POS1060 GAPS IN TRAINING AND EXPERTISE IN MYOSITIS OUTCOME MEASURES: INSIGHTS FROM THE 2024 MCTC INVESTIGATORS' SURVEY

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Background: The Myositis Clinical Trials Consortium (MCTC) is an international collaboration of clinical investigators, research professionals, medical centres, patient partners, and industry stakeholders allied in advancing clinical trials for patients with idiopathic inflammatory myopathies (IIM). Standardised outcome measures are essential in assessing therapeutic responses in clinical trials, ensuring consistency, comparability, and reliability across studies, and ultimately improving patient care. However, the level of clinical experience and expertise in IIM-specific outcome measures among myositis investigators remains unclear. Objectives: To identify gaps in training and expertise with standardized myositis outcome measures among investigators. Methods: The online 2024 Myositis Investigator Survey was broadly distributed worldwide to IIM-focused physicians joining the MCTC via professional societies, peer networks, and MCTC membership rolls. The survey collected information on the investigators' affiliations, subspecialties, medical training, and self-reported data on clinical and research experience in myositis, including training and experience with various commonly used standardised myositis outcome measures. Respondents rated their expertise with outcome measures using a 5-point Likert scale, ranging from "no experience" to "certified," the latter indicating successful completion of a formal training programs for that specific assessment tool. Results: A total of 382 investigators participated in the survey, representing diversity in geographic location, education and subspecialty. Half (50.0%) were affiliated with academic centres, followed by community hospitals (45.3%). Most held MD degrees (91.4%) and were specialised in Rheumatology (79.6%), with smaller groups in Pulmonology (6.5%), Neurology (6.0%), and other specialities (7.9%). Most (81.4%) treated adult IIM patients, 8.1% treated paediatric patients, and 7.3% managed both. While 88.5% of investigators were board-certified or board-eligible, only 20.9% had completed myositis-specific training programs. Investigators reported an extended clinical research experience, with a median (IQR) of 10 (5-20) years in general clinical research and 9 (5-15) years in IIM-specific research. The number of myositis research studies or clinical trials they conducted or participated in within the last five years averaged 3 (1-6) for clinical research studies and 1 (0-3) for interventional/drug trials (Table 1). We identified notable gaps between the investigators' stated clinical experience versus their self-reported proficiency in myositis outcome measures. For Manual Muscle Testing (MMT-8), one of the myositis core set measures, only 16.8% were certified, while 42.1% had limited or no experience. Similarly, for Total Improvement Score defined in the 2016 EULAR/ACR Myositis Response Criteria and used as the primary endpoint in most myositis clinical trials, 21.5% reported limited or no experience, and only 3.1% were certified. Skin-specific measures, including Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) and Cutaneous Dermatomyositis Investigator Global Assessment (CDM-IGA) also showed higher proportions of limited or no experience in 51.0% and 58.4%, respectively. Clinical trial experience with functional assessments such as Timed Up-and-Go (TUG) and 30-Second Sit-to-Stand (30s STS) was limited to only 6.0% and 6.3%, respectively (Figure 1a). Among physicians managing paediatric patients (n=59), juvenile myositis outcome measures such as Childhood Myositis Assessment Scale (CMAS) and Juvenile Dermatomyositis Activity Index (JDMAI) had limited or no experience rates of 44.0% and 55.9%, respectively (Figure 1b). Conclusion: The MCTC Myositis Investigator Information Survey underscores notable gaps in training and experience with standardized myositis outcome measures among investigators. Despite extensive clinical and research experience, investigators' proficiency in multiple myositis outcome measures in clinical trials and formal certification rates were substantially limited, highlighting a critical need for structured training programs to support clinical trial readiness. Addressing these knowledge gaps through targeted training and certification initiatives is essential to enhance the quality of myositis clinical trials and ensure consistent, reliable assessments across studies. Abbreviations: MMT - Manual Muscle Testing, ExGLB - Extramuscular Global disease activity, TIS - Total Improvement Score, CDASI - Cutaneous Dermatomyositis Disease Area and Severity Index, CDM-IGA - Cutaneous Dermatomyositis Investigator Global Assessment, MDAAT - Myositis Disease Activity Assessment Tool, MDI - Myositis Damage Index, IBM-FRS - Inclusion Body Myositis Functional Rating Scale, TUG - Timed Up-and-Go, STS - 30-Second Sit-to-Stand, CMAS - Childhood Myositis Assessment Scale, JDMAI - Juvenile Dermatomyositis Activity Index, DAS JDM – Disease Activity Score Juvenile Dermatomyositis. REFERENCES: NIL . Acknowledgements: 1. The authors wish to thank the MCTC steering committee members and all MCTC investigators for their contribution and participation in the Myositis Investigator Member Information Survey. 2. NIAMS IRP (HK). Disclosure of Interests: Shiri Keret: None declared, Iris Yan Ki Tang: None declared, Hanna Kim Expert panel for Cabaletta Panel, previous CRADA with Eli Lilly and Company providing study support and drug (baricitinib), Bristol Myers Squibb – providing drug for research study, Akira Yoshida: None declared, Eduardo Dourado: None declared, Edoardo Conticini Received speaking honoraria from GSK, Eli Lilly, Chiesi and Dr Schar, Océane Landon-Cardinal: None declared, Kaveh Ardalan Dr. Ardalan has received consulting fees and served on a scientific advisory board for Cabaletta Bio, and is Vice Chair of the Childhood Arthritis and Rheumatology Research Alliance Juvenile Dermatomyositis Committee (paid consultant position), Anuradha Bishnoi: None declared, Faye Pais: None declared, Sabeena Y Usman: None declared, Solciris Dominguez: None declared, Gayathri Tulluru: None declared, Chengappa Kavadichanda: None declared, Daphne Rivero Gallegos: None declared, Francisca Bozan: None declared, James B. Lilleker: None declared, Kaushik Sreerama Reddy: None declared, Rachid Smaili: None declared, Thomas Khoo: None declared, Ting-Yuan Lan: None declared, Valérie Leclair: None declared, Silvia Rosina: None declared, Marc Jansen: None declared, Belina Yi: None declared, Chester V Oddis: None declared, Jiří Vencovský: None declared, Masataka Kuwana Speaking honoraria from Boehringer-Ingelheim, Consulting honoraria from Argenx, Boehringer-Ingelheim, and GSK, Research grants from Boehringer-Ingelheim and MBL, Lisa Rider Hope Pharmaceuticals, Prateek C. Gandiga Received consulting honoraria from Kezar Life Sciences and Janssen Pharmaceuticals, Rohit Aggarwal Consulting fees from Alexion, ANI Pharmaceutical, Argenx, Artasome, AstraZeneca, Boehringer Ingelheim, Bristol Myers-Squibb, CabalettaBio, Capella, Capstan, Corbus, CSL Behring, EMD Serono, Galapagos, Horizontal Therapeutics, I-Cell, Immunovant, Janssen, Kezar, Kyverna, Lilly, Manta Medicines Corporation, Merck, Novartis, Nuvig Therapeutic, Octapharma, Pfizer, Roivant, Sanofi, Teva, Tourmaline Bio, and Verismo Therapeutics, Received research grants from Boehringer Ingelheim, Bristol-Myers Squibb, EMD Serono, Janssen, Mallinckrodt, Pfizer, and Q32. © The Authors 2025. This abstract is an open access article published in Annals of Rheumatic Diseases under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/). Neither EULAR nor the publisher make any representation as to the accuracy of the content. The authors are solely responsible for the content in their abstract including accuracy of the facts, statements, results, conclusion, citing resources etc.

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