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Today’s Topic: 

Final Guidance, Principles for Selecting, Developing, Modifying, and Adapting 

Patient - Reported Outcome Instruments for Use in Medical Device Evaluation 

March 01, 2022
Final Guidance: 

Principles for Selecting, Developing, Modifying, and 

Adapting Patient - Reported Outcome Instruments for 

Use in Medical Device Evaluation

Fraser Bocell, MEd, PhD 

Psychometrician, Patient Science and Engagement 

Office of Strategic Partnerships and Technology Innovation 

Center for Devices and Radiological Health
Final Guidance: 

Principles for Selecting, Developing, Modifying, and 

Adapting Patient - Reported Outcome Instruments for 

Use in Medical Device Evaluation

Fraser Bocell, MEd, PhD 

Psychometrician, Patient Science and Engagement 

Office of Strategic Partnerships and Technology Innovation 

Center for Devices and Radiological Health
Learning Objectives

• Summarize FDA’s current patient - reported outcome (PRO) guidances , as 

well as the Clinical Outcome Assessment efforts underway 

• Describe contents of CDRH final guidance on PRO instruments including: 

− Key principles 

− Steps for ensuring instruments are fit for purpose 

− Best practices for selecting, developing, modifying and adapting PRO instruments 

• List available PRO - related resources 

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Background on 

Patient-Reported Outcome Guidance Efforts

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Types of Clinical Outcome Assessments 

(COAs)

ClinRO = Clinician - Reported Outcome PRO = Patient - Reported Outcome 

ObsRO = Observer-Reported Outcome PerfO = Performance Outcome

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Biomarkers, Endpoints, and Other Tools (BEST) Resource: www.ncbi.nlm.nih.gov/books/NBK338448/
Patient - Reported Outcome Measures: Use in 

Medical Product Development to Support 

Labeling Claims

• Focused on use of PRO instruments to 

support efficacy claims made in medical 

product labeling 

• Often cited in development and review of 

PRO instruments in literature 

• Other potential uses of PRO instruments are 

not directly addressed

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Patient - Focused Drug Development (PFDD) 

Guidance Series

Guidance 1: Collecting Comprehensive and 

Representative Input 

§ Workshop held December 18, 2017 

§ Issued Final Guidance June 18, 2020

§ Workshop held October 15 - 16, 2018 

§ Issued Draft Guidance December 2019

§ Workshop held October 15-16, 2018 

§ Draft in progress

§ Workshop held December 6, 2019 

§ Draft in progress

Guidance 2: Methods to Identify What is 

Important to Patients 

Guidance 4: Incorporating Clinical Outcome 

Assessments into Endpoints for Regulatory 

Decision Making

Guidance 3: Selecting, Developing or 

Modifying Fit - for - Purpose Clinical Outcome 

Assessments 

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www.fda.gov/drugs/development-approval-process-drugs/fda-patient-focused-drug-development-guidance-series-enhancing-incorporation-patients-voice-medical
Contents of the Final Guidance: 

Principles for Selecting, Developing, Modifying, and Adapting 

Patient - Reported Outcome Instruments for Use in Medical 

Device Evaluation

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Objectives of Guidance

1. Describe principles that may be considered when using PRO 

instruments in the evaluation of medical devices 

2. Provide recommendations about importance of ensuring PRO 

instruments are fit - for - purpose 

3. Outline best practices to help ensure relevant, reliable, and sufficiently 

robust PRO instruments are developed, modified, adapted using the 

least burdensome approach

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Scope of Guidance

• Applies to PRO instruments used in medical device evaluation across 

total product life cycle 

• Intended to supplement the aforementioned resources 

• Communicates what the FDA believes are some best practices 

• Does not detail methods and steps of developing, modifying or adapting 

a PRO instrument 

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Key Principles 

1. Establish and define the concept of interest (COI) that the PRO 

instrument intends to capture 

2. Clearly identify the role of the PRO (e.g., primary, secondary, ancillary, 

effectiveness, safety) in clinical study protocol and statistical analysis plan 

3. Provide evidence showing that the PRO instrument reliably assesses COI 

4. Effectively and appropriately communicate PRO - related results in labeling 

to inform healthcare provider and patient decision making 

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Ensuring PRO Instruments are 

Fit-For-Purpose

Fit - for - purpose: A conclusion that the level of validation associated with a 

biomarker or COA is sufficient to support its context of use. 

