Welcome To Today’s Webinar

Thanks for joining us! 

We’ll get started in a few minutes 

Today’s Topic: 

Final Guidance, Patient Engagement in the Design and Conduct of Medical 

Device Clinical Studies 

March 22, 2022
Final Guidance: 

Patient Engagement in the Design and Conduct of 

Medical Device Clinical Studies

Tracy L. Gray 

Patient Engagement Lead 

Patient Science and Engagement Program 

Office of Strategic Partnerships and Technology Innovation 

Center for Devices and Radiological Health 

U.S. Food and Drug Administration
Final Guidance

• Patient Engagement in the Design and Conduct of Medical Device 

Clinical Studies 

− www.fda.gov/regulatory-information/search-fda-guidance-documents/patientengagement-design-and-conduct-medical-device-clinical-studies

− Docket Number: FDA-2019-D-3846

3
Learning Objectives

• Describe background, development and contents of the patient 

engagement guidance 

• Discuss meaning of patient engagement and how patients as advisors can 

help improve clinical study design and conduct 

• Review examples of opportunities to engage patients

• Identify helpful resources when developing patient engagement 

approaches in clinical studies

4
Background on 

Patient Engagement

5
Patient Input Across Total Product Lifecycle 

6
Patient Engagement Guidance Development

7

JANUARY 2022

FINAL Guidance 

Published

SEPT 2019

Draft Guidance 

Published

MAR 2019

Discussion 

Paper Shared at 

CTTI-FDA Public 

Workshop

NOV 2018

Discussion 

Paper Shared at 

PEAC

OCT 2017

FDA’s PEAC on 

Clinical Trials

PEAC = Patient Engagement Advisory Committee 

CTTI = Clinical Trials Transformation Initiative
Key Terms and Definitions

• Patient Engagement 

− Intentional, meaningful interactions with patients that provide opportunities for 

mutual learning, and effective collaborations 

• Patients 

− 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 

− Are the individuals who directly experience the benefits and harms associated 

with medical products 

8
Key Terms and Definitions

• Study/Research Participants 

− 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 

• Patient Advisors 

− 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 

− Are not study/research participants themselves or caregivers of study/research 

participants

9
Perceived Barriers and Challenges 

to PE in Clinical Studies 

Patients’ perceptions that their input is not “allowed” or “valued”

Challenges finding patient advisors

Site investigators’ reluctance to allow sponsors to engage with patients

Logistical challenges of engaging with patient advisors

Challenges determining which patient perspectivesto include

10
Overview of Patient Engagement Guidance

11
Guidance Purpose

• Help sponsors understand how to voluntarily use patient engagement: 

− to elicit experience, perspectives, and other relevant information from patient advisors to improve 

the design and conduct of medical device clinical studies 

• Highlight benefits of engaging with patient advisors early in device development process 

• Illustrate which PE activities are generally not considered by FDA to constitute research 

or an activity subject to FDA's regulations, including regulations regarding IRB 

• Address common questions and misconceptions about collecting and submitting to FDA 

PE information regarding the design and conduct of a medical device clinical study

12
Structure of Final Guidance

• Introduction with guidance objectives specified 

• Scope 

• Definition of patient engagement 

• Questions and Answers on patient engagement in clinical studies 

• Ways in which industry might engage with patients

13
Scope of Guidance

• Focuses on application of patient engagement: 

− By using patient advisors to inform and improve design and conduct of 

medical device clinical studies 

• Does not address: 

− Study/research participant or patient advisor reimbursement or 

compensation 

− Promotion of investigational devices 

−Dissemination of clinical study results

14
Potential Benefits of Diverse 

Patient Advisor Input in Clinical Studies

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 and 

violations

Greater 

study/research 

participation by 

diverse populations

Fewer protocol 

revisions

Streamlined data 

collection resulting 

in better quality data

More relevant data 

on outcomes that 

matter to patients

15
Patient Engagement Activities

16
When Can Patient Advisors Be Involved?

Planning

During study plan 

development, 

especially in 

innovative areas or 

new targeted 

populations

Conduct

When significant 

recruitment and 

retention challenges 

occur

Future Efforts

To inform 

improvements for 

future studies

17
Some PE Activities that May Enhance 

Design and Conduct of Clinical Studies

• Informed consent improvement

• Flexible options for data collection and follow up visits 

• Recruitment barriers and study delays 

• Potential endpoints 

• Patient - Reported Outcomes (PRO) concepts 

• Patient Preference Information (PPI) study design

18
What Are Roles of IRBs and 

Other Institutional Groups in PE? 

• IRB Purpose 

− Assure protection of rights and welfare of humans who are study research 

participants 

• No IRB Involvement – for PE activities with patient advisors 

− Primarily involve interaction in a consultative or advisory capacity 

− FDA does not generally consider PE activities with patient advisors to constitute 

research or an activity subject to FDA’s regulations on their own 

• IRB Involvement 

− Interactions between study/research participants and investigators 

19
Resources on Patient Engagement

20
How Can a Sponsor Receive Feedback from FDA 

on Patient Engagement Plan or 

Patient-Centered Study Design?

• Q - Submission Program 

− Final Guidance: Requests for Feedback and Meetings for Medical Device 

Submissions: The Q Submission Program

− www.fda.gov/regulatory-information/search-fda-guidance-documents/requestsfeedback-and-meetings-medical-device-submissions-q-submission-program

21
Summary

• Patient engagement in medical device clinical investigations has value and is 

appropriate in certain circumstances 

• PE guidance provides an overview of potential value and summary of challenges and 

potential solutions related to involving patient advisors in design and conduct of clinical 

investigations 

• We encourage early interaction with FDA to obtain feedback on your approach for 

incorporating patient input in design and conduct of device investigations 

• More patient - centric device clinical studies may lead to improved efficiency and quality 

of clinical studies and greater uptake of results

22
23
Let’s Take Your Questions

• To Ask a Question: 

• Raise your hand in Zoom 

• Moderator will announce your name and invite you to ask your question 

• Unmute yourself when invited to ask your question 

• When Asking a Question: 

• Ask one question only 

• Keep question short 

• No questions about specific submissions 

• After Question is Answered: 

• Mute yourself and lower your hand 

• If you have more questions - raise your hand again

24
Thanks for Joining Today!

• Presentation and Transcript will 

be available at CDRH Learn 

• www.fda.gov/Training/CDRHLearn

• Additional questions about 

today’s presentation

• Email: DICE@fda.hhs.gov

• Upcoming Webinars 

• www.fda.gov/CDRHWebinar

25