the journal of rheumatology volume 41, no. 1 omeract 11 ... · the journal of rheumatology volume...

145Simon, et al: Introduction

Personal non-commercial use only. The Journal of Rheumatology Copyright © 2014. All rights reserved.

EXECUTIVE COMMITTEE Clifton Bingham, USAMaarten Boers, Netherlands Peter Brooks, AustraliaPhilip Conaghan, UKMaria-Antonietta D’Agostino, FranceLaure Gossec, FranceJohn Kirwan, UKRobert Landewé, NetherlandsLyn March, AustraliaLee Simon, USAJasvinder Singh, USAVibeke Strand, USAPeter Tugwell, CanadaGeorge Wells, Canada

FELLOWS Mary Bach, USALaura Coates, UKAndrea Delle Sedie, ItalyMaria Del Grande, SwitzerlandCatia Duarte, PortugalStephanie Finzel, GermanyIda K. Haugen, NorwayRobert Hemke, NetherlandsJacob Jaremko, Canada

Ying Ying (Katy) Leung, SingaporeYiu Tak Leung, USAElisabeth Lie, NorwaySarah Mackie, UKNataliya Milman, CanadaShikha Mittoo, CanadaSarah Ohrndorf, GermanyJennifer O’Neill, CanadaAna-Maria Orbai, USAKristine Phillips, USAJoanna Robson, UKKenneth Tang, CanadaGunnar Tomasson, USA

DELEGATES Harris Ahmad, USAHelene Alexanderson, SwedenRieke Alten, GermanySibel Zehra Aydin, TurkeyMarina Backhaus, GermanySubhashis Banerjee, USASusan Bartlett, CanadaDorcas Beaton, CanadaBirgitta Benda, USAPaul Bird, AustraliaClaire Bombardier, Canada

Annelies Boonen, NetherlandsAilsa Bosworth, UKPernille Boyesen, NorwayGeorge Bruyn, NetherlandsVivian Bykerk, CanadaIna Campbell, CanadaVinod Chandran, CanadaThomas Chong, New ZealandErnest Choy, UKRobin Christensen, DenmarkLisa Christopher-Stine, USAJolanda Cibere, CanadaMary Cifaldi, USAMaria Paz Collado Ramos, SpainBernard Combe, FranceChristina Cornell, USAOmar Dabbous, USANicola Dalbeth, New ZealandMaarten deWit, NetherlandsJulie DiCarlo, USAHaner Direskeneli, TurkeyAndrea Doria, CanadaMaxime Dougados, FranceKrysia Dziedzic, UKEmily Edson Heredia, USAN. Lawrence Edwards, USA

OMERACT 11: International Consensus Conference on Outcome Measures in Rheumatology

www.jrheum.orgDownloaded on November 13, 2020 from

http://www.jrheum.org/

146 The Journal of Rheumatology 2014; 41:1; doi:10.3899/jrheum.130937

Bo Ejbjerg, DenmarkMartin Englund, SwedenSait Burak Erer, TurkeyReuben Escorpizo, SwitzerlandBruno Fautrel, FranceJose Luis Fernandez Sueiro, SpainOliver FitzGerald, IrelandViolaine Foltz, FranceSidney Frankel, CanadaJane Freeston, UKJames Fries, USADaniel Furst, USACarol Gaich, USAFrederique Gandjbakhch, FranceDon Gebhart, USADafna Gladman, CanadaNiti Goel, USARebecca Grainger, New ZealandFrancis Guillemin, FranceSusan Halliday, UKHilde Berner Hammer, NorwayDavid Harrison, USAGulen Hatemi, TurkeyMieke Hazes, NetherlandsTurid Heiberg, NorwayPhilip Helliwell, UKChristina Hernandez Diaz, MexicoMarta Herrero, SpainSarah Hewlett, UKCatherine Hofstetter, CanadaRobert Holt, USARod Hughes, UKAnnamaria Iagnocco, ItalyKei Ikeda, JapanMariko Ishimori, USA Robert Jackson, USAMarkus John, SwitzerlandSandrine Jousse-Joulin, FranceSusanne Juhl Pedersen, DenmarkGurjit Kaeley, USAKeith Kanik, USAJeffrey Kent, USATanaz Kermani, USADinesh Khanna, USAMargreet Kloppenburg, NetherlandsMarion Kortekaas, NetherlandsGerald Krueger, USATore Kvien, Norway