Factors to consider: 

1. Is the concept (and change) being measured by the PRO instrument meaningful to patients? 

2. What role (e.g., primary, secondary, ancillary, effectiveness, safety) will the PRO instrument 

serve in the clinical study protocol and statistical analysis plan? 

3. Does the evidence support the PRO instrument’s use in measuring the concept of interest as 

specified in the clinical study protocol and statistical analysis plan?

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Best Practices for Least Burdensome Selection, 

Development, Modification, and Adaptation of 

Patient-Reported Outcome Instruments

Measure concepts important to patients

Ensure PRO instruments are understandable to patients

Be clear about the role of PRO instrument in the clinical study 

protocol and statistical analysis plan

Leverage existing PRO instrument and validity evidence

Consider alternative platforms and parallel development for 

generating validity evidence for PRO instruments

Collaborate with others in the pre-competitive space

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CDRH PRO Guidance: Best Practices 

Measure concepts important to patients

• Helps integrate factors important to patients in FDA benefit-risk determinations 

• Outcomes important to patients appropriately included in labeling help inform 

patient and healthcare provider conversations

Ensure patient-reported outcome instruments are understandable 

to patients

• Plain language helps ensure patients can provide informed responses

• Engage with patients to help generate evidence

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CDRH PRO Guidance: Best Practices 

Be clear about role of PRO instrument in clinical study protocol 

and statistical analysis plan

• Strength of evidence needed to support the instrument depends on its role in 

the clinical study and whether it is fit-for-purpose

Leverage existing PRO instruments and validity evidence

• Peer-reviewed literature is a starting point for identifying validity evidence 

• Existing measures may be modified or adapted, and new validity evidence may 

be needed, depending on the modification(s) made

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CDRH PRO Guidance: Best Practices 

Consider alternative platforms and parallel development for 

generating validity evidence for PRO instruments

• Early feasibility, phased clinical studies, pivotal clinical studies, post-market clinical 

studies, and/or real-world data platforms can be used to generate validity evidence

Collaborate with others in the pre-competitive space

• Consider partnering with relevant stakeholders such as patient organizations, health 

professional organizations, and research institutions

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Patient - Related Outcomes 

Resources

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• Patient - Reported Outcome Measures: Use in Medical Product 

Development to Support Labeling Claims 

www.fda.gov/regulatory-information/search-fda-guidance-documents/patient-reported-outcomemeasures-use-medical-product-development-support-labeling-claims

• Principles for Selecting, Developing, Modifying, and Adapting PatientReported Outcome Instruments for Use in Medical Device Evaluation

www.fda.gov/regulatory-information/search-fda-guidance-documents/principles-selecting-developingmodifying-and-adapting-patient-reported-outcome-instruments-use

FDA Guidances

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• Clinical Outcome Assessments in Medical Device Decision Making 

www.fda.gov/about-fda/cdrh-patient-science-and-engagement-program/clinicaloutcome-assessments-coas-medical-device-decision-making

• FDA Patient Focused Drug Development Guidance Series 

www.fda.gov/drugs/development-approval-process-drugs/fda-patient-focused-drugdevelopment-guidance-series-enhancing-incorporation-patients-voice-medical

FDA Website Resources

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Patient - Reported Outcome or other 

Clinical Outcome Assessment Questions: 

CDRH-PRO@fda.hhs.gov

Contact Us

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• New guidance complements current 2009 PRO guidance, as well as the PFDD 

guidances under development 

• Key principles highlight considerations important to evaluation of use of a PRO 

instrument in a clinical study 

• These best practices may help ensure relevant, reliable, and sufficiently robust PRO 

instruments are developed, modified, or adapted using a least burdensome approach 

• Use of PRO instruments is voluntary if other methods of measuring the concept of 

interest exist

Summary

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Let’s Take Your Questions

• To Ask a Question: 

1. Please “Raise Your Hand” 

2. Moderator will Announce Your Name to Invite You to Ask Your Question 

3. Unmute yourself when called 

• When Asking a Question: 

• Ask 1 question only 

• Keep question short 

• No questions about individual submissions 

• After Question is Answered: 

• Please lower hand 

• If you have more questions - raise your hand again

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Thanks for Joining Today!

• Presentation and Transcript will be available at CDRH Learn: 

• www.fda.gov/Training/CDRHLearn

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