Developing a Core Set of Outcome Measures for Behçet Disease: Report from OMERACT 2016

Objective.The Outcome Measures in Rheumatology (OMERACT) Vasculitis Working Group has been working toward developing a data-driven core set of outcome measures for use in clinical trials of Behçet’s syndrome [Behçet disease (BD)]. This paper summarizes the group’s work through OMERACT 2016, discussions during the meeting, and the future research agenda.Methods.Qualitative patient interviews were conducted among 20 patients with BD who have different types of organ involvement. A 3-round Delphi among BD experts and patients was initiated to identify domains, subdomains, and outcomes to be assessed in clinical trials of BD. The results of these studies were discussed during OMERACT 2016 and next steps were planned.Results.Patients’ perspectives and priorities were identified through qualitative interviews that identified candidate domains and subdomains for inclusion in the Delphi and characterized some shortcomings of the currently used patient-reported outcomes in BD. The first round of the Delphi was completed and several domains or subdomains were endorsed by the experts and/or the patients. Because many more items were endorsed than would be feasible to assess during a clinical trial, rating and ranking of items by physicians and patients was planned as a next critical step. The challenges of assessing specific organ system involvement was also discussed.Conclusion.The OMERACT Behçet Syndrome Working Group research program will identify core domains for assessment in BD with the goal of developing a core set of outcome measures for use in all trials of BD with the option to incorporate additional outcomes for specific organ involvement.

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Exploration, Development, and Validation of Patient-reported Outcomes in Antineutrophil Cytoplasmic Antibody–associated Vasculitis Using the OMERACT Process

The Journal of Rheumatology ◽

10.3899/jrheum.141143 ◽

2015 ◽

Vol 42 (11) ◽

pp. 2204-2209 ◽

Cited By ~ 12

Author(s):

Joanna C. Robson ◽

Nataliya Milman ◽

Gunnar Tomasson ◽

Jill Dawson ◽

Peter F. Cronholm ◽

...

Keyword(s):

Clinical Trials ◽

Outcome Measures ◽

Working Group ◽

Patient Reported Outcomes ◽

Outcome Measurement ◽

Antineutrophil Cytoplasmic Antibody ◽

International Classification Of Functioning ◽

Measurement Information ◽

Core Set ◽

Patient Reported

Objective.Antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) is a group of linked multisystem life- and organ-threatening diseases. The Outcome Measures in Rheumatology (OMERACT) vasculitis working group has been at the forefront of outcome development in the field and has achieved OMERACT endorsement of a core set of outcomes for AAV. Patients with AAV report as important some manifestations of disease not routinely collected through physician-completed outcome tools; and they rate common manifestations differently from investigators. The core set includes the domain of patient-reported outcomes (PRO). However, PRO currently used in clinical trials of AAV do not fully characterize patients’ perspectives on their burden of disease. The OMERACT vasculitis working group is addressing the unmet needs for PRO in AAV.Methods.Current activities of the working group include (1) evaluating the feasibility and construct validity of instruments within the PROMIS (Patient-Reported Outcome Measurement Information System) to record components of the disease experience among patients with AAV; (2) creating a disease-specific PRO measure for AAV; and (3) applying The International Classification of Functioning, Disability and Health to examine the scope of outcome measures used in AAV.Results.The working group has developed a comprehensive research strategy, organized an investigative team, included patient research partners, obtained peer-reviewed funding, and is using a considerable research infrastructure to complete these interrelated projects to develop evidence-based validated outcome instruments that meet the OMERACT filter of truth, discrimination, and feasibility.Conclusion.The OMERACT vasculitis working group is on schedule to achieve its goals of developing validated PRO for use in clinical trials of AAV.

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Current Status, Goals, and Research Agenda for Outcome Measures Development in Behçet Syndrome: Report from OMERACT 2014

The Journal of Rheumatology ◽

10.3899/jrheum.141147 ◽

2015 ◽

Vol 42 (12) ◽

pp. 2436-2441 ◽

Cited By ~ 12

Author(s):

Gulen Hatemi ◽

Yesim Ozguler ◽

Haner Direskeneli ◽

Alfred Mahr ◽

Ahmet Gul ◽

...