Diane Lacaille, CanadaRobert Lambert, CanadaRobert Landewé, NetherlandsManuela LeBars, FranceRichard Leff, USAAmye Leong, USADaphne Lesage, USATong Li, USASiri Lillegraven, NorwayDamien Loeuille, FranceIsabelle Logeart, FranceMaria Angeles Lopez-Olivo, USAJolanda Luime, NetherlandsRaashid Luqmani, UKAnne Lyddiatt, CanadaMario Maas, NetherlandsDavid Magnusson, SwedenWalter Maksymowych, CanadaMichael Maldonado, USALisa Marshall, USAMichelle Marshall, UKEric Matteson, USAEllen Matzkin, USALara Maxwell, CanadaJames May, USAEllen McCroskery, USANeil McHugh, UKFiona McQueen, New ZealandPhilip Mease, USAPeter Merkel, USAPatricia Minnock, IrelandIngrid Moller, SpainPamela Montie, CanadaEsi Morgan DeWitt, USAEsperanza Naredo, SpainJonathan Nelsen, USAEnkeleida Nikai, NetherlandsAlexis Ogdie-Beatty, USAJennifer O’Neill, CanadaBrian Ortmeier, USAMikkel Ostergaard, DenmarkJordi Pardo, CanadaColin Pease, UKCharles Peterfy, USAElisabeth Piault-Louis, USACarlos Pineda, MexicoChristoph Pohl, GermanyRichard Polisson, USA

Tamara Rader, CanadaHelga Radner, AustriaVeena Ranganath, USAPamela Richards, UKKenneth Saag, USARoger Sabata, SpainLesley Saketkoo, USACatherine Sarver, USAPiercarlo Sarzi Puttini, ItalyGeorge Schett, GermanyNaomi Schlesinger, USAPhilip Seo, USAJeff Sherman, USAVictor Sloan, USAWillemijntje Smeets, NetherlandsMaria Simona Stoenoiu, BelgiumMaria Suarez-Almazor, USAJohn Sundy, USAJean Tayar, USAMark Tengowski, USAAlan Tennant, UKLene Terslev, DenmarkCaroline Terwee, NetherlandsWilliam Tonkins, USANikolay Tzaribachev, GermanyTill Uhlig, NorwayRaymond Urbanski, USADesiree van der Heijde, NetherlandsHubert van Hoogstraten, NetherlandsMarion van Rossum, NetherlandsAna Beatriz Vargas, BrazilDouglas Veale, IrelandGust Verbruggen, BelgiumJosep Verges, SpainSuzanne Verstappen, UKMarieke Voshaar, NetherlandsRichard Wakefield, UKUlrich Weber, SwitzerlandMichael Weiswasser, GermanyJames Witter, USAThasia Woodworth, UKGlen Wunderlich, CanadaDeborah Zarin, USA

OMERACT SECRETARIAT Shawna Grosskleg, CanadaKerry O’Brien, Canada






Introduction

OMERACT 11 — International Consensus Conferenceon Outcome Measures in Rheumatology Clinical TrialsThe biennial OMERACT 11 meeting was held in Pinehurst,North Carolina, USA, from May 12 to 17, 2012, with partic-ipants from North America, Europe, Asia, and Australia.One hundred ninety-eight attendees enjoyed the rural butmanicured ambience of this famous golf retreat; however, asusual, given the grueling daily dawn-to-late-night scheduledmeetings for discussions, few attendees were able toexperience the famous golf courses. OMERACT partici-pants came from a range of backgrounds and expertise, withclinical outcome methodologists who work in academicenvironments, regulatory agencies, pharmaceutical andbiotech companies or clinical research organizations; clini-cians with an interest in outcomes research; fellows;trainees; and patients. As has been the case for the last 10 years, patients played

an integral part in each aspect of the meeting, as well asspending time engaged in detailed discussions in their owndedicated sessions. Patient participation has proven to beextraordinarily informative in the development ofOMERACT because we are attempting to develop outcomemeasures that are clinically relevant — and patient input isat the core of determining what might or might not be clini-cally relevant. At this meeting, 18 people living withrheumatic diseases being addressed by the working partieswere an important constituency, providing unique perspec-tives that continually helped to reorient the larger audienceconcerning disease effects. In addition to a commitment to patient participants in all

