Contains Nonbinding Recommendations 
 
Patient Engagement in the Design and 
Conduct of Medical Device Clinical 
Studies
 
 
Guidance for Industry,  
Food and Drug Administration Staff, 
and Other Stakeholders  
 
Document issued on January 26, 2022.  
 
 
The draft of this document was issued on September 24, 2019. 
 
 
For questions about this document regarding CDRH-regulated devices, contact Michelle Tarver in 
the Office of Strategic Partnerships and Technology Innovation (OST) at (301) 796-6884 or by 
email CDRH_PatientEngagement@fda.hhs.gov. For questions about this document regarding 
CBER-regulated devices, contact the Office of Communication, Outreach, and Development 
(OCOD) at 1-800-835-4709 or 240-402-8010, or by email at ocod@fda.hhs.gov. 
 
     
 
 
 
 
 
 
 
 
U.S. Department of Health and Human Services 
Food and Drug Administration 
Center for Devices and Radiological Health 
Center for Biologics Evaluation and Research 
   
        1  Contains Nonbinding Recommendations 
 
Preface 
 
Public Comment 
 
You may submit electronic comments and suggestions at any time for Agency consideration to 
https://www.regulations.gov. Submit written comments to the Dockets Management Staff, Food 
and Drug Administration, 5630 Fishers Lane, Room 1061, (HFA-305), Rockville, MD 20852.  
Identify all comments with the docket number FDA-2019-D-3846. Comments may not be acted 
upon by the Agency until the document is next revised or updated. 
 
Additional Copies 
 
CDRH 
Additional copies are available from the Internet. You may also send an email request to 
CDRH-Guidance@fda.hhs.gov to receive a copy of the guidance. Please include the document 
number 18040 and complete title of the guidance in the request. 
 
CBER 
Additional copies are available from the Center for Biologics Evaluation and Research (CBER), 
Office of Communication, Outreach, and Development (OCOD), 10903 New Hampshire Ave., 
WO71, Room 3128, Silver Spring, MD 20903, or by calling 1-800-835-4709 or 240-402-8010, 
by email, ocod@fda.hhs.gov, or from the Internet at https://www.fda.gov/vaccines-blood-
biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances.  
 
 
   
        2  Contains Nonbinding Recommendations 
 
Patient Engagement in the Design and 
Conduct of Medical Device Clinical 
Studies 
 
 
Guidance for Industry,  
Food and Drug Administration Staff, 
and Other Stakeholders 
 
This guidance represents the current thinking of the Food and Drug Administration (FDA or 
Agency) on this topic.  It does not establish any rights for any person and is not binding on 
FDA or the public.  You can use an alternative approach if it satisfies the requirements of the 
applicable statutes and regulations.  To discuss an alternative approach, contact the FDA staff 
or Office responsible for this guidance as listed on the title page.  
 
I.  Introduction 
The U.S. Food and Drug Administration (FDA or the Agency) values the experience and 
perspectives of patients and their caregivers.1 FDA believes that these individuals can and should 
be able to provide their insights about a disease or condition, including living with that 
disease/condition, and the impact of medical devices in the diagnosis, treatment, and 
management of the disease/condition, through engagement activities. Patient advisors serving in 
an advisory or consultative capacity can share their experiences living with a medical condition 
to help improve clinical study design and conduct, without participating in the research study 
themselves as explained in more detail below. 
 
This guidance is intended to: 
(1) help sponsors understand how they can voluntarily use patient engagement to elicit 
experience, perspectives, and other relevant information from patient advisors (see 
definition in Section IV) to improve the design and conduct of medical device clinical 
studies;  
(2) highlight the benefits of engaging with patient advisors early in the medical device 
development process; 
 
1 For the purposes of this guidance, the term caregivers is used to mean care-partners.  Caregivers include adult 
family members or other individuals who have a significant relationship with, and who provide a broad range of 
assistance to an individual with a chronic or other health condition, disability, or functional limitation. 
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(3) illustrate which patient engagement activities are generally not considered by FDA to 
constitute research or an activity subject to FDAs regulations, including regulations 
regarding institutional review boards (IRBs); and 
(4) address common questions and misconceptions about collecting and submitting to FDA 
patient engagement information regarding the design and conduct of a medical device 
clinical study.  
 
