The FDA has a difficult task when it comes to evaluating and approving new and innovative medical products.
Individual patients may experience the effects of diseases and therapies differently and each individual patient has a unique perspective about treatments or diagnostic procedures that differ from those perspectives of other patients or of their healthcare provider. The FDA has included the patient perspective in FDA Advisory Committee meetings since 1991. This page summarizes the different opportunities that patient and caregivers can get involved in at the FDA.
On this page you will find information about:
- Evolution of Patient Engagement at FDA
- Patient Engagement Collaborative
- FDA and European Medicines Agency Patient Engagement Cluster
- FDA Patient Council
- MOU with the National Organization of Rare Disorders
- FDA Patient Representative Program
- Food and Drug Administration Safety and Innovation Act (FDASIA) Section 1137
- Patient Reported Outcomes
- Patient Focused Drug Development Initiative
- Device Patient Preference Initiative
- Devices Patient Engagement Advisory Committee
- Devices Patient & Caregiver Connection (P&CC)
- Additional Patient Resources
The FDA and the Clinical Trials Transformation Initiative (CTTI) created a new work group with patient advocacy organizations to talk about patient engagement at the FDA. The Patient Engagement Collaborative (PEC) will provide an ongoing forum to discuss how to achieve more meaningful patient engagement in medical product development and other regulatory discussions. PEC is being led by the FDA’s new Patient Affairs Staff (PAS) in the Office of Medical Products and Tobacco (OMPT), which is responsible for the coordination of agency-wide and cross-center projects related to patient engagement. For more information, visit the Patient Engagement Collaborative website.
For more information about the Patient Engagement Collaborative please email PatientEngagementCollaborative@fda.hhs.gov
The Food and Drug Administration and the European Medicines Agency created a working group on patient engagement called the FDA/EMA Patient Engagement Cluster. The FDA/EMA Patient Engagement cluster joins a series of currently existing EMA/FDA clusters. The cluster allows FDA and EMA to share best practices involving patients along drug and biologic regulatory life-cycles. Information that is discussed is covered by confidentiality agreements signed by the FDA and EMA.
- More information can be found at FDA and European Medicines Agency Patient Engagement Cluster.
The FDASIA 1137 work group evolved into the cross-Center Patient Council, which was established in 2016. This FDA-wide Patient Council is designed to bring together all Centers and Offices to better coordinate and integrate the role of patient perspectives in regulatory decision-making over the total product lifecycle.
FDA has a Memorandum of Understanding (MOU) with the National Organization for Rare Disorders (NORD) to conduct outreach on ways to enhance incorporating patient experiences into regulatory discussions. Planned are joint series of pilot rare disease listening sessions for early and iterative engagement to provide clinical and regulatory understanding of diseases and conditions, provide a common understanding of the most urgent needs, and inform drug development programs.
- More information on the MOU can be found at Memorandum of Understanding Between FDA and the National Organization for Rare Disorders.
The FDA Patient Representative Program is managed by the Advisory Committee Oversight and Management Staff (ACOMS) within the Office of the Commissioner. ACOMS coordinates the recruitment, training, and retention for over 200 FDA Patient Representatives, who are patients or primary caregivers to patients.
These FDA Patient Representatives provide direct input to inform the Agency’s decision-making associated with medical products for drugs, biologics, and medical devices.
The Patient Affairs Staff is responsible for overseeing section 1137 of FDASIA -Patient Participation in Medical Product Discussions. Section 1137 strengthens FDA’s ability to safeguard and advance public health for patients in the agency’s activities. The statue recognizes the value of patient input by facilitating increased involvement of patients earlier in the regulatory process for medical product review.
A patient reported outcome (PRO) is a direct response from the patient regarding his/her health condition, without a healthcare provider or caregiver interpretation. Patient reported outcomes are used in clinical studies reviewed by the:
- Center for Biologics Evaluation and Research.
- Center for Devices and Radiological Health.
- Center for Drug Evaluation and Research.
