Abstract
Objectives To detail the greatest areas of unmet scientific and clinical needs in rheumatology. Methods The 21st annual international Advances in Targeted Therapies meeting brought together more than 100 leading basic scientists and clinical researchers in rheumatology, immunology, epidemiology, molecular biology and other specialties. During the meeting, breakout sessions were convened, consisting of 5 disease-specific groups with 20-30 experts assigned to each group based on expertise. Specific groups included: rheumatoid arthritis, psoriatic arthritis, axial spondyloarthritis, systemic lupus erythematosus and other systemic autoimmune rheumatic diseases. In each group, experts were asked to identify unmet clinical and translational research needs in general and then to prioritise and detail the most important specific needs within each disease area. Results Overarching themes across all disease states included the need to innovate clinical trial design with emphasis on studying patients with refractory disease, the development of trials that take into account disease endotypes and patients with overlapping inflammatory diseases, the need to better understand the prevalence and incidence of inflammatory diseases in developing regions of the world and ultimately to develop therapies that can cure inflammatory autoimmune diseases. Conclusions Unmet needs for new therapies and trial designs, particularly for those with treatment refractory disease, remain a top priority in rheumatology.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 88-93 |
| Number of pages | 6 |
| Journal | Annals of the rheumatic diseases |
| Volume | 79 |
| Issue number | 1 |
| DOIs | |
| State | Published - Jan 1 2020 |
Keywords
- Sjögren's syndrome
- ankylosing spondylitis
- inflammatory myopathies
- psoriatic arthritis
- rheumatoid arthritis
- spondyloarthritis
- systemic lupus erythematosus
- systemic sclerosis
- vasculitis
ASJC Scopus subject areas
- Immunology and Allergy
- Rheumatology
- Immunology
- Biochemistry, Genetics and Molecular Biology(all)
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Unmet need in rheumatology : Reports from the Targeted Therapies meeting 2019. / Winthrop, Kevin L.; Weinblatt, Michael E.; Bathon, Joan; Burmester, Gerd R.; Mease, Philip J.; Crofford, Leslie; Bykerk, Vivian; Dougados, Maxime; Rosenbaum, James (Jim); Mariette, Xavier; Sieper, Joachim; Melchers, Fritz; Cronstein, Bruce N.; Breedveld, Ferry C.; Kalden, Joachim; Smolen, Josef S.; Furst, Daniel.
In: Annals of the rheumatic diseases, Vol. 79, No. 1, 01.01.2020, p. 88-93.Research output: Contribution to journal › Review article › peer-review
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TY - JOUR
T1 - Unmet need in rheumatology
T2 - Reports from the Targeted Therapies meeting 2019
AU - Winthrop, Kevin L.
AU - Weinblatt, Michael E.
AU - Bathon, Joan
AU - Burmester, Gerd R.
AU - Mease, Philip J.
AU - Crofford, Leslie
AU - Bykerk, Vivian
AU - Dougados, Maxime
AU - Rosenbaum, James (Jim)
AU - Mariette, Xavier
AU - Sieper, Joachim
AU - Melchers, Fritz
AU - Cronstein, Bruce N.
AU - Breedveld, Ferry C.
AU - Kalden, Joachim
AU - Smolen, Josef S.
