scholarly journals Testing patient-reported outcome measurement equivalence in multinational clinical trials: An exemplar using the 12-item Multiple Sclerosis Walking Scale

2017 ◽  
Vol 3 (3) ◽  
pp. 205521731772874 ◽  
Author(s):  
Hussein Dib ◽  
Yusuf Tamam ◽  
Murat Terzi ◽  
Jeremy Hobart
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trials ◽  
Outcome Measurement ◽  
Measurement Equivalence ◽  
Patient Reported Outcome ◽  
Patient Reported ◽  
Testing Patient

Commentary on ‘Disability outcome measures in multiple sclerosis clinical trials’

2012 ◽  
Vol 18 (12) ◽  
pp. 1718-1720 ◽  
Author(s):  
J Gareth Noble ◽  
Lisa A Osborne ◽  
Kerina H Jones ◽  
Rod M Middleton ◽  
David V Ford
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trials ◽  
Outcome Measures ◽  
Well Being ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Health Technologies ◽  
Additional Information ◽  
Patient Reported ◽  
Disability Outcome

In order to fully understand and explore the effectiveness of any intervention for the management of multiple sclerosis (MS), it is important to have robust, valid, reliable, and universally applied measures. The recent article, ‘Disability outcome measures in multiple sclerosis clinical trials’ by Cohen, Reingold, Polman and Wolinsky (2012), explores this issue in regards to the effective measurement of MS-related disability, and the utilisation of patient-reported outcome measures, whilst highlighting the need for collaboration between the academic and clinical communities. Although it is important to examine disability measures, it is also equally important to recognise that physical function is only one aspect of a person’s experience; for example, quality of life and psychological well-being are also important aspects to assess. The application of e-health technologies and patient registers could be a useful method of gaining additional information, using patient-reported outcomes. This commentary explores these issues in relation to points raised by the Cohen et al. paper.

scholarly journals Past and Current Practice of Patient-Reported Outcome Measurement in Randomized Cancer Clinical Trials: A Systematic Review

2021 ◽  
Vol 24 (4) ◽  
pp. 585-591
Author(s):  
Johannes M. Giesinger ◽  
Fabio Efficace ◽  
Neil Aaronson ◽  
Melanie Calvert ◽  
Derek Kyte ◽  
...  
Keyword(s):  
Systematic Review ◽  
Clinical Trials ◽  
Current Practice ◽  
Outcome Measurement ◽  
Patient Reported Outcome ◽  
Cancer Clinical Trials ◽  
Patient Reported

scholarly journals Understanding the patient experience in hepatocellular carcinoma: a qualitative patient interview study

2021 ◽  
Author(s):  
Nikunj Patel ◽  
Joshua Maher ◽  
Xandra Lie ◽  
Chad Gwaltney ◽  
Afsaneh Barzi ◽  
...  
Keyword(s):  
Hepatocellular Carcinoma ◽  
Drug Development ◽  
Conceptual Model ◽  
Patient Experience ◽  
Outcome Measurement ◽  
Qualitative Interviews ◽  
Patient Reported Outcome ◽  
Weight Changes ◽  
Patient Reported ◽  
Pro Instruments

Abstract Purpose This study aimed to elucidate the patient experience of hepatocellular carcinoma (HCC) to guide patient-centered outcome measurement in drug development. Methods Patients with HCC participated in qualitative interviews to elicit disease-related signs/symptoms and impacts, using discussion guides developed from literature searches and discussions with oncologists. Interview participants rated the disturbance of their experiences (0–10 scale). A conceptual model was developed and mapped against patient-reported outcome (PRO) instruments identified from database reviews. Results Interviews were conducted with 25 individuals with HCC (68% were men; median age: 63 years; 12% Barcelona clinic liver cancer (BCLC) stage A; 32% stage B; and 56% stage C) in the USA. Fifty-one HCC-related concepts were identified from the interviews and were grouped into eight sign/symptom categories (eating behavior/weight changes; extremities [arms, legs]; fatigue and strength; gastrointestinal; pain; sensory; skin; other) and four impact categories (emotional; physical; cognitive function; other) for the conceptual model. The most prevalent and disturbing experiences across the disease stages were fatigue/lack of energy and emotional impacts such as frustration, fear, and depression. Abdominal pain and skin-related issues were particularly common and disturbing in individuals with HCC stage C. The EORTC QLQ-C30 and HCC18 were identified as commonly used PRO instruments in HCC studies and captured the relevant signs/symptoms associated with the patient experience. Conclusion Patients with HCC reported a range of signs/symptoms and impacts that negatively affect daily functioning and quality of life. Including PRO measures in HCC clinical trials can provide meaningful patient perspectives during drug development.