The contents of this document do not have the force and effect of law and are not meant to bind 
the public in any way, unless specifically incorporated into a contract. This document is intended 
only to provide clarity to the public regarding existing requirements under the law. FDA 
guidance documents, including this guidance, should be viewed only as recommendations, unless 
specific regulatory or statutory requirements are cited.  The use of the word should in Agency 
guidance means that something is suggested or recommended, but not required.  
 
II.  Background
 
On October 11-12, 2017, FDAs Patient Engagement Advisory Committee (PEAC)2 met to 
discuss and make recommendations to FDA regarding patient engagement in medical device 
clinical studies.3 Discussion topics included patient advisor involvement in design of clinical 
investigations; recruitment, enrollment, and retention of study/research participants in clinical 
studies; and opportunities and barriers patient advisors face when collaborating with industry in 
the clinical study process. In a consensus recommendation, the PEAC stated that some type of 
framework should be developed by FDA and industry to clarify how patient advisors can engage 
in the clinical study process. The importance of engaging patients in clinical trials was also 
discussed in a public workshop convened by the FDA and the Clinical Trials Transformation 
Initiative (CTTI) on March 18, 2019.4  This workshop discussed best practices and key 
considerations for enhancing the incorporation of patient perspectives on clinical trial access, 
design, conduct and post-trial follow up.  Based on the PEAC recommendations and the 
discussion at the CTTI workshop, FDA is pursuing various efforts to encourage patient 
engagement in clinical studies, including issuing this guidance document. 
Before issuing this guidance document, FDA released a discussion document to facilitate further 
public discourse on patient engagement in medical device clinical trials.5 The discussion 
document described FDAs initial thoughts about patient engagement and its potential impact on 
medical device clinical studies. The discussion document included targeted questions on which 
the Agency sought public feedback through an open public docket.6 The Agency also sought 
 
2 See 2017 Meeting Materials of the Patient Engagement Advisory Committee, available at: 
https://www.fda.gov/advisory-committees/patient-engagement-advisory-committee/2017-meeting-materials-patient-
engagement-advisory-committee. 
3 The 2017 PEAC meeting discussed patient engagement in clinical trials. For purposes of this guidance, we use the 
term clinical study as a broader term that includes clinical trial and clinical investigations.  
4 See the 2019 Meeting Materials from the FDA CTTI Public Workshop, entitled Enhancing the Incorporation of 
Patient Perspectives in Clinical Trials available at https://www.ctti-clinicaltrials.org/sites/www.ctti-
clinicaltrials.org/files/meeting_summary_-_enhancing_incorporation_of_patient_perspectives_-_final.pdf.  
5 See discussion document entitled Patient Engagement in Medical Device Clinical Trials, available 
at: https://www.fda.gov/media/122893/download. 
6 FDA requested comments on the discussion document through docket FDA-2018-N-4171. 
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public feedback on these questions during the second PEAC meeting, on November 15, 2018.7 
FDA considered comments from the discussion held during both PEAC meetings and the public 
docket in completing this guidance. 
 