AU - Furst, Daniel
N1 - Funding Information: The convening of the 21st ATT afforded the possibility to discuss and articulate major unmet needs in the field of rheumatology, and across domains there were several overarching perceived unmet needs ( table 1 ). It was generally understood that there has not been sufficient emphasis on trial designs which concentrated on well-defined disease subtypes. Many diseases have multiple subtypes (eg, axial and peripheral PsA or limited/diffuse systemic sclerosis with multiple serological subtypes) and trial designs which mix those subtypes could obscure the success of treatments in specific subgroups. Likewise, trial designs which are able to dissect (or include) overlapping diseases are also needed. Table 1 Identified unmet research needs of high priority within RA, PSA, AxSpa, SLE and other systemic autoimmune rheumatic diseases Rheumatoid arthritis The need to better define treatment ‘refractory’ states both phenotypically and molecularly The need to focus on refractory patients in both the study of novel targeted therapies and in the study of existing therapies in novel combinations or sequences Psoriatic arthritis Understanding differential therapeutic effects on different clinical domains in PsA such as enthesitis Further evaluation of combination therapies and strategic trials including the use of sequential therapies, controlled withdrawal, the treatment of early disease and the treatment of monoarticular or oligoarticular disease Ankylosing spondyloarthritis Understanding the role of the microbiome in disease pathogenesis and potential therapy Understanding disease pathology specifically with regard to why Il-23 inhibition does not improve the disease. Systemic lupus erythematosus Improving clinical trial design by reducing heterogeneity of participants, developing new outcome disease activity measures, standardising serological testing and conducting organ-specific trials Consider alternative trial designs including adaptive trials and withdrawal trials Other systemic autoimmune rheumatic diseases Improving clinical trial design, specifically with reducing heterogeneity in disease endotypes and the use of organ-specific outcome measures Identification of predictive biomarkers and the inclusion of patient-reported outcomes of specific manifestations (eg, calcinosis) for clinical trials axSpA, axial spondyloarthritis; PsA, psoriatic arthritis; RA, rheumatoid arthritis; SLE, systemic lupus erythematous. While there has been some success in treating moderate to severe patients with various inflammatory rheumatic diseases and even inclusion of some patients with Disease Modifying anti-Rheumatic Drugs (DMARD)-refractory disease in RA, this remains a top unmet need in RA that has been even less carefully examined in patients with other diseases. For example, patients with PsA are often included in trials only if they have been naïve to previous conventional synthetic DMARD (csDMARDs) or biologic DMARD (bDMARDs); more attention needs to be paid to patients who are more ‘difficult-to-treat’ across all conditions, as well as those who have multiple complications or comorbidities or those who have failed other csDMARDs or bDMARDs. Last, while progress has been made in treating patients who used to have unmet need within countries and regions such as Australia, Japan, North America and the European Union, it was highlighted that more emphasis needed to be placed on understanding unmet needs in other countries and continents such as Africa, multiple areas in Asia and Central and South America. Contributors All coauthors contributed to this manuscript’s creation in all aspects including data gathering, analysis, writing and critical revision of the manuscript. Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors. Competing interests KLW reports personal fees from Pfizer, grants and personal fees from BMS, personal fees from AbbVie, personal fees from UCB, personal fees from Lilly, personal fees from Galapagos, personal fees from GSK, personal fees from Roche outside the submitted work. JB reports personal fees from AbbVie outside the submitted work. MD reports grants and personal fees from Pfizer, grants and personal fees from Abbvie, grants and personal fees from Lilly, grants and personal fees from Novartis, grants and personal fees from UCB, grants and personal fees from Merck, grants and personal fees from Roche, during the conduct of the study. JTR reports grants from Pfizer, personal fees from Abbvie, personal fees from Gilead, personal fees from Santen, personal fees from Roche, personal fees from Novartis, personal fees from UCB, personal fees from Corvus, personal fees from Horizon, personal fees from Celldex, personal fees from Eyevensys, personal fees from UpToDate, personal fees from Janssen outside the submitted work. XM reports personal fees from BMS, personal fees from GILEAD, personal fees from PFIZER, personal fees from SAMSUNG, personal fees from UCB outside the submitted work. BNC has multiple patents, none of which are relevant. In addition he has multiple grants from NIH. JSS reports grants and personal fees from AbbVie, personal fees from Amgen, personal fees from AstraZeneca, personal fees from Astro, personal fees from Bristol-Myers Squibb, personal fees from Celgene, personal fees from Celltrion, personal fees from ILTOO, grants and personal fees from Janssen, grants and personal fees from Lilly, personal fees from MSD, grants and personal fees from Novartis-Sandoz, personal fees from Novo-Nordisk, grants and personal fees from Roche, personal fees from Samsung Bioepis, personal fees from Sanofi, personal fees from UCB, grants and personal fees from Pfizer outside the submitted work. DF reports grant/research support from Actelion, grant/research support from Amgen, grant/research support from BMS, grant/research support from Corbus, grant/research support from Galapagos GSK, grant/research support from NIH, grant/research support from Novartis, grant/research support from Pfizer, grant/research support from Sanofi, grant/research support from Roche/Genentech, personal fees from Actelion, personal fees from Amgen, personal fees from BMS, personal fees from Corbus, personal fees from Galapagos, personal fees from Novartis, personal fees from Pfizer outside the submitted work. Patient consent for publication Not required. Provenance and peer review Not commissioned; externally peer reviewed. Data availability statement All data relevant to the study are included in the article or uploaded as supplementary information.