aspects of our work, we are committed to further developingthe field of outcome measure research by requiring eachworking group to provide a leadership role for their identifiedfellow. We collaborated with the European League AgainstRheumatism and the American College of Rheumatology,who provided support for 5 fellows each. Twenty-two fellowsparticipated within the meeting, with additional separatesessions including presentation of their abstracts of workwithin each OMERACT working group, which werecritiqued by senior investigators involved in OMERACT andin outcomes research. Additionally, patients and fellows eachhad a separate daily discussion “track” including introduc-tions to sessions for the following day. This provided a morepersonalized experience, to foster further development ofpatient research partners and to encourage young investi-gators to engage in outcomes research. The traditional format was divided between 1 module, 5

workshops, and 10 special interest groups (SIG), with theresultant discussions summarized in 21 articles published in

consecutive issues of The Journal. The meeting also intro-duced 2 changes within the structure of OMERACT. Prior tothis meeting, the executive leadership expanded itsmembership from 5 to 14; and OMERACT work wasdivided into 4 streams: patients, methods, diseases, andimaging/biomarkers. This format allowed for more effectivementoring, monitoring, and management of working groupswithin each stream. Five separate sessions were dedicated to reevaluating the

current OMERACT filter of truth, discrimination, and feasi-bility, evaluating the need for revision or expansion, andproviding a better framework for groups interested in devel-oping core sets and improving outcome measures inrandomized controlled trials (RCT) and other research settings.An important first step for RCT is to define a core set ofdomains that reflect clinically relevant outcomes for inclusionwithin such trials. Validated instruments are then needed toassess each domain, and a core set of outcome measures mustthen be consistently measured in RCT within the designateddisease state. The original OMERACT filter (Filter 1.0) statesthat any outcome measure should meet criteria for beingtruthful, be discriminative between groups and responsive tothe intended intervention, and be feasible to perform. Given that initial development of the filter took place

more than 20 years ago, it was recognized that some aspectsrequired updating. To address those issues, about one-half ofthe OMERACT 11 meeting was dedicated to daily discus-sions regarding the filter in the context of updated generaldomains of health status. The original OMERACT filter waspredicated on considering a simpler domain construct of the4 “Ds”: discomfort, disability, dollar cost, and death. It wasrecognized that broadening these original 4 Ds into a moreglobal conceptual framework for outcome measurementacross health conditions was needed (Filter 2.0), offering anopportunity to define a more explicit process to developcore measurement sets. The first step was to modify theoriginal 4 D framework to comprise 3 core “areas:” death,life impact, and pathophysiologic manifestations. Followingfurther discussion, it was recognized that adding resourceuse as a domain of measurement was desirable. Individual sessions regarding Filter 2.0 included Session

1: Truth (1) Domains and How to Define Them; Session 2:Truth (2) Instruments and How to Validate Them; Session 3:Discrimination, Including Responsiveness & Feasibility;Session 4: Putting It All Together; The Example of PatientReported Outcomes; and Session 5: Putting It All Together;The Example of Imaging and Biomarkers.



The entire meeting was integrated around the Filter 2.0sessions. Specific disease or topic groups were invited toparticipate and present “case studies” for each of the Filter2.0 discussions. Thus, practical issues (pros and cons)related to utilization, and examples of implementation,informed further evolution of the filter. As with other OMERACT meetings, sessions were struc-

tured as (1) SIG to present data and obtain feedback forongoing work streams and to aid in evaluation of theirresearch agendas; (2) workshops, in which a group provideddata and subsequent breakout groups for topic refinement ormore intensive discussion followed by a plenary voteseeking endorsement; and (3) modules, with longerbreakout sessions during which groups sought endorsementfor finalization of a core set of outcome measures or aresponder index. SIG presented at this year’s meetingincluded connective tissue disease–interstitial lung disease;equity; hand osteoarthritis; Patient Reported OutcomesMedical Information System (PROMIS); Rasch analysis;magnetic resonance imaging (MRI) in hip osteoarthritis;MRI in inflammatory arthritis; MRI in juvenile idiopathicarthritis; myositis; and polymyalgia rheumatica. Workshopsincluded acute gout; vasculitis; flare in rheumatoid arthritis;ultrasound: responsiveness in rheumatoid arthritis; andworker productivity; and there was a single module onpsoriatic arthritis. A special meeting was held immediately preceding the

larger OMERACT meeting to discuss different approachesto data analysis, specifically addressing similarities anddifferences between item response theory (IRT) and Raschapproaches — both potentially applicable to a number ofworking groups in their development of outcome measures.Examples were presented of the IRT-developed PROMIS

developed by the US National Institutes of Health, and fordevelopment of measures using the Rasch approach.Discussions of these topics were followed up in individualRasch and PROMIS SIG held within the formal meeting. The biennial meeting continues to be the highlight of the

