Guidance Snapshots are a communication tool and are not a substitute for the guidance document.

To learn more about Patient-Focused Drug Development, read the guidance: 

http://www.fda.gov/regulatory-information/search-fda-guidance-documents/patient-focused-drugdevelopment-selecting-developing-or-modifying-fit-purpose-clinical-outcome www.fda.gov 

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Patient-Focused Drug Development: Selecting, Developing, 

or Modifying Fit-for-Purpose Clinical Outcome Assessments 

Draft Guidance 

Why is this guidance important? 

This guidance helps ensure that clinical outcome assessments (COAs) used 

during medical product development measure what matters to patients; are 

clear about what was measured; appropriately evaluate the effectiveness, 

tolerability, and safety of treatments; and avoid misleading claims. 

What is recommended in the guidance? 

This guidance provides recommended approaches for selecting, modifying, 

developing, and validating measurements in clinical trials that evaluate 

outcomes important to patients. 

Clinical Outcome Assessment Key Points 

A COA IS A MEASURE THAT DESCRIBES HOW A PATIENT FEELS, FUNCTIONS, OR SURVIVES. THERE ARE 

FOUR TYPES OF COAS: 

A CONCEPT OF INTEREST AND THE CONTEXT OF USE SHOULD BE EXPLICITLY DEFINED 

• Patient-reported outcome (PRO) measures 

• Observer-reported outcome (ObsRO) measures 

• Clinician-reported outcome (ClinRO) measures 

• Performance outcome (PerfO) measures 

• Concept of Interest is the aspect of an individual’s • Context of Use should clearly specify the way COA 

experience or clinical, biological, physical, or scores will be used as the basis for an endpoint, 

functional state that the COA is intended to including the interpretation of the endpoint results 

capture (reflect). in a medical product development program. 

COA SHOULD BE FIT FOR PURPOSE 

A COA is considered fit for purpose when “the level of validation associated with a medical 

product development tool is sufficient to support its context of use” (BEST (Biomarkers, 

Endpoints and Other Tools) Resource, 2016). 
Guidance Snapshots are a communication tool and are not a substitute for the guidance document.

To learn more about Patient-Focused Drug Development, read the guidance: 

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/patient-focused-drugdevelopment-selecting-developing-or-modifying-fit-purpose-clinical-outcome www.fda.gov 

 

 

Roadmap for Developing a Fit-For-Purpose, Patient-Focused COA 

This is a general roadmap for developing fit-for-purpose, patient-focused COAs in clinical trials. Sponsors and COA developers are 

not required to use this approach, and it may not fit every development program. FDA recommends sponsors seek FDA input as 

early as possible and throughout medical product development to ensure COAs are fit-for-purpose for the intended context of use. 

UNDERSTANDING CONCEPTUALIZING SELECTING/DEVELOPING THE 

THE DISEASE CLINICAL BENEFITS OUTCOME MEASURE 

OR CONDITION & RISKS 

COA exists for Use existing COA • Patient/caregiver • Identify 

FIT-FORPURPOSE 

COA 

• COI & COU 

clearly 

described

• Clear 

rationale 

• Sufficient 

evidence 

to justify 

rationale 

No COA exists for 

COI and COU 

Search for 

existing 

COA 

measuring

concept 

of interest 

in context 

of use 

COI, can be used 

perspectives concept(s) of unmodified for COU 

interest (COI), Select • Natural history i.e., how a patient clinical of the disease feels, functions, outcome or condition or survives assessment 

• Patient (COA) type: • Define context subpopulations of use (COU) for PRO, Collect evidence and modify 

• Health care ObsRO, COA exists for COI, COA as necessary clinical trial but might need to environment ClinRO, be modified for COU or PerfO • Other expert 

measure input 

(healthcare 

providers, 

payers, Develop new COA and regulators) empirically evaluate 
Guidance Snapshots are a communication tool and are not a substitute for the guidance document.

To learn more about Patient-Focused Drug Development, read the guidance: 

http://www.fda.gov/regulatory-information/search-fda-guidance-documents/patient-focused-drugdevelopment-selecting-developing-or-modifying-fit-purpose-clinical-outcome

Eight Evidence-Based Considerations for Supporting a COA as 

Fit-for-Purpose 

Evidence collected in support of the use of a COA should support the rationale that explains how and 

why the specific COA is expected or intended to work. This table lists eight considerations that should 

be included in the rationale and supporting evidence or justification section of submissions to FDA. It is 

important for FDA to understand each part of a sponsor’s rationale and the evidence being offered in 

support of each part. The considerations listed below are likely but not necessarily needed in the rationale 

for a specific COA, concept of interest, and context of use. Each rationale can be tailored to the specific 

situation. 

The reason for the choice of type of COA (i.e., PRO, ObsRO, ClinRO, or 

PerfO) selected to assess the concept of interest is clear. 

All important aspects of the concept of interest are covered by the 

chosen COA. 

Respondents understand the instructions and items/tasks of the 

measure as intended by the measure developer. 

Scores of the COA are not be overly influenced by processes/concepts 

that are not part of the concept of interest. 

The method of scoring responses to the COA is appropriate for assessing 

the concept of interest. 

Scores from the COA correspond to the specific health experience(s) the 

patient has related to the concept of interest. 

Scores are sufficiently sensitive to reflect clinically meaningful changes 

within patients over time in the concept of interest within the context of 

use. 

Differences in COA scores are interpreted and communicated clearly in 

terms of the expected impact on patients’ experiences. 

A 

B 

C 

D 

E 

F 

G 

H 

www.fda.gov 
 

What is the difference between a COA, a COA score, and an endpoint? 

This guidance differentiates a COA, from a COA score, and an endpoint in the following ways: 

COA COA SCORE ENDPOINT 

A COA score refers to 

any numeric or rated 

values generated by a COA 

through a standardized 

process. A COA might 

produce more than one 

type of score, especially 

An endpoint is a 

if the COA is designed to 

measure more than one 

question (e.g., mean COA 

concept. randomization). 

precisely defined variable 

intended to reflect an 

outcome of interest 

that is statistically 

analyzed to address 

a particular research 

Score at 12 weeks postMedical Product Timeline – When to Apply the Guidance Recommendations? 

A COA is a measure 

that describes or reflects 

how a patient feels, 

functions, or survives and 

includes any instructions, 

administration materials, 

content, formatting, and 

scoring rules. 

During clinical development: When a sponsor is developing or significantly modifying a COA, FDA encourages 

sponsors to request a meeting with FDA to discuss plans early in the development process. Earlier trials represent 

an opportune time to evaluate measurement properties of COAs, and sponsors are encouraged to include 

prospectively planned analyses to inform subsequent trials. If this is not a feasible option, FDA recommends 

conducting a standalone observational study prior to the initiation of a registration trial(s) to aid in the development 

of a fit-for-purpose COA measure(s). 

www.fda.gov 

Guidance Snapshots are a communication tool and are not a substitute for the guidance document. 

To learn more about Patient-Focused Drug Development, read the guidance: 

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/patient-focused-drugdevelopment-selecting-developing-or-modifying-fit-purpose-clinical-outcome.gov

To see additional Guidance Snapshots, check out the pilot program: 

https://www.fda.gov/drugs/guidances-drugs/guidance-snapshot-pilot 

Guidance Recap Podcast – Hear Highlights Straight 

from FDA Staff 

Speakers: David Reasner, PhD, Director of the Division of Clinical Outcome Assessment, 

and Laura Lee Johnson, PhD, Director for the Division of Biometrics III. 

Click here to listen Click here to read transcript 

Continue the Conversation 

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