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LO-033 Towards a novel clinical outcome assessment for systemic lupus erythematosus trials – first outcomes of an international consensus process
  1. Kathryn Connelly1,
  2. Laura Eades1,
  3. Rachel Koelmeyer1,
  4. Darshini Ayton2,
  5. Vera Golder1,
  6. Rangi Kandane-Rathnayake1,
  7. Kate Gregory-Wong1,
  8. Hermine Brunner3,
  9. Laurie Burke4,
  10. Laurent Arnaud5,
  11. Anca Askanase6,
  12. Cynthia Aranow7,
  13. Edward Vital8,
  14. Guillermo Pons-Estel9,
  15. Khadija Dantata10,
  16. Ying Sun11,
  17. Yoshiya Tanaka12,
  18. Simon Lee13,
  19. Youmna Lahoud14,
  20. Alan Friedman15,
  21. Kenneth Kalunian16,
  22. Qing Zuraw17,
  23. Victoria Werth18,
  24. Sandra Garces19 and
  25. Eric Morand1
  1. 1School of Clinical Sciences at Monash Health, Monash University, Australia
  2. 2School of Public Health and Preventive Medicine, Monash University, Australia
  3. 3Division of Rheumatology, Cincinnati Children’s Hospital, University of Cincinnati, USA
  4. 4University of Maryland School of Pharmacy, LORA Group, Royal Oak Michigan, USA
  5. 5Department of Rheumatology, National Reference Center for Autoimmune Diseases (RESO), Hopitaux Universitaires de Strasbourg, France
  6. 6Lupus Center, Columbia University Medical Center, USA
  7. 7Lupus Centre of Excellence, Feinstein Institutes for Medical Research, Northwell Health, USA
  8. 8Leeds Institute of Rheumatic and Musculoskeletal Medicine, University of Leeds and NIHR Leeds Biomedical Research Centre, Leeds Teaching Hospitals NHS Trust, UK
  9. 9Departamento de Medicina Interna, Centro Regional de Enfermedades Autoinmunes y Reumáticas de Grupo Oroño, Argentina
  10. 10Health Education, Lupus Foundation of America, USA
  11. 11Global Evidence and Value Development, Merck Healthcare KGaA, Germany
  12. 12The First Department of Internal Medicine, University of Occupational and Environmental Health, Japan
  13. 13SDG LLC, SDG LLC Cambridge, USA
  14. 14Clinical Development Immunology, Biogen Inc, USA
  15. 15Immunology Clinical Development, AbbVie Inc., USA
  16. 16Division of Rheumatology, Allergy and Immunology,, University of California San Diego, USA
  17. 17Clinical Development, Janssen Research and Development, USA
  18. 18Department of Dermatology, Hospital of the University of Pennsylvania, USA
  19. 19Global Clinical Development – Inflammation, Amgen, Inc. Thousand Oaks, USA


Background Measurement of treatment effects in systemic lupus erythematosus (SLE) randomised controlled trials (RCTs) remains challenging. Current RCT endpoints are not based on contemporary outcome assessment methodology. We report the current status of a global academic-industry-patient partnership aiming to develop a novel, patient-centred clinical outcome assessment (COA) for SLE RCTs: Treatment Response Measure for SLE (TRM-SLE).

Methods We convened a global Taskforce of clinical-academic, metrology and regulatory experts, patient representatives and experts from ten pharmaceutical companies. A study protocol outlining a structured process for COA development was drafted and refined by TRM-SLE Taskforce members (figure 1), with reference to updated regulatory guidance. We next defined the high-level measurement goals for TRM-SLE using formal consensus methods, first by generating a conceptual definition using the PICO-C framework then establishing the context of use. Taskforce members participated in moderated discussion and real-time voting via virtual platforms with a pre-defined consensus threshold of 70% agreement.

Results A panel of 45 Taskforce members representing key stakeholder groups formulated the conceptual definition and context of use for TRM-SLE. Moderated discussion and multiple rounds of voting resulted in high agreement (81–100%, table 1). The current project phase (Aim 1.3) will establish consensus on the domain-level concepts to be measured by TRM-SLE. To date, TRM-SLE Taskforce members, including patients, have nominated 64 concepts, which have been grouped into a core list of 34 candidate domains.

Conclusions A global academic-industry-patient partnership has completed the first steps towards developing a novel SLE COA that is a quantitative measure of treatment response. Subsequent steps will combine consensus techniques with evaluation of supporting evidence, to determine the final set of concepts and associated measurements to be included in TRM-SLE. These measures will be integrated as a multi-domain COA in an SLE RCT endpoint that will be validated in clinical trials.

Abstract LO-033 Figure 1

Steps in the development of a novel COA for SLE clinical trials

Abstract LO-033 Table 1

High level measurement goals for the TRM-SLE COA

  • Systemic lupus erythematosus
  • outcome measurement

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