OMERACT process, which now covers an increasinglydiverse range of rheumatic conditions. This meetingexperience brings together diverse stakeholders, includingpatients, to ensure that decisions and discussions arerelevant and applicable. The updating of the OMERACTFilter 2.0 to be more broadly applicable was a major focusof this meeting to provide a more explicit framework for theOMERACT process. A formative research agenda wasdeveloped for a number of groups that will provide consid-erable opportunities for ongoing collaborative researchleading to the next meeting. Without a doubt, the products ofOMERACT 11 will engender further debate and discussionthat will be continued at the OMERACT meeting to be heldin Budapest, Hungary, in 2014.

LEE S. SIMON, MD, West Newton,Massachusetts, USA;VIBEKE STRAND, MD,Stanford University,Portola Valley, California, USA;CLIFTON O. BINGHAM III, MD,Johns Hopkins University, Baltimore, Maryland, USA;JASVINDER A. SINGH, MBBS, MPH,Birmingham VA Medical Center and Division of Rheumatology,Department of Medicine and Division of Epidemiology, University of Birmingham, Birmingham, Alabama, USA.

Address correspondence to Dr. Simon; E-mail: [email protected]

148 The Journal of Rheumatology 2014; 41:1; doi:10.3899/jrheum.130937


ORGANIZING COMMITTEELee S. Simon, USAVibeke Strand, USAClifton O. Bingham III, USAJasvinder A. Singh, USA

THE OMERACT EXECUTIVEMaarten Boers, NetherlandsPeter M. Brooks, AustraliaPhilip G. Conaghan, UK Maria-Antonietta D’Agostino, FranceLaure Gossec, FranceJohn R. Kirwan, UKRobert Landewé, NetherlandsLyn March, AustraliaPeter Tugwell, CanadaGeorge A. Wells, Canada

STEERING GROUPDorcas Beaton Arvind ChopraMaxime Dougados Paul EmeryDan Furst Sherine GabrielDuncan Gordon Walter MaksymowychPhilip Mease Girish ModyTed Pincus Pam RichardsKen Saag Eduardo SamoyoaRalph Schumacher Josef SmolenRandall Stevens Desiree van der HeijdeJanet Woodcock



ACKNOWLEDGMENTFinancial support for OMERACT 11 was provided by thefollowing pharmaceutical companies and their subsidiaries: Amgen, USA; Astra Zeneca, UK; Bioberica, Spain; BristolMyers Squibb, USA; Celgene, USA; Eli Lilly, USA; Forest,USA; Genentech/Roche, USA; Genzyme, USA; HorizonPharma Inc., USA; Novartis, Switzerland; Pfizer, USA;Quintiles, Netherlands; Regeneron, USA; Savient, USA;Takeda, USA; UCB, USA.

The Organizing Committees thank the European LeagueAgainst Rheumatism and the American College ofRheumatology for bursaries provided to young researchers

to participate in the OMERACT program. The OMERACTconferences are possible only through the ongoingcommitment of the (co-)chairs and their subgroups of themodules, workshops, and special interest groups, whoprovide the science. A special thank you goes to ICG(Innovations Consulting Group) for organizing the logisticsof this conference. A very special thank you goes to theOMERACT Secretariat, and especially to its organizationalhead, Shawna Grosskleg, who has provided terrific supportthroughout the years.

J Rheumatol 2014;41:145–9; doi:10.3899/jrheum.130937



Papers presented at the OMERACT 11 Conference, Pinehurst, North Carolina, USA, May 12-17, 2012:

Part 1 Methods

Part 2 Imaging and Other Biomarkers

Part 3 Disease-specific Outcomes I

Part 4 Disease-specific Outcomes II

Part 5 The OMERACT Filter 2.0

Part 2 will appear in the February issue.



the journal of rheumatology volume 41, no. 1 omeract 11 ... · the journal of rheumatology volume...

Documents