Article Text
Abstract
The Advances in Targeted Therapies meets annually, convening experts in the field of rheumatology to both provide scientific updates and identify existing scientific gaps within the field. To review the major unmet scientific needs in rheumatology. The 23rd annual Advances in Targeted Therapies meeting convened with more than 100 international basic scientists and clinical researchers in rheumatology, immunology, infectious diseases, epidemiology, molecular biology and other specialties relating to all aspects of immune-mediated inflammatory diseases. We held breakout sessions in five rheumatological disease-specific groups including: rheumatoid arthritis (RA), psoriatic arthritis (PsA), axial spondyloarthritis (axSpa), systemic lupus erythematosus (SLE), systemic sclerosis (SSc) and vasculitis, and osteoarthritis (OA). In each group, experts were asked to identify and prioritise current unmet needs in clinical and translational research. An overarching theme across all disease states is the continued need for clinical trial design innovation with regard to therapeutics, endpoint and disease endotypes. Within RA, unmet needs comprise molecular classification of disease pathogenesis and activity, pre-/early RA strategies, more refined pain profiling and innovative trials designs to deliver on precision medicine. Continued scientific questions within PsA include evaluating the genetic, immunophenotypic, clinical signatures that predict development of PsA in patients with psoriasis, and the evaluation of combination therapies for difficult-to-treat disease. For axSpA, there continues to be the need to understand the role of interleukin-23 (IL-23) in pathogenesis and the genetic relationship of the IL-23-receptor polymorphism with other related systemic inflammatory diseases (eg, inflammatory bowel disease). A major unmet need in the OA field remains the need to develop the ability to reliably phenotype and stratify patients for inclusion in clinical trials. SLE experts identified a number of unmet needs within clinical trial design including the need for allowing endpoints that reflect pharmacodynamic/functional outcomes (eg, inhibition of type I interferon pathway activation; changes in urine biomarkers). Lastly, within SSc and vasculitis, there is a lack of biomarkers that predict response or disease progression, and that allow patients to be stratified for therapies. There remains a strong need to innovate clinical trial design, to identify systemic and tissue-level biomarkers that predict progression or response to therapy, endotype disease, and to continue developing therapies and therapeutic strategies for those with treatment-refractory disease. This document, based on expert consensus, should provide a roadmap for prioritising scientific endeavour in the field of rheumatology.
- Rheumatoid Arthritis
- Psoriatic Arthritis
- Spondyloarthritis
- Systemic Lupus Erythematosus
- Systemic Sclerosis
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Footnotes
Handling editor David S Pisetsky
Twitter @ProfJohnIsaacs, @CarolineOspelt, @StefanSiebert1
Contributors All authors contributed to the study design, data collection, discussion and critical revision of the manuscript.
Funding The Advances in Targeted Therapies meeting was supported by: AbbVie, AnaptysBio, BMS, Chugai, Eli Lilly, Galapagos, GSK, Janssen, Mitsubishi, Novartis, and Pfizer.
Competing interests KLW has received research grants and/or consulting honoraria from Janssen Pfizer, AbbVie, Union Chimique Belge (UCB), Eli Lilly & Company, Galapagos, GlaxoSmithKline (GSK), Roche, Gilead, BMS, Regeneron, Sanofi, AstraZeneca, Novartis; JDI has received grants and/or consulting honoraria from Janssen, GSK, Pfizer, Abbvie, BMS, Gilead, Roche, Eli Lilly, Anaptys Bio, Sonoma; PM has received grants and/or consulting honoraria from AbbVie, Acelyrin, Aclaris, Amgen, Boehringer Ingelheim, BMS, Eli Lilly, Galapagos, Gilead, GlaxoSmithKline, Inmagene, Janssen, Moonlake, Novartis, Pfizer, Sun Pharma, Takeda, UCB, Ventyx; JEG has received grants and/or consulting honoraria from Abbvie, BMS, Gilead, Galapagos, Janssen, Lilly, MSD, Novartis, Pfizer, Roche Chugai, Sanofi; MHB has received grants and/or consulting honoraria (all paid to host institution) from Abbvie, Boehringer-Ingelheim, Galapagos, Gilead and Pfizer Ltd. MKC has received grants or consulting honoraria from AMPEL BioSolutions, Astra Zeneca, BMS, Gilead Sciences, and GSK; JK has received research grants (paid to UMass Chan Medical School) from Aker BioMarine AS; Galapagos NV; and Novartis Pharmaceuticals Corp.; and consulting honoraria from Alvotech Swiss AG; Boehringer Ingelheim GmbH; Bristol Myers Squibb Co.; Fresenius Kabi; Inmagene LLC; Kolon TissueGene, Inc.; Novartis Pharmaceuticals Corp.; Organon LLC; Pfizer Inc.; Samsung Bioepis; Sandoz Inc.; Scipher Medicine; Teijin Pharma Ltd.; Viatris Inc.; and UCB Inc; LC serves on a Data Safety Monitoring Board for Kezar Life Sciences and has research grants from Argenyx; AK has received grants and/or consulting honoraria from Amgen, Eli Lilly, Galapagos, Gilead, Janssen, Novartis, UCB and Pfizer; XB has received grants and/or consulting honoraria from AbbVie, BMS, MSD, Celgene, Chugai, Merck, Novartis, Pfizer, Sandoz, and UCB; RFvV has received grants and/or consulting honoraria; CO has no competing interests; HPK has received grants and/or consulting honoraria from SS has received grants and/or consulting honoraria from Amgen, AbbVie, AstraZeneca, Boehringer-Ingelheim, BMS, Eli Lilly, GSK, Janssen and UCB; MK has received grants and/or consulting honoraria (all paid to institution) from UCB, Pfizer, Novartis, Galapagos, CHDR and Jansen; DA has received grants and/or consulting honoraria from Abbvie, Amgen, Galapagos, Lilly, Janssen, Merck, Novartis, Pfizer, Sandoz, and Sanofi; IBM has received grants and/or consulting honoraria from AbbVie, Amgen, BMS, Causeway Therapeutics, Cabaletta, Eli Lilly, Gilead, Janssen, Novartis, Pfizer, Sanofi, UCB, Evelo, Compugen, AstraZeneca, Moonlake, and serves as a NHS GGC board member, Evelo Board of Directors, Versus Arthritis Trustee Status; TWJH has no competing interests to disclose; REV has received grants and/or consulting honoraria from AbbVie, Amgen, Boehringer Ingelheim, BMS, Janssen-Cilag, GSK, Hexal, Neutrolis, Novartis, and Pfizer; EMG: serves as an associate editor at the New England Journal of Medicine and receives royalties from Elsevier; FCB has no competing interests to disclose; JSS has received research grants and/or consulting honoraria from AbbVie, Amgen, AstraZeneca, Astro, Bristol-Myers Squibb, Chugai, Janssen, Lilly, Merck Sharp & Dohme, Novartis-Sandoz, Pfizer, R-Pharma, Roche, Samsung, and UCB serves as the editor of Annals of Rheumatic Diseases but was not involved in the handling or review of this manuscript.
Patient and public involvement Patients and/or the public were not involved in the design, or conduct, or reporting, or dissemination plans of this research.
Provenance and peer review Not commissioned; externally peer reviewed.