scholarly journals Measurement properties of patient-reported outcome measures (PROMs) in hyperhidrosis: a systematic review

2021 ◽  
Author(s):  
Michaela Gabes ◽  
Helge Knüttel ◽  
Gesina Kann ◽  
Christina Tischer ◽  
Christian J. Apfelbacher
Keyword(s):  
Systematic Review ◽  
Clinical Trials ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Measurement Properties ◽  
High Quality ◽  
High Quality Evidence ◽  
Patient Reported ◽  
Quality Evidence

Abstract Purpose To critically appraise, compare and summarize the quality of all existing PROMs that have been validated in hyperhidrosis to at least some extend by applying the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) methodology. Thereby, we aim to give a recommendation for the use of PROMs in future clinical trials in hyperhidrosis. Methods We considered studies evaluating, describing or comparing measurement properties of PROMs as eligible. A systematic literature search in three big databases (MEDLINE, EMBASE and Web of Science) was performed. We assessed the methodological quality of each included study using the COSMIN Risk of Bias checklist. Furthermore, we applied predefined quality criteria for good measurement properties and finally, graded the quality of the evidence. Results Twenty-four articles reporting on 13 patient-reported outcome measures were included. Three instruments can be further recommended for use. They showed evidence for sufficient content validity and moderate- to high-quality evidence for sufficient internal consistency. The methodological assessment showed existing evidence gaps for eight other PROMs, which therefore require further validation studies to make an adequate decision on their recommendation. The Hyperhidrosis Disease Severity Measure-Axillary (HDSM-Ax) and the short-form health survey with 36 items (SF-36) were the only questionnaires not recommended for use in patients with hyperhidrosis due to moderate- to high-quality evidence for insufficient measurement properties. Conclusion Three PROMs, the Hyperhidrosis Quality of Life Index (HidroQoL), the Hyperhidrosis Questionnaire (HQ) and the Sweating Cognitions Inventory (SCI), can be recommended for use in future clinical trials in hyperhidrosis. Results obtained with these three instruments can be seen as trustworthy. Nevertheless, further validation of all three PROMs is desirable. Systematic review registration PROSPERO CRD42020170247

scholarly journals Enabling patient-reported outcome measures in clinical trials, exemplified by cardiovascular trials

2021 ◽  
Vol 19 (1) ◽  
Author(s):  
Theresa M. Coles ◽  
Adrian F. Hernandez ◽  
Bryce B. Reeve ◽  
Karon Cook ◽  
Michael C. Edwards ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Outcome Measures ◽  
Contextual Factors ◽  
The United States ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Think Tank ◽  
Level Of Evidence ◽  
Patient Reported ◽  
Evidentiary Standards

Abstract Objectives There has been limited success in achieving integration of patient-reported outcomes (PROs) in clinical trials. We describe how stakeholders envision a solution to this challenge. Methods Stakeholders from academia, industry, non-profits, insurers, clinicians, and the Food and Drug Administration convened at a Think Tank meeting funded by the Duke Clinical Research Institute to discuss the challenges of incorporating PROs into clinical trials and how to address those challenges. Using examples from cardiovascular trials, this article describes a potential path forward with a focus on applications in the United States. Results Think Tank members identified one key challenge: a common understanding of the level of evidence that is necessary to support patient-reported outcome measures (PROMs) in trials. Think Tank participants discussed the possibility of creating general evidentiary standards depending upon contextual factors, but such guidelines could not be feasibly developed because many contextual factors are at play. The attendees posited that a more informative approach to PROM evidentiary standards would be to develop validity arguments akin to courtroom briefs, which would emphasize a compelling rationale (interpretation/use argument) to support a PROM within a specific context. Participants envisioned a future in which validity arguments would be publicly available via a repository, which would be indexed by contextual factors, clinical populations, and types of claims. Conclusions A publicly available repository would help stakeholders better understand what a community believes constitutes compelling support for a specific PROM in a trial. Our proposed strategy is expected to facilitate the incorporation of PROMs into cardiovascular clinical trials and trials in general.

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