Keyword(s):

Clinical Trials ◽

Outcome Measures ◽

Working Group ◽

Research Agenda ◽

Composite Measure ◽

Behçet Syndrome ◽

Advantages And Disadvantages ◽

Behcet Syndrome ◽

Core Set ◽

Organ Specific

Objective.There is an unmet need for reliable, validated, and widely accepted outcomes and outcome measures for use in clinical trials in Behçet syndrome (BS). Our report summarizes initial steps taken by the Outcome Measures in Rheumatology (OMERACT) vasculitis working group toward developing a core set of outcome measures for BS according to the OMERACT methodology, including the OMERACT Filter 2.0, and discussions during the first meeting of the BS working group held during OMERACT 12 (2014).Methods.During OMERACT 12, some of the important challenges in developing outcomes for BS were outlined and discussed, and a research agenda was drafted.Results.Among topics discussed were the advantages and disadvantages of a composite measure for BS that evaluates several organs/organ systems; bringing patients and physicians together for discussions about how to assess disease activity; use of organ-specific measures developed for other diseases; and the inclusion of generic, disease-specific, or organ-specific measures. The importance of incorporating patients’ perspectives, concerns, and ideas into outcome measure development was emphasized.Conclusion.The planned research agenda includes conducting a Delphi exercise among physicians from different specialties that are involved in the care of patients with BS and among patients with BS, with the aim of identifying candidate domains and subdomains to be assessed in randomized clinical trials of BS, and candidate items for a composite measure. The ultimate goal of the group is to develop a validated and widely accepted core set of outcomes and outcome measures for use in clinical trials in BS.

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Reconciling Healthcare Professional and Patient Perspectives in the Development of Disease Activity and Response Criteria in Connective Tissue Disease–related Interstitial Lung Diseases

The Journal of Rheumatology ◽

10.3899/jrheum.131251 ◽

2014 ◽

Vol 41 (4) ◽

pp. 792-798 ◽

Cited By ~ 24

Author(s):

Lesley Ann Saketkoo ◽

Shikha Mittoo ◽

Sid Frankel ◽

Daphne LeSage ◽

Catherine Sarver ◽

...

Keyword(s):

Clinical Trials ◽

Connective Tissue ◽

Connective Tissue Disease ◽

Outcome Measures ◽

Working Group ◽

Lung Diseases ◽

Interstitial Lung Diseases ◽

Patient Perspectives ◽

High Morbidity ◽

Core Set

Interstitial lung diseases (ILD), including those related to connective tissue disease (CTD), and idiopathic pulmonary fibrosis (IPF) carry high morbidity and mortality. Great efforts are under way to develop and investigate meaningful treatments in the context of clinical trials. However, efforts have been challenged by a lack of validated outcome measures and by inconsistent use of measures in clinical trials. Lack of consensus has fragmented effective use of strategies in CTD-ILD and IPF, with a history of resultant difficulties in obtaining agency approval of treatment interventions. Until recently, the patient perspective to determine domains and outcome measures in CTD-ILD and IPF had never been applied. Efforts described here demonstrate unequivocally the value and influence of patient involvement on core set development. Regarding CTD-ILD, this is the first OMERACT working group to directly address a manifestation/comorbidity of a rheumatic disease (ILD) as well as a disease not considered rheumatic (IPF). The OMERACT 11 proceedings of the CTD-ILD Working Group describe the forward and lateral process to include both the medical and patient perspectives in the urgently needed identification of a core set of preliminary domains and outcome measures in CTD-ILD and IPF.

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Development of a Provisional Core Domain Set for Polymyalgia Rheumatica: Report from the OMERACT 12 Polymyalgia Rheumatica Working Group

The Journal of Rheumatology ◽

10.3899/jrheum.141179 ◽

2015 ◽

Vol 43 (1) ◽

pp. 182-186 ◽

Cited By ~ 19

Author(s):

Toby Helliwell ◽

Elisabeth Brouwer ◽

Colin T. Pease ◽

Rodney Hughes ◽

Catherine L. Hill ◽

...

Keyword(s):

Clinical Trials ◽

Literature Review ◽

Outcome Measures ◽

Polymyalgia Rheumatica ◽

Working Group ◽

Face Validity ◽

Measurement Instruments ◽

Core Domain ◽

Core Set ◽

Core Domain Set

Objective.The Outcome Measures in Rheumatology (OMERACT) polymyalgia rheumatica (PMR) working group aims to develop a core set of outcome measures to be used in clinical trials for PMR. Previous reports from OMERACT 11 included a qualitative study of the patient experience and a preliminary literature review.Methods.A 3-round Delphi survey of clinicians and patients with PMR was undertaken to identify a candidate core domain set for PMR research. Additionally, a literature review of outcome measures and their respective measurement instruments was undertaken. Meetings of patient research partners and clinicians were convened to review face validity of the provisional core domain set, which was subsequently presented and discussed at the OMERACT 12 congress.Results.Of the 60 clinicians taking part in round 1, 55 took part in round 2 and 51 in round 3. Of the 55 patients who took part in round 1, 46 and 35 took part in subsequent rounds. In total, 91% of participants in round 3 deemed the resulting draft core domain set reasonable. The literature review identified 28 studies for full review. Measurement instruments for each proposed domain were identified. Clinicians are highly aware of glucocorticoid-related adverse effects, but there is relatively little evidence about their true prevalence and severity, especially in PMR.Conclusion.A provisional core domain set, presented for clinical trials in PMR, comprises acute phase markers, physical function, death, glucocorticoid-related adverse events, and development of giant cell arteritis. Measurement instruments are suggested that may cover each domain, but these require formal validation for clinical trials in PMR.

