For Patients

Learn About Patient Engagement at the FDA

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.

FDA Patient Network

The Office of Health and Constituent Affairs created the FDA Patient Network in 2012 as a one-stop-shop of FDA web-based resources to help patients and their families find relevant information from FDA’s website.

The FDA Patient Network help patients, caregivers and consumers find the information they need to support their own health care, whether they want to learn more about clinical trials and where to find studies or when accessing investigational products outside of trials might be appropriate.  More information

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FDA Patient Representative Program

The FDA Patient Representative Program is managed by the Office of Health and Constituent Affairs within the Office of the Commissioner.  The Office of Health and Constituent Affairs-Patient Liaison Program 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. More information

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Food and Drug Administration Safety and Innovation Act (FDASIA) Section 1137

The Office of Health and Constituent Affairs 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.  More information

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FDA and European Medicines Agency Patient Engagement Cluster

The Food and Drug Administration and the European Medicines Agency have created a new work-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

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Patient Engagement Initiative

The FDA and the Clinical Trials Transformation Initiative (CTTI) a working together to create a new work group with patient advocacy organizations to talk about patient engagement at the FDA. More information

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Patient Reported Outcomes (PRO)

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. More information

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Patient Focused Drug Development Initiative (PFDD)

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). 
FDA disease areas selected were based on comments received in a public docket. Comments addressing over 90 disease areas were submitted by:

  • patients,

  • patient advocates and advocacy groups,

  • caregivers,

  • healthcare providers,

  • professional societies,

  • scientific and academic experts,

  • pharmaceutical companies

As of April 20, 2017 the FDA has held 22 of the 25 disease specific meetings. For a complete list of meetings please visit the Patient Focused Drug Development page.   After each disease specific meeting, the FDA publishes a meeting report called The Voice of the Patient.  The report included proceedings and a summary analysis of the input received by FDA relevant to FDA’s consideration of disease severity and unmet medical need. More information

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Device Patient Preference Initiative

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. More information.

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Patient Engagement Advisory Committee (PEAC)

In 2015, the 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. More information

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Page Last Updated: 04/20/2017
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