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© 2022 Clinical Data Interchange Standards Consortium, Inc. All rights reserved. 

1 Introduction

This document describes the CDISC implementation of the Exacerbations of Chronic Pulmonary Disease Tool (EXACT^®) - Patient-Reported Outcome (PRO) (EXACT) instrument.

The Evaluating Respiratory Symptoms (E-RS^™) in COPD (E-RS^™:COPD) scale is part of the EXACT instrument. It comprises 11 items (of the 14-item EXACT) which provide information specific to respiratory symptoms in stable chronic obstructive pulmonary disease (COPD). The E-RS:COPD is not administered separately from the EXACT. For this reason, a separate SDTM questionnaire supplement for the E-RS:COPD will not be developed. 

Instructions for scoring the EXACT and E-RS:COPD, done during analysis of a clinical trial, can be found in the user manuals provided from the copyright holder when copyright use permission is obtained. Both measures will have Analysis Data Model (ADaM) QRS supplements explaining the scoring and total of the results based on the user manual.

CDISC did not modify this questionnaires, ratings, and scales (QRS) instrument to meet Clinical Data Acquisition Standards Harmonization (CDASH) case report form (CRF) standards.

The representation of data collected for this instrument is based on the Study Data Tabulation Model Implementation Guide (SDTMIG) Questionnaires (QS) domain model, which can be found on the CDISC website at: https://www.cdisc.org/standards/foundational/sdtmig.

These specific implementation details for this instrument are meant to be used in conjunction with the SDTMIG. All CDISC QRS documentation packages can be found on the CDISC website at: https://www.cdisc.org/standards/foundational/qrs.

The CDISC Intellectual Property Policy can be found on the CDISC website at: https://www.cdisc.org/about/bylaws.

1.1 Representations and Warranties, Limitations of Liability, and Disclaimers

This document is a supplement to the SDTMIG for Human Clinical Trials and is covered under Appendix F of that document, which describes representations, warranties, limitations of liability, and disclaimers. Please see Appendix F of the SDTMIG for a complete version of this material.

CDISC specifies how to structure the data that has been collected in a database, not what should be collected or how to conduct clinical assessments or protocols.

Although the United States Food and Drug Administration (US FDA) has provided input with regard to this supplement, this input does not constitute US FDA endorsement of any particular instrument.

2 Copyright Status

Evidera, Inc., owns the copyright for the EXACT instrument and has granted CDISC permission to include this supplement in the CDISC library of QRS data standards supplements. Hence, CDISC developed QSTESTCD and QSTEST for each item based on the actual text on the instrument.

The CDISC documentation of this instrument consists of: (1) controlled terminology, (2) standard database structure with examples, and (3) CRF(s) annotated with the CDISC SDTMIG variables with submission values. 

Note: CDISC Controlled Terminology is maintained by National Cancer Institute (NCI) Enterprise Vocabulary Services (EVS). The most recent version should be accessed through the CDISC website at: https://www.cdisc.org/standards/terminology/controlled-terminology.

Note that the EXACT and E-RS:COPD have been validated for electronic administration (ePRO); pen-and-paper CRF administration is not recommended by the copyright holders.

CDISC has developed this documentation at no cost to the copyright holder or any additional cost to users of the instrument beyond the normal license fees charged by the copyright holder.

CDISC acknowledges principal author Nancy K. Leidy, EXACT-PRO Initiative, Evidera, Inc., for the approval to include the EXACT in the CDISC data standards.

Reference for the EXACT:

• Nancy K. Leidy, EXACT-PRO Initiative, Evidera, 7101 Wisconsin Avenue, Suite 1400, Bethesda, MD; EXACT copyright 2013, Evidera, Inc. All rights reserved.
  • This also includes the subset EXACT-Respiratory Symptoms (E-RS) questions. http://www.exactproinitiative.com

• The Exacerbations of Chronic Pulmonary Disease Tool (EXACT) Patient-Reported Outcome (PRO) USER MANUAL (Version 7.0). October 2014, EXACT© 2013, Evidera, Inc. All rights reserved.

3 The QS Domain Model

3.1 Assumptions for the QS Domain Model

All assumptions and business rules described in the SDTMIG QS domain are applicable to this supplement. Additional assumptions specific to the EXACT are listed below.

EXACT is a multiple-choice self-administered daily diary completed by respondents that clinicians may use to measure of patient-reported symptoms of COPD exacerbations. It consists of 14 items, each rated on a 5- or 6-point scale depending on the question. These items together provide information on the frequency, severity, and duration of symptom-defined exacerbations. The E-RS:COPD uses 11 of these 14 items to evaluate respiratory symptoms of COPD. Instructions for electronic data capture are described in the user manual.

1. The scale points include a text rating (e.g., "Not at all", "Slightly"). 

   1. The E-RS (EXACT-Respiratory Symptoms)* USER MANUAL (Version 4.0) May 2015 that is provided upon receiving copyright permission provides the numeric scoring of the responses for the questions. Because EXACT is a copyrighted instrument, the numeric scores cannot be provided in this supplement. Upon receiving permission, sponsors must apply these numeric responses in their SDTMIG and ADaM datasets. For this EXACT supplement, QSORRES is populated with the text description; the numeric rating is represented as "xxx" in QSSTRESC and QSSTRESN.

2. The time period of evaluation for the EXACT is populated in the QSEVINTX field. The evaluation interval for the EXACT is QSEVINTX = "EVERY EVENING BEFORE BEDTIME", as represented on the CRF.