Patient reported outcome instruments are important tools that can be used in trials to directly measure how the patient feels and functions. Developing new PRO instruments requires determining what patient symptoms are important to measure and creating easily understood questions that can be answered reliably.
Well defined and reliable PRO instruments can be used to support a claim in medical product labeling if the claim is consistent with the instrument’s documented measurement capability.
Therefore, understanding and learning from patients’ perspectives is an important step towards developing instruments that measure outcomes important to patients. Integrating these measures into the design of a clinical study is one method to enhance a patient’s assessment of a medical product’s effects.
- Learn more about the Clinical Outcome Assessment Qualification Program
- Learn more about the Medical Device Development Tools Program (MDDT)
- Learn more about the CDRH Patient-Reported Outcomes (PRO) Compendium
The Center for Drug Evaluation and Research and Center for Biologics Evaluation and Review are committed to successfully implementing the Patient Focused Drug Development (PFDD) initiative. This initiative has been implemented to better obtain the patient perspective on certain diseases and their treatments as part of the authorization of the Prescription Drug User Fee Act V (PDUFA V).
A glossary of standardized nomenclature and terminologies specifically intended for PFDD 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).
The Center for Devices and Radiological Health (CDRH) introduced the Patient Preference Initiative in 2013. This initiative provides information, guidance, and the framework necessary to incorporate the patient voice into the full spectrum of medical device development and regulatory processes.
The initiative aims to advance the science of measuring patient preferences to inform benefit-risk assessments used in regulatory decision-making. In August 2016, a final guidance Patient Preference Information - Submission, Review in PMAs, HDE Applications, and De Novo Requests, and Inclusion in Device Labeling was released. The Final Guidance encourages medical device manufacturers to voluntarily include in their premarket submissions information about the tradeoffs patients may consider when evaluating the benefits and risks of a treatment option. The final guidance does not change any review standards for these types of submissions. It includes recommendations on patient preference studies that may result in valid scientific evidence and how stakeholders, including industry and patient advocacy organizations, can voluntarily collect and submit to FDA patient preference information. It also outlines how the FDA includes patient preference information in FDA’s decision summaries that explain what information the FDA relied on in its approval or marketing authorization of the product.
As the medical device community conducts more patient preference studies, we will gain a better understanding of the tradeoffs that patients are willing to make when weighing their treatment options. The FDA encourages medical device manufacturers to consult with the Agency early when considering patient preference studies.
In 2015, the devices PEAC was established to help assure the needs and experiences of patients are incorporated into FDA’s work, by providing advice to FDA on complex issues relating to medical devices, the regulation of devices, and their use by patients. The PEAC may consider topics such as:
- Agency guidance and policies
- clinical trial or registry design
- patient preference study design
- benefit-risk determinations
- device labeling
- unmet clinical needs and available alternatives
- patient reported outcomes and device-related quality of life or health status issues
- other patient-related topics
The PEAC will provide relevant skills and perspectives, in order to improve communication of benefits, risks, clinical outcomes, and increase integration of patient perspectives into the regulatory process for medical devices. It will perform its duties by discussing and providing advice and recommendation in ways such as: Identifying new approaches, promoting innovation, recognizing unforeseen risks or barriers, and identifying unintended consequences that could result from FDA policy.
In 2018, the FDA established the Patient and Caregiver Connection program (P&CC) designed to provide the Center for Devices and Radiological Health (CDRH) staff with a formal process to obtain patient and caregiver input.
The development of this program is part of CDRH’s Patient Science and Engagement Program aimed at fostering engagement with patients, caregivers, and patient advocates throughout the development, evaluation and surveillance of medical devices. The P&CC aims to partner with patient organizations to provide a means for CDRH staff to gain insights from patients and caregivers about their experiences living with their diseases as well as interfacing with medical devices intended to diagnose and treat their medical conditions.
- Read the Patient Engagement Report
- Review the Patient-Focused Drug Development: Disease Area Meetings Planned held 2013-2017
- Find out more about MedWatch: The FDA Safety Information and Adverse Event Reporting Program