register. 

The opportunity for patient engagement with FDA increased dramatically in 2012 with the enactment of the Food and Drug Administration Safety and Innovation Act (FDASIA).  For the first time, FDA was mandated by law to: develop and implement strategies to solicit the views of patients during the medical product development process and consider the perspectives of patients during regulatory discussions (FDASIA Sec 1137).   Under the MDUFA and PDUFA reauthorizations, FDA launched several programs including CDER/CBER’s Patient Focused Drug Development (PFDD) program, the Patient Preference Initiative out of CDRH, the Patient Engagement Advisory Committee (PEAC), and the FDA Patient Council.  Most recently, the Office of Patient Affairs was created to provide a central point for all patient engagement activities at the Agency. 

All these initiatives and programs aim to provide mechanisms through which patients can have a greater influence on FDA regulatory process in their disease area.  Though much remains to be done to ensure the patient voice is considered at FDA, patients now have a much greater role to play in the development and regulatory review of potentially life-changing therapies than ever before.

Sources:

1. Epstein, S. (1996). Impure Science.  AIDS, Activism, and the Politics of Knowledge. Berkeley: University of California Press

2. Manganiello, M., & Anderson, M. (2013). Back to Basics:  HIV/AIDS Advocacy as a Model for Catalyzing Change. Washington, D.C.: HCM Strategists

3. Cox, V., & Scott, M. C. (2014). FDA Advisory Committee Meetings:  What they are, why they happen, and what they mean for regulatory professionals. Regulatory Rapporteur, 11(11), 5-8

4. Klein, R. (2013). Patient Representation at FDA - Patient Input. Paper presented at the 4th Annual Patient-Reported Outcome (PRO) Consortium Workshop, Silver Spring, MD. https://c-path.org/wp-content/uploads/2013/09/PRO_Consortium_FDAPtRepresentativePgm.pdf

5. Food and Drug Administration Safety and Innovation Act (FDASIA), Pub. L. No. 112-144, 126 Stat. 993 Stat. (2012 July 9)

6. FDA. (2017). Enhancing Patient Engagement Efforts Across the Food and Drug Administration; Establishment of a Public Docket; Request for Comments.  Washington, DC: Federal Register Retrieved from https://s3.amazonaws.com/public-inspection.federalregister.gov/2017-04982.pdf