Successful adoption of legally marketed medical devices increasingly depends on patient 
acceptance of that technology and patients being more engaged in the healthcare process, along 
with demonstrated public health benefits. FDA believes effective patient engagement can help 
mitigate some of the practical challenges to robust clinical studies, including challenges 
concerning study/research participant enrollment and retention in the study, particularly when 
protocols include lengthier follow-up periods (e.g., through 2 years post-procedure) and/or 
frequent visits to the clinical site, which may require significant travel. Additionally, study plans8 
for medical device studies may be complex, with many endpoints as well as eligibility criteria 
that exclude some study/research participants living with the disease/condition from participating 
in clinical studies. When not adequately addressed, each of these factors can contribute to 
increased time and cost to study sponsors, increased burden to study/research participants and the 
healthcare system, and delays in U.S. patient access to beneficial medical technologies. 
FDA believes medical device clinical studies prospectively designed with input from diverse 
patient advisors, including those from racially and ethnically diverse populations, may help to 
address common challenges faced in these clinical studies and could result in: 
  Faster study/research participant recruitment, enrollment, and study completion; 
  Greater study/research participant commitment and retention, resulting in decreased loss 
to follow-up; 
  Greater study/research participant adherence resulting in fewer protocol 
deviations/violations; 
  Greater study/research participation by diverse patient populations; 
  Fewer protocol revisions; 
  Streamlined data collection resulting in better quality data; and 
  More relevant data on outcomes that matter to patients. 
Feedback received from patients and industry at the PEAC meetings on October 11-12, 2017, 
and November 15, 2018, and the public docket comments related to the PEAC discussion 
document entitled Patient Engagement in Medical Device Clinical Trials indicated broad 
support for patient engagement in clinical studies. Responses to questions posed by FDA at the 
2017 PEAC meeting and in the docket indicated perceived barriers and challenges to such 
engagement including, but not limited to:  
  Perception that FDA does not allow patient engagement in the design and conduct of 
clinical studies;  
  Patient perceptions that their input is not valued by the study plan development teams; 
 
7 See 2018 Meeting Materials of the Patient Engagement Advisory Committee, available at: 
https://www.fda.gov/advisory-committees/advisory-committee-calendar/november-15-2018-patient-engagement-
advisory-committee-meeting-announcement-11152018-11152018.  
8 The term study plan includes the investigational plan as defined under 21 CFR 812.25, which includes the 
protocol.   
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  Sponsors limited awareness, resources, and time to participate in patient engagement 
activities; 
  Challenges finding patient advisors knowledgeable about clinical study methodology; 
  Site investigators reluctance to allow sponsors to engage with patients except as 
study/research participants; 
  Logistical challenges of engaging with patient advisors in-person, which may preclude 
their involvement in the design of clinical studies; and 
  Challenges with determining which patient advisors or patient organizations should be 
engaged, and if multiple patient advisors are engaged, how to reconcile the disparate 
perspectives.  
 
Similar comments were expressed at the FDA CTTI Public Workshop on March 18, 2019. This 
guidance intends to address some of these perceived barriers and challenges. 
 
III.  Scope 
FDA acknowledges that patient engagement may be beneficial across the total product lifecycle. 
This guidance focuses on the application of patient engagement in the design and conduct of 
medical device clinical studies. This guidance does not address study/research participant or 
patient advisor reimbursement or compensation, promotion of investigational devices (see 21 
CFR 812.7), or dissemination of clinical study results.  
 
IV.  Defining Patient Engagement  
For purposes of this guidance, patient engagement is defined as intentional, meaningful 
interactions with patients that provide opportunities for mutual learning, and effective 
collaborations. 
In the context of planning for a clinical study, engaging with patient advisors (see definition 
below) creates an opportunity to share patient experiences, perspectives, needs, and priorities 
during the design and conduct of a clinical study. Importantly, FDA views this type of patient 
engagement differently from interactions that sponsors or clinical researchers (also called 
investigators) may have with individuals who participate in a specific clinical study as 
study/research participants.  
For purposes of this guidance, patients are defined as individuals with or at risk of a specific 
disease or health condition, whether or not they currently receive any therapy to prevent or treat 
that disease/condition. Patients are the individuals who directly experience the benefits and 
harms associated with medical products.9 Patients may include healthy individuals who may be 
undergoing screening or diagnostic tests or individuals living with a medical condition and 
interfacing with medical devices to treat the specific disease or health condition. For the 
purposes of this guidance, we identify two distinct roles for patients who interact with 
 
