Development of a Symptom-Focused Patient-Reported Outcome Measure for Functional Dyspepsia: The Functional Dyspepsia Symptom Diary (FDSD)

Fiona Taylor, Sophie Higgins, Robyn T Carson, Sonya Eremenco, Catherine Foley, Brian E Lacy, Henry P Parkman, David S Reasner, Alan L Shields, Jan Tack, Nicholas J Talley, Patient-Reported Outcome Consortiumʼs Functional Dyspepsia Working Group, Fiona Taylor, Sophie Higgins, Robyn T Carson, Sonya Eremenco, Catherine Foley, Brian E Lacy, Henry P Parkman, David S Reasner, Alan L Shields, Jan Tack, Nicholas J Talley, Patient-Reported Outcome Consortiumʼs Functional Dyspepsia Working Group

Abstract

Objectives: The Functional Dyspepsia Symptom Diary (FDSD) was developed to address the lack of symptom-focused, patient-reported outcome (PRO) measures designed for use in functional dyspepsia (FD) patients and meeting Food and Drug Administration recommendations for PRO instrument development.

Methods: Concept elicitation interviews were conducted with FD participants to identify symptoms important and relevant to FD patients. A preliminary version of the FDSD was constructed, then completed by FD participants on an electronic device in cognitive interviews to evaluate the readability, comprehensibility, relevance, and comprehensiveness of the FDSD, and to preliminarily evaluate its measurement properties.

Results: During concept elicitation interviews, 45 participants spontaneously reported 19 symptom concepts. Of those, seven symptoms were selected for assessment by the eight-item FDSD. Cognitive interviews with 57 participants confirmed that participants were able to comprehend and provide meaningful responses to the FDSD, and that the handheld electronic FDSD format was suitable for use in the target population. Scores of the FDSD were well-distributed among response options, item discrimination indices suggested that the FDSD items differentiate among patients with varying degrees of FD severity, and inter-item correlations suggested that no items of the FDSD were capturing redundant information. Internal consistency estimates (0.87) and construct-related validity estimates using known-groups methods were within acceptable ranges.

Conclusions: The FDSD is a content-valid PRO measure, with preliminary psychometric evidence providing support for the FDSD's items and total score. Further psychometric evaluations are recommended to more fully test the FDSD's score performance and other measurement properties in the target patient population.

Conflict of interest statement

Guarantor of the article: Fiona Taylor, MBiochem.

Specific author contributions: The authors meet criteria for authorship as recommended by the International Committee of Medical Journal Editors (ICMJE), were fully responsible for all aspects of manuscript development, and approved this manuscript for submission. Fiona Taylor and Alan L. Shields participated in planning and executing the study, interpreting the data, and drafting the manuscript. Catherine Foley participated in planning and executing the concept elicitation stage of the study; collecting, analyzing, and interpreting the data; and provided input on the manuscript. Sophie Higgins participated in planning and executing the cognitive interview stage of the study; collecting, analyzing and interpreting the data; and drafting the manuscript. Robyn T. Carson, Sonya Eremenco, and David S. Reasner provided input on the study design, interpretation of results, and manuscript. Brian Lacy, Henry Parkman, Jan Tack, and Nick J. Talley were expert panel members on the study and provided input on the study design, interpretation of results, and manuscript.

Financial support: Three PRO Consortium member companies sponsor the Functional Dyspepsia Working Group. These companies support this effort through financial, in-kind, and intellectual contributions. Funding for this Functional Dyspepsia Working Group research was provided by the following PRO Consortium member firms: Allergan Plc., Ironwood Pharmaceuticals, Inc., and Shire. The Critical Path Institute’s PRO Consortium is supported, in part, by grant number U18 FD005320 from the US Food and Drug Administration. A list of the members of the PRO Consortium is available at http://c-path.org/programs/pro/.

Potential competing interests: F.T., S.H., C.F., and A.L.S. are employees of Adelphi Values, which received payment from the sponsors to conduct the research. B.L., H.P.P., J.T., and N.J.T. received payment from the sponsors to participate as expert panel members on this study. R.T.C. is an employee of Allergan and owns stock and stock options in Allergan. S.E. is an employee of the Critical Path Institute and has no competing interests to report. B.L. serves on scientific advisory boards for Ironwood, Salix, and Prometheus. H.P.P. has no further competing interests to report. D.S.R. is a member of Albemarle Scientific Consulting and an employee of Ironwood Pharmaceuticals, and owns stock and stock options in Ironwood. J.T. has provided scientific advice to Abide Therapeutics, AlfaWassermann, Allergan, Mylan, Novartis, Rhythm, Shire, SK Life Sciences, Takeda, Theravance, Tsumura, Yuhan, and Zeria Pharmaceuticals; has received research support from Abide, Shire, Tsumura, and Zeria; and has served on the speaker bureau for Abbott, Allergan, Shire, Takeda, and Zeria. J.T. was involved in development of the Leuven Postprandial Distress Scale PRO. N.J.T. has received Grant/Research Support from NHMRC, NIH, Rome Foundation, Aus EE, Abbott Pharmaceuticals, Allergan, Datapharm, Pfizer, Salix, Prometheus Laboratories, Commonwealth Laboratories, and Janssen. Consultant/Advisory Boards: GI Therapies, Yuhan, Prometheus Laboratories, and Commonwealth Laboratories. US Patent Holder: Biomarkers of irritable bowel syndrome. N.J.T. developed the Nepean Dyspepsia Index Long and Short forms. Any views expressed in this publication represent the personal opinions of the authors, not those of their respective employers. The authors’ respective organizations were given the opportunity to review the manuscript for medical and scientific accuracy, as well as intellectual property considerations.

Figures

Figure 1
Figure 1
Literature-based conceptual model for functional dyspepsia symptoms (9). BM, bowel movement; GERD, gastroesophageal reflux disease; IBS, irritable bowel syndrome; PRO, patient-reported outcome. (a) Concepts in Rome III criteria for FD; (b) concepts assessed by existing FD instruments; (c) concepts reported by patients in published qualitative research. This figure was originally published in Taylor et al. (9).
Figure 2
Figure 2
List of functional dyspepsia signs and symptoms. *Percentages represent frequency of participant (N=45) report during concept elicitation interviews † (2).
Figure 3
Figure 3
Participant-reported functional dyspepsia concepts by subtype. *BM, bowel movement; EPS, epigastric pain-syndrome; PDS, postprandial distress syndrome.

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