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DRAFT GUIDANCE
 
 
 
  This guidance document is being distributed for comment purposes only.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publication in the Federal Register of the notice announcing the availability of the draft 
guidance.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This draft guidance, when finalized, will represent the current thinking of the Food and Drug 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   Contains Nonbinding Recommendations 
Draft  Not for Implementation  
Evaluating Respiratory Symptoms in Chronic Obstructive 
1 
Pulmonary Disease, a Patient-Reported Outcome Instrument 
2 
for the Measurement of Severity of Respiratory Symptoms in 
3 
Stable Chronic Obstructive Pulmonary Disease:  Qualification 
4 
for Exploratory Use 
5 
6  Guidance for Industry1
  
7 
  
8  DDT Type:  Clinical outcome assessment (COA) 
 
9 
  
10  DDT Tracking Number:  [DDTCOA-0000017] 
11   
12  Referenced COA: Evaluating Respiratory Symptoms in Chronic Obstructive Pulmonary 
13  Disease (E-RS: COPD) 
14   
15  Type of COA: Patient-reported outcome (PRO) instrument  
16   
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.   
20   
21  The contact information for public access to the E-RS: COPD and its user manual also 
22  appear below.  
23   
24  Section I: Concept of Interest  
25   
26  The E-RS: COPD total score measures respiratory symptoms of stable COPD. 
27   
28  Section II:  Context of Use  
29   
30  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.  
35   
36  We encourage additional research and analyses to evaluate the E-RS: COPDs 
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 
40  statement will be expanded to aid in interpretation of clinically meaningful change.  
41   
                                                 
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. 
1 

  Contains Nonbinding Recommendations 
Draft  Not for Implementation  
42  A.   Study population 
43   
44  Adult outpatients with stable COPD 
45   
46  B.   Clinical trial design  
47   
48  Superiority trial 
49   
50  C.   Endpoint positioning  
51   
52  The E-RS: COPD total score is currently qualified as an exploratory endpoint in clinical 
53  studies. 
54   
55  D.   Labeling or promotional claim(s) based on the COA 
56   
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.  
59   
60  Section III:  COA Interpretation (If Available)  
61   
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. 
65   
66  Section IV: Contact Information for Access to the Qualified COA 
67   
68  Evidera 
69  7101 Wisconsin Avenue, Suite 1400 
70  Bethesda, MD 20814 
71  exactpro@evidera.com 
72  Phone: (301) 664-7272 
73   
74  Instructions for Use in a Regulatory Submission:  Please reference DDT # [DDTCOA-
75  0000017] and this guidance in your application.  
2