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      Development of a Symptom-Based Patient-Reported Outcome Instrument for Functional Dyspepsia: A Preliminary Conceptual Model and an Evaluation of the Adequacy of Existing Instruments


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          The aim was to document, from the perspective of the empirical literature, the primary symptoms of functional dyspepsia (FD), evaluate the extent to which existing questionnaires target those symptoms, and, finally, identify any missing evidence that would impact the questionnaires’ use in regulated clinical trials to assess treatment efficacy claims intended for product labeling.


          A literature review was conducted to identify the primary symptoms of FD and existing symptom-based FD patient-reported outcome (PRO) instruments. Following a database search, abstracts were screened and articles were retrieved for review. The primary symptoms of FD were organized into a conceptual model and the PRO instruments were evaluated for conceptual coverage as well as compared against evidentiary requirements presented in the FDA’s PRO Guidance for Industry.


          Fifty-six articles and 16 instruments assessing FD symptoms were reviewed. Concepts listed in the Rome III criteria for FD ( n = 7), those assessed by existing FD instruments ( n = 34), and symptoms reported by patients in published qualitative research ( n = 6) were summarized in the FD conceptual model. Except for vomiting, all of the identified symptoms from the published qualitative research reports were also specified in the Rome III criteria. Only three of the 16 instruments, the Dyspepsia Symptom Severity Index (DSSI), Nepean Dyspepsia Index (NDI), and Short-Form Nepean Dyspepsia Index (SF-NDI), measure all seven FD symptoms defined by the Rome III criteria. Among these three, each utilizes a 2-week recall period and 5-point Likert-type scale, and had evidence of patient involvement in development. Despite their coverage, when these instruments were evaluated in light of regulatory expectations, several issues jeopardized their potential qualification for substantiation of a labeling claim.


          No existing PRO instruments that measured all seven symptoms adhered to the regulatory principles necessary to support product labeling. As such, the development of a new FD symptom PRO instrument is supported.

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          Most cited references22

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          Functional dyspepsia--symptoms, definitions and validity of the Rome III criteria.

          Dyspepsia refers to a heterogeneous group of symptoms that are localized in the epigastric region. Typical dyspeptic symptoms include postprandial fullness, early satiation, epigastric pain and epigastric burning, but other upper gastrointestinal symptoms such as nausea, belching or abdominal bloating often occur. Functional dyspepsia is defined as the presence of dyspeptic symptoms in the absence of an organic cause that readily explains them. The Rome III consensus proposed the subdivision of functional dyspepsia into postprandial distress syndrome (PDS), characterized by postprandial fullness and early satiation, and epigastric pain syndrome (EPS), characterized by epigastric pain or burning. Epidemiological studies in the USA and Europe confirmed the presence of both subgroups, with good separation between EPS and PDS. By contrast, other studies have found major overlap between EPS and PDS in patients with functional dyspepsia in specialist care centres in Europe and Asia. Preliminary pathophysiological studies suggest that PDS might be characterized by a higher prevalence of impaired gastric accommodation than EPS and raised duodenal eosinophil counts. Whether different treatment approaches are needed for EPS and PDS is currently unclear; only acotiamide, a new drug for the treatment of functional dyspepsia, has been found to be efficacious in PDS but not in EPS. Further randomized controlled trials testing treatment response by subgroup are urgently needed.
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            Development and psychometric evaluation of the patient assessment of upper gastrointestinal symptom severity index (PAGI-SYM) in patients with upper gastrointestinal disorders.

            Describe the development and evaluation of a new self-report instrument, the patient assessment of upper gastrointestinal disorders-symptom severity index (PAGI-SYM) in subjects with gastroesophageal reflux disease (GERD), dyspepsia, or gastroparesis.
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              Quality of life in patients with upper gastrointestinal symptoms. An improved evaluation of treatment regimens?

              There is growing interest in measuring quality of life (QoL) in medicine. Together with objective variables such as healing rate, as recorded with endoscopy, this is believed to give a better basis for evaluation of treatment regimens in patients with upper gastrointestinal diseases. A strategy for the assessment of QoL in patients with upper gastrointestinal symptoms is presented here. The QoL evaluation was based on a battery of questionnaires, covering both general and specific aspects of life. General well-being was evaluated with the Psychological General Well-being Index (PGWB), and subjective symptoms with two specific questionnaires, the Gastrointestinal Symptom Rating Scale (GSRS) and the Ulcus Esophagitis Subjective Symptoms Scale (UESS). This new strategy was applied clinically in a study including 146 outpatients with suspected peptic ulcer. Initially, the patients reported a low degree of general well-being as evaluated with the PGWB, but the values returned to those found in normal populations within 4 weeks. Evaluation of the GSRS and UESS with regard to internal consistency, construct validity, and the ability to detect changes showed them to be satisfactory. The psychometric documentation of the measures indicates that they may give reliable and clinically valid information when used for evaluation of medical treatments in upper gastrointestinal disease. Further documentation of the methods is, however, needed to establish a generally acceptable QoL assessment in gastroenterology.

                Author and article information

                (617) 374-5094 , DReasner@IronwoodPharma.com
                The Patient
                Springer International Publishing (Cham )
                28 March 2016
                28 March 2016
                : 9
                : 5
                : 409-418
                [1 ]Adelphi Values, Boston, MA USA
                [2 ]Study Endpoints, Ironwood Pharmaceuticals, Inc., 301 Binney Street, Cambridge, MA 02142 USA
                [3 ]Allergan plc., Jersey City, NJ USA
                [4 ]Pfizer, Collegeville, PA USA
                [5 ]Outcometrix, Tucson, AZ USA
                [6 ]Shire, Zug, Switzerland
                © The Author(s) 2016

                Open AccessThis article is distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0 International License (http://creativecommons.org/licenses/by-nc/4.0/), which permits any noncommercial use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made.

                Funded by: FundRef http://dx.doi.org/10.13039/100000038, U.S. Food and Drug Administration (US);
                Award ID: U01FD003865
                Award Recipient :
                Original Research Article
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                © Springer International Publishing Switzerland 2016


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