9 See FDA website entitled, Patient-Focused Drug Development Glossary available at:  
https://www.fda.gov/drugs/development-approval-process-drugs/patient-focused-drug-development-glossary. 
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researchers, sponsors, or FDA regarding clinical studies: study/research participants and 
patient advisors. 
In this guidance, the term study/research participants are individuals who are or become a 
participant in research, as a recipient of the test article, on whom or on whose specimen the test 
article is used, or as a control, and may include healthy individuals. FDA acknowledges that its 
regulations use the term subject or human subject,10 to refer to these individuals, but patients 
may be familiar with a different term. Therefore, in this guidance, the term study/research 
participant is used instead.  
For purposes of this guidance, the term patient advisors refers to individuals who have 
experience living with a disease or condition, and can serve in an advisory or consultative 
capacity to improve clinical study design and conduct, but who are not study/research 
participants themselves or caregivers of study/research participants. Patient advisors are not 
individuals responsible for the decision to prescribe or recommend a medical product such as 
healthcare professionals.  They may include, but are not limited to, individuals who have 
participated in previous clinical studies of the same disease/condition or similar device-type, 
individuals who were screened for but ultimately did not qualify for or did not elect to participate 
in a similar clinical study, representatives from a disease-specific or cross-cutting patient 
organization, healthy individuals who may be potential non-therapeutic (e.g., diagnostic) device 
users, or caregivers (also known as care-partners) of patients who may have experience with the 
disease/condition/device. For example, a clinical study being designed to evaluate the 
performance of a mammography device might enlist women (regardless of whether they have a 
medical diagnosis) to be patient advisors. We recognize that it may be challenging to identify 
patient advisors for clinical studies of diagnostics or treatments for rare diseases.  Sponsors can 
discuss appropriate approaches for engaging with the specific rare disease community with the 
appropriate FDA review divisions.    
Similar to clinical advisors and experienced clinical researchers, patient advisors may provide 
recommendations that positively impact how a study is designed and conducted, improve the 
patient experience during the study, and improve the relevance, quality, and impact of study 
results. However, to avoid potential real or perceived conflicts of interest, these patient advisors 
should not be study/research participants in the same study for which they are advising. 
 
V.  Questions and Answers on Patient Engagement in 
Medical Device Clinical Studies
 
A.  What approaches might sponsors use to engage patient 
advisors to inform the design and conduct of medical 
device clinical studies?  
We recommend sponsors voluntarily identify patient advisors and clearly define the patient 
advisors role early in the study planning process. We encourage sponsors to be clear in their 
planning process about which activities are part of the study plan (i.e., for study/research 
 
10 See 21 CFR 50.3(g), 56.102(e), and 812.3(p). 
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participants) versus those that are non-research patient engagement efforts (i.e., for patient 
advisors) that may improve the design and conduct of the clinical study.  
 
Patient advisors who are educated about clinical studies, the various approaches to managing the 
disease/condition of interest, and how a device may work may be better equipped and feel more 
empowered to voice their perspective in engagement activities. We encourage sponsors to 
consider using existing educational materials and/or partner with organizations that provide 
training for patient advisors to help them most effectively contribute. 
 
Some patient engagement activities that may enhance the design and conduct of clinical studies 
include, but are not limited to:11  
 
  Working with patient advisors to improve the informed consent document to ensure 
patients understand the information presented for the clinical study; 
  Obtaining input from patient advisors on flexible options for follow-up visits and data 
collection techniques to reduce unnecessary burden on study/research participants who 
may have challenges fulfilling the follow-up schedule. Such techniques suggested may 
include allowing extended and/or weekend hours, permitting the study/research 
participants primary healthcare provider to perform some follow-up assessments, 
allowing phone or home visits by clinical researchers, allowing more convenient test labs 
to process routine bloodwork, or using mobile or online technologies to enable virtual or 
remote follow-up12; 
  Working with patient advisors as needed during an ongoing study to discuss barriers to 
recruitment or other issues such as causes of study delays or challenges not anticipated 
before the study13;  
  Discussing with patient advisors their views on which potential endpoints are meaningful 
in the treatment of the specific disease/condition; 
  Working with patient advisors to inform the concepts that should be captured by patient-
reported outcome (PRO)14,15 measures in the clinical study to better reflect outcomes that 
are important to patients; and 
 