3. The EXACT instrument includes 3 raw and domain respiratory symptom scores, a raw total score, and an EXACT total score (not represented on the CRF, but described in the user manual), that are considered as captured data and is not considered as derived in the example below. These scores may be submitted in SDTM or derived in ADaM per scoring instructions from the user manual. The E-RS (EXACT-Respiratory Symptoms)* USER MANUAL (Version 4.0) May 2015 that is provided upon receiving copyright permission provides information to calculate these items. The EXACT raw total score is computed across the 14 items and then transformed via a table lookup to obtain the EXACT total score that has a theoretical range of 0 to 100. Three raw respiratory symptom scores are also computed for breathlessness, cough and sputum, and chest symptoms. These raw respiratory scores are transformed via a table lookup to obtain the respiratory symptom domain scores that have a theoretical range of 0 to 100.  

   1. If operationally defined by the sponsor, it is the sponsor's responsibility to set the --DRVFL flag based on their eCRF process to derive subtotals and total scores. An investigator-derived score will be considered a captured score and not flagged. When subtotal and total scores are derived by the sponsor, the derived flag (--DRVFL) is set to "Y". However, when the subtotal and total scores are received from a central provider or vendor, the value would go into --ORRES and --DRVFL would be null (see SDTMIG Section 4.1.8.1, Origin Metadata for Variables).

   2. If scores are received by the sponsor, it is recommended that they are submitted to SDTM and verified in ADaM.

4. Timing of the EXACT instrument diary collection

   1. The QRS EXACT instrument diary or manual describes that it is a self-administered daily diary, completed by respondents each evening before bedtime. Any additional information regarding the timing of the diary capture should be described in a protocol.
   2. The following CDISC timing variables represent the EXACT collection timing.
      1. VISITNUM variable is represented as a null value because sponsors have different operating procedures to assign visit information relative to diary collection.
         1. QSDTC variable represents the date/time the diary information was collected by the subject and is used for the timing of this measure.
         2. QSEVINTX variable represents the evaluation interval text timing described on the instrument relative to diary collection.
5. As described in the user manual, where no diary entry exists for a given day, create a record in the dataset for that collection date. Each day a patient is followed in the study must have a record for the day. For missing diary days, a record is created for each item with QSORRES, QSSTRESC and QSSTRESN represented with missing values and QSSTAT = "NOT DONE". If a reason for not collecting an item is collected, it is represented in the QSREASND variable. Collecting this reason would be a good practice, but is not specifically described in the manual.

   1. “Presence of Data” items missing data rule:

      1. A record is created in qs.xpt for all items.
      2. If the reason for the not done record is collected, then QSSTAT = “NOT DONE” and QSREASND = reason provided (e.g., “PREFER NOT TO ANSWER”). If a reason for the not done record is not collected, the QSREASND variable is not populated,

6. Terminology

   1. QSCAT, QSTESTCD, and QSTEST values are included in CDISC Controlled Terminology.

   2. A full list of value sets for the result fields is provided in Section 4, SDTM Mapping Strategy.

3.2 Example for the EXACT QS Domain Model

The EXACT example below shows the terminology used to implement the instrument in the QS domain. This example shows the data for 1 subject collected at 7 evenings of diary data capture on the EXACT instrument. The example uses CDISC Controlled Terminology for QSTESTCD, QSTEST, and QSCAT. All original results are represented with preferred terminology in QSORRES. Based on assumption 1 above, the numeric values from the user manual are represented as "xxx" in QSSTRESC and QSSTRESN. There is no baseline observation flag represented because the user manual describes how to derive and potentially rederive the baseline EXACT domain and total scores as an analysis variable based on the subject disease status.

The table represents the items from the EXACT instrument. The timing of diary collection is explained in assumption 4. Rows 15-22 represent that the 08-Nov-2012 diary collected raw, domain, and EXACT total scores as described in assumption 3 above. These are also represented for each additional diary collection date. The subject did not fill out the EXACT diary on 09-Nov-2012 and no reason for not collecting the diary was provided. Although the reason for not completing the assessments was not collected, the protocol specified that evaluations occur nightly for 7 straight days, so the collection date is included for these missing evaluations. The remaining diary collection follows the same pattern through the completion of the dairy.

4 SDTM Mapping Strategy

This section is used for reference regarding the CRF data capture and to understand the alignment of the instrument to the SDTM QS domain. It also provides guidance on how the result variables (QSORRES, QSSTRESC, and QSSTRESN) should be populated.

Based on assumption 1 above, the numeric values from the user manual are represented as "xxx" in QSSTRESC and QSSTRESN.

QSTESTCD = "EXACT101" QSTEST = "EXACT1-Chest Feel Congested"

QSTESTCD = "EXACT106" QSTEST = "EXACT1-Chest Feel Tight"

QSTESTCD = "EXACT107" QSTEST = "EXACT1-Breathless"

QSTESTCD = "EXACT112" QSTEST = "EXACT1-Tired or Weak"

QSTESTCD = "EXACT113" QSTEST = "EXACT1-Night Sleep Disturbed"

QSTESTCD = "EXACT114" QSTEST = "EXACT1-Worried About Lung Problems"

QSTESTCD = "EXACT102" QSTEST = "EXACT1-How Often Cough"

QSTESTCD = "EXACT103" QSTEST = "EXACT1-Bring Up Mucus When Coughing"

QSTESTCD = "EXACT104" QSTEST = "EXACT1-Difficult to Bring Up Mucus"

QSTESTCD = "EXACT105" QSTEST = "EXACT1-Chest Discomfort"

QSTESTCD = "EXACT108" QSTEST = "EXACT1-Describe How Breathless"

QSTESTCD = "EXACT109" QSTEST = "EXACT1-Short Breath Personal Care"

QSTESTCD = "EXACT110" QSTEST = "EXACT1-Short Breath Indoor Activities"

QSTESTCD = "EXACT111" QSTEST = "EXACT1-Short Breath Outside Activities"

End of Document