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The OMERACT Core Set of Outcome Measures for Use in Clinical Trials of ANCA-associated Vasculitis

The Journal of Rheumatology ◽

10.3899/jrheum.110276 ◽

2011 ◽

Vol 38 (7) ◽

pp. 1480-1486 ◽

Cited By ~ 62

Author(s):

PETER A. MERKEL ◽

SIBEL Z. AYDIN ◽

MAARTEN BOERS ◽

HANER DIRESKENELI ◽

KAREN HERLYN ◽

...

Keyword(s):

Clinical Trials ◽

Outcome Measures ◽

Outcome Measurement ◽

Measurement Instrument ◽

The United States ◽

Anca Associated Vasculitis ◽

Core Set ◽

Patient Reported ◽

Development Evaluation ◽

Multicenter Clinical Trials

There has been a marked increase in the past 15 years in the number and quality of clinical trials in the idiopathic inflammatory vasculitides, especially the small-vessel vasculitides known as antineutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis [AAV; granulomatosis, with polyangiitis (Wegener’s)]. These trials have been conducted by multicenter, international groups in Europe and the United States with financial support provided by government agencies and biopharmaceutical companies. This increased clinical trial activity in vasculitis has been accompanied by the development and validation of new outcome measures — a challenging process for these complex, multiorgan system diseases. The international OMERACT Vasculitis Working Group has developed and implemented an iterative research agenda that has utilized accumulated experience and datasets from several multicenter clinical trials and large cohort studies. This work has led to the development, evaluation, validation, and endorsement, through the OMERACT consensus and validation processes, of a “core set” of outcome measurements for use in clinical trials of AAV. The core set includes domains of disease activity, damage assessment, patient-reported outcomes, and mortality; there is at least one validated outcome measurement instrument available for each domain. This report reviews the domains of illness in AAV included in the OMERACT core set, describes the instruments validated to measure these domains, and presents the approved core set.

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Defining Outcome Measures for Psoriatic Arthritis: A Report from the GRAPPA-OMERACT Working Group

The Journal of Rheumatology ◽

10.3899/jrheum.170150 ◽

2017 ◽

Vol 44 (5) ◽

pp. 697-700 ◽

Cited By ~ 18

Author(s):

Alexis Ogdie ◽

Maarten de Wit ◽

Kristina Callis Duffin ◽

Willemina Campbell ◽

Jeffrey Chau ◽

...

Keyword(s):

Clinical Trials ◽

Psoriatic Arthritis ◽

Outcome Measures ◽

Working Group ◽

Development Process ◽

Outcome Measurement ◽

Core Outcome ◽

Core Domain ◽

Core Set ◽

Core Domain Set

The Group for Research and Assessment of Psoriasis and Psoriatic Arthritis (GRAPPA)-Outcome Measures in Rheumatology (OMERACT) Psoriatic Arthritis (PsA) Core Set working group recently published the updated 2016 psoriatic arthritis (PsA) core domain set, a set of disease features that should be measured in all clinical trials. At the GRAPPA annual meeting in July 2016, the PsA working group presented the updated PsA core domain set endorsed by 90% of participants at OMERACT in May 2016 and drafted a roadmap for the development of the PsA core outcome measurement set. In this manuscript, we review the development process of the PsA core domain set and the ongoing and proposed work streams for development of a PsA core measurement set.

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Clinical outcome measures for progressive MS trials

Multiple Sclerosis Journal ◽

10.1177/1352458517729465 ◽

2017 ◽

Vol 23 (12) ◽

pp. 1627-1635 ◽

Cited By ~ 19

Author(s):

Daniel Ontaneda ◽

Jeffrey A Cohen ◽

Maria Pia Amato

Keyword(s):

Multiple Sclerosis ◽

Clinical Trials ◽

Clinical Outcome ◽

Outcome Measures ◽

Treatment Options ◽

Unmet Need ◽

Screening Tests ◽

Future Research ◽

Neuroprotective Effects ◽

Patient Reported

Treatment options for progressive multiple sclerosis remain the main unmet need of the field. As the understanding of multiple sclerosis (MS) pathogenesis improves, new pathways and molecules will be tested for potential reparative, remyelinating, or neuroprotective effects. The clinical outcomes used will determine successful demonstration of beneficial treatment effects to regulatory agencies, clinicians, and persons with MS. This review focuses on clinical outcome measures including the Expanded Disability Status Scale, Multiple Sclerosis Functional Composite, and novel composite measures of disability. The paper also covers cognitive outcomes and screening tests for use in clinical trials. The growing importance of patient-reported outcomes and their suitability for clinical trials is also presented. The review aims to create consensus in regard to these topics and suggestions for future research.

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

Health and Quality of Life Outcomes ◽

10.1186/s12955-021-01800-1 ◽

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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