11 In addition to these patient engagement activities, obtaining feedback from study/research participants and from 
patients who did not participate in the clinical study (particularly those from underrepresented groups) can reveal 
barriers to participation, approaches to improve recruitment, challenges or other experiences during the study to help 
to streamline and improve future studies. 
12 Please note that this guidance is not intended to focus on the FDAs review of such procedural changes in a 
clinical investigation. Please see FDAs guidance Changes or Modifications During the Conduct of a Clinical 
Investigation, available at: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/changes-
or-modifications-during-conduct-clinical-investigation-final-guidance-industry-and-cdrh.  
13 FDA recommends that patient engagement occur early in the study plan development process.  Incorporating 
input from patient advisors through engagement activities after a study has started would likely be more limited in 
scope since the study plan has been finalized.  
14 For more information on PROs see FDAs guidance Patient-Reported Outcome Measures: Use in Medical 
Product Development to Support Labeling Claims, available at: https://www.fda.gov/regulatory-
information/search-fda-guidance-documents/patient-reported-outcome-measures-use-medical-product-development-
support-labeling-claims.  
15 Please see FDAs guidance Principles for Selecting, Developing, Modifying, and Adapting Patient-Reported 
Outcome Instruments for Use in Medical Device Evaluation available at: https://www.fda.gov/regulatory-
 
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  Working with patient advisors to inform the design of  patient preference16 studies that 
may be used to inform the development of clinical studies or to help understand the 
benefit-risk tradeoffs among patients for the proposed treatment or multiple treatment 
options used for the disease/condition.  
B.  When can input be gathered from patient advisors and 
incorporated into the clinical study? 
Sponsors should consider involving patient advisors during the early planning phases of the 
clinical study so that their input can be incorporated while the study plan is being developed. 
Especially in innovative areas or new target patient populations, we encourage sponsors to confer 
with patient advisors when designing or planning the clinical study. Sponsors may also want to 
consider involving patient advisors post-study to inform improvements for future studies.  
 
In more established areas, patient advisor input on draft study plans may translate into time and 
cost-saving improvements that also make the design more patient-centric. Such input should 
generally be incorporated before the final protocol and informed consent documents are 
submitted to the IRB17 for review.   
 
For clinical studies that require submission of an investigational device exemption (IDE) 
application, this information should be included in the final protocols and informed consent 
documents submitted to the FDA for review as part of the IDE application.18 
 
For ongoing studies that face significant challenges with study/research participant recruitment 
and/or retention, sponsors may want to consider involving patient advisors along with the study 
coordinator to troubleshoot and propose potential solutions. 
 
C.  What are the roles of IRBs and other institutional groups 
in patient engagement? 
Under FDAs regulations, an IRB is any board, committee, or other group formally designated 
by an institution to review, to approve initiation of, and to conduct periodic review of, 
biomedical research involving human subjects.19 The primary purpose of IRB review is to 
assure the protection of the rights and welfare of humans participating as study/research 
participants. Access to personal information or direct engagement with study/research 
 
information/search-fda-guidance-documents/principles-selecting-developing-modifying-and-adapting-patient-
reported-outcome-instruments-use. 
16 For more information on patient preference information, see FDAs guidance Patient Preference Information-- 
Voluntary Submission, Review in Premarket Approval Applications, Humanitarian Device Exemption Applications, 
and De Novo Requests, and Inclusion in Decision Summaries and Device Labeling, available at: 
https://www.fda.gov/regulatory-information/search-fda-guidance-documents/patient-preference-information-
voluntary-submission-review-premarket-approval-applications.  
17 Institutional Review Board is defined in 21 CFR 56.102(g). See also 21 CFR 50.3(i). 
18  For more information and resources on IDEs, please visit: https://www.fda.gov/medical-devices/device-advice-
investigational-device-exemption-ide/ide-guidance.  
19 21 CFR 56.102(g). 
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participants requires careful consideration of Federal, State, and local laws and institutional 
policies for their protection. 
 
