Evaluating Respiratory Symptoms in Chronic Obstructive Pulmonary Disease, a Patient-Reported Outcome Instrument for the Measurement of Severity of Respiratory Symptoms in Stable Chronic Obstructive Pulmonary Disease: Qualification for Exploratory Use

Administrative draft guidance, not enacted legislation. This captured publisher edition preserves the listed document and attachments; later developments are not independently consolidated.

Attachment to
Guidance on Qualification Process for Drug
Development Tools
Evaluating Respiratory Symptoms in Chronic
Obstructive Pulmonary Disease, a Patient-Reported
Outcome Instrument for the Measurement of Severity of
Respiratory Symptoms in Stable Chronic Obstructive
Pulmonary Disease: Qualification for Exploratory Use
DRAFT GUIDANCE
This guidance document is being distributed for comment purposes only.
Comments and suggestions regarding this draft document should be submitted within 90 days of
publication in the Federal Register of the notice announcing the availability of the draft
guidance. Submit electronic comments to http://www.regulations.gov. Submit written
comments to the Division of Dockets Management (HFA-305), Food and Drug Administration,
5630 Fishers Lane, rm. 1061, Rockville, MD 20852. All comments should be identified with
the docket number listed in the notice of availability that publishes in the Federal Register.
For questions regarding this draft document, contact Dr. Elektra Papadopoulos at 301-796-0900.
This draft guidance, when finalized, will represent the current thinking of the Food and Drug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 responsible
for this guidance as listed on the title page.
U.S. Department of Health and Human Services
Food and Drug Administration
Center for Drug Evaluation and Research (CDER)
March 2016
Clinical/Medical
2373295dft.doc
3/15/2016

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Contains Nonbinding Recommendations
Draft — Not for Implementation
1 	Evaluating Respiratory Symptoms in Chronic Obstructive
2 	Pulmonary Disease, a Patient-Reported Outcome Instrument
3 	for the Measurement of Severity of Respiratory Symptoms in
4 	Stable Chronic Obstructive Pulmonary Disease: Qualification
for Exploratory Use
6 	Guidance for Industry1
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8 	DDT Type: Clinical outcome assessment (COA)
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DDT Tracking Number: [DDTCOA-0000017]
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12 	Referenced COA: Evaluating Respiratory Symptoms in Chronic Obstructive Pulmonary
13 	Disease (E-RS: COPD)
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Type of COA: Patient-reported outcome (PRO) instrument
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17 	The Center for Drug Evaluation and Research (CDER) has determined that the E-RS:
18 	COPD is qualified for exploratory use as a PRO instrument to measure respiratory
19 	symptoms in patients with stable COPD in the context of use described below.
21 	The contact information for public access to the E-RS: COPD and its user manual also
22 	appear below.
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24 	Section I: Concept of Interest
26 	The E-RS: COPD total score measures respiratory symptoms of stable COPD.
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28 	Section II: Context of Use
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The E-RS: COPD total score is qualified for exploratory use as a PRO instrument to
31 	measure respiratory symptoms of stable COPD in clinical studies. Additional
32 	development work is needed to further assess measurement properties, including the
33 	ability to detect clinically meaningful change with treatment or to assess the effect of
34 	treatment on reducing respiratory symptoms from baseline levels.
36 	We encourage additional research and analyses to evaluate the E-RS: COPD’s
37 	longitudinal measurement properties including the amount of change in an individual
38 	patient that can be considered meaningful for use in the interpretation of effectiveness.
39 	We expect that as further experience with the instrument is gained, the qualification
statement will be expanded to aid in interpretation of clinically meaningful change.
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1 This guidance has been prepared by the Office of New Drugs in the Center for Drug Evaluation and
Research at the Food and Drug Administration.
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Contains Nonbinding Recommendations
Draft — Not for Implementation
42 	A. Study population
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44 	Adult outpatients with stable COPD
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46 	B. Clinical trial design
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48 	Superiority trial
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50 	C. Endpoint positioning
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52 	The E-RS: COPD total score is currently qualified as an exploratory endpoint in clinical
53 	studies.
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55 	D. Labeling or promotional claim(s) based on the COA
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57 	The E-RS: COPD total score ultimately is intended to support labeling claims related to
58 	change in overall respiratory symptoms of stable COPD.
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60 	Section III: COA Interpretation (If Available)
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62 	We recommend that the proposed responder definitions be evaluated further. When
63 	designing clinical trials, sponsors should discuss with the appropriate CDER review
64 	division how E-RS: COPD may be used.
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66 	Section IV: Contact Information for Access to the Qualified COA
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68 	Evidera
69 	7101 Wisconsin Avenue, Suite 1400
70 	Bethesda, MD 20814
71 	exactpro@evidera.com
72 	Phone: (301) 664-7272
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74 	Instructions for Use in a Regulatory Submission: Please reference DDT # [DDTCOA-
75 	0000017] and this guidance in your application.
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