PY - 2020/1/1
Y1 - 2020/1/1
N2 - Objectives To detail the greatest areas of unmet scientific and clinical needs in rheumatology. Methods The 21st annual international Advances in Targeted Therapies meeting brought together more than 100 leading basic scientists and clinical researchers in rheumatology, immunology, epidemiology, molecular biology and other specialties. During the meeting, breakout sessions were convened, consisting of 5 disease-specific groups with 20-30 experts assigned to each group based on expertise. Specific groups included: rheumatoid arthritis, psoriatic arthritis, axial spondyloarthritis, systemic lupus erythematosus and other systemic autoimmune rheumatic diseases. In each group, experts were asked to identify unmet clinical and translational research needs in general and then to prioritise and detail the most important specific needs within each disease area. Results Overarching themes across all disease states included the need to innovate clinical trial design with emphasis on studying patients with refractory disease, the development of trials that take into account disease endotypes and patients with overlapping inflammatory diseases, the need to better understand the prevalence and incidence of inflammatory diseases in developing regions of the world and ultimately to develop therapies that can cure inflammatory autoimmune diseases. Conclusions Unmet needs for new therapies and trial designs, particularly for those with treatment refractory disease, remain a top priority in rheumatology.
AB - Objectives To detail the greatest areas of unmet scientific and clinical needs in rheumatology. Methods The 21st annual international Advances in Targeted Therapies meeting brought together more than 100 leading basic scientists and clinical researchers in rheumatology, immunology, epidemiology, molecular biology and other specialties. During the meeting, breakout sessions were convened, consisting of 5 disease-specific groups with 20-30 experts assigned to each group based on expertise. Specific groups included: rheumatoid arthritis, psoriatic arthritis, axial spondyloarthritis, systemic lupus erythematosus and other systemic autoimmune rheumatic diseases. In each group, experts were asked to identify unmet clinical and translational research needs in general and then to prioritise and detail the most important specific needs within each disease area. Results Overarching themes across all disease states included the need to innovate clinical trial design with emphasis on studying patients with refractory disease, the development of trials that take into account disease endotypes and patients with overlapping inflammatory diseases, the need to better understand the prevalence and incidence of inflammatory diseases in developing regions of the world and ultimately to develop therapies that can cure inflammatory autoimmune diseases. Conclusions Unmet needs for new therapies and trial designs, particularly for those with treatment refractory disease, remain a top priority in rheumatology.
KW - Sjögren's syndrome
KW - ankylosing spondylitis
KW - inflammatory myopathies
KW - psoriatic arthritis
KW - rheumatoid arthritis
KW - spondyloarthritis
KW - systemic lupus erythematosus
KW - systemic sclerosis
KW - vasculitis
UR - http://www.scopus.com/inward/record.url?scp=85076875005&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85076875005&partnerID=8YFLogxK
U2 - 10.1136/annrheumdis-2019-216151
DO - 10.1136/annrheumdis-2019-216151
M3 - Review article
C2 - 31662322
AN - SCOPUS:85076875005
VL - 79
SP - 88
EP - 93
JO - Annals of the Rheumatic Diseases
JF - Annals of the Rheumatic Diseases
SN - 0003-4967
IS - 1
ER -