Because patient engagement activities with patient advisors primarily involve interaction in a 
consultative or advisory capacity, FDA does not generally consider these types of activities to 
constitute research or an activity subject to FDAs regulations on their own.20 Therefore, FDAs 
research regulations, including IRB requirements, generally would not apply.  
 
In contrast, interactions between study/research participants and investigators typically include 
collecting information as part of a research plan that outlines the methodological approaches to 
be used. Such interactions are generally in the context of a clinical investigation subject to 
FDAs regulations and must satisfy the applicable requirements, including applicable 
requirements at 21 CFR Part 812 (Investigational Device Exemptions), 21 CFR Part 56 (IRBs), 
and 21 CFR Part 50 (Protection of Human Subjects). 
 
Because there are a variety of research contexts in which sponsors may engage with patients to 
obtain information on their experiences and perspectives, a full discussion of which laws may 
apply to such activities is beyond the scope of this guidance. FDA recommends that sponsors 
work with IRBs and Health Insurance Portability and Accountability Act (HIPAA) Privacy 
Boards to determine what laws may apply for a specific research activity.  
D.  How can a sponsor receive feedback on its patient 
engagement plan or patient-centered study design from 
FDA?  
FDA encourages sponsors to integrate patient advisor input in the design and conduct of clinical 
studies for medical devices in appropriate circumstances and is open to discussing patient 
engagement approaches.  For additional information on how to obtain FDA feedback, see the 
CDRH Patient Science and Engagement Program webpage21, as well as FDAs guidance, 
Requests for Feedback and Meetings for Medical Device Submissions: The Q-Submission 
Program. 22  
 
We encourage sponsors to reference any previous patient engagement activities used to inform 
the development of the study plan. Sponsors may also use and cite relevant information from 
their patient engagement activities in their subsequent marketing applications to FDA. 
 
VI.  Summary 
 
20 It should be noted, however, that sponsors of clinical studies are subject to the same applicable statutory and 
regulatory requirements regardless of whether patient engagement is incorporated in the design and conduct of the 
studies.  
21 https://www.fda.gov/about-fda/center-devices-and-radiological-health/cdrh-patient-engagement 
22 https://www.fda.gov/regulatory-information/search-fda-guidance-documents/requests-feedback-and-meetings-
medical-device-submissions-q-submission-program. 
 
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FDA encourages patient engagement in medical device clinical studies in appropriate 
circumstances. This document provides an overview of the potential value, as well as a summary 
of the challenges and potential solutions related to involving patient advisors in the design and 
conduct of clinical studies. This document also identifies a variety of ways sponsors may engage 
patient advisors to design more patient-centric studies that may be more likely to enroll and 
retain study/research participants, as well as collect information that is meaningful to patients.  
 
If you are considering incorporating input from patient advisors in the design or conduct of your 
medical device clinical study, you are encouraged to engage in early interactions with FDA and 
to obtain feedback from the relevant FDA office/division on appropriate design and any 
applicable regulatory requirements. 
 
FDA believes appropriate patient engagement may lead to improved efficiency and quality in the 
design and conduct of medical device clinical studies and greater uptake of results by patients 
and providers when making treatment decisions about a legally marketed medical device, 
ultimately leading to earlier U.S. patient access to beneficial medical devices.  
 
For additional resources and updates on patient engagement, see https://www.fda.gov/about-
fda/center-devices-and-radiological-health/cdrh-patient-engagement. 
 
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