Innovative clinical trial designs, therapies for refractory disease top unmet needs in rheumatology
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New treatments and trial designs, particularly focused on patients with refractory disease, remain among the top unmet needs in rheumatology in 2019, according to expert opinions culled from the Advances in Target Therapies annual meeting and published in the Annals of the Rheumatic Diseases.
“The Advances in Targeted Therapies meeting (ATT) has met annually for 21 years, bringing together clinical scientists and immunology and molecular biology experts from around the world,” Kevin L. Winthrop, MD, MPH, of the Oregon Health Sciences University, and colleagues wrote. “The meeting’s objective is to update participants regarding the latest insights regarding disease mechanism(s) and pathophysiology and recent developments with both existing and novel targeted therapies in the field of [immune-related inflammatory diseases] with a focus on rheumatological diseases.”
“Previously, a consensus document describing the recommended use of targeted therapies within rheumatology was produced from this meeting,” they added. “However, with the expanse of targeted therapies and the recent clinical recommendations published from both American College of Rheumatology and the European Union League Against Rheumatism, a document covering all targeted therapies across all disease indications became too complex and voluminous as a single manuscript. Accordingly, the annual meeting’s output was modified to discuss key unmet needs within the field, consistent with the meeting’s underlying objective of promoting innovation and collaboration.”
To describe the greatest areas of unmet need in rheumatology, Winthrop and colleagues organized breakout sessions during the 21st annual international Advances in Targeted Therapies meeting, which attracted more than 100 scientists and clinical researchers specializing in rheumatology, immunology, epidemiology, molecular biology and other specialties.
Within the breakout sessions were five disease-specific groups — covering rheumatoid arthritis, psoriatic arthritis, axial spondyloarthritis, systemic lupus erythematosus and “other systemic autoimmune rheumatic diseases” — each made up of 20 to 30 members who were assigned there based on expertise. During these sessions, group members were asked to identify important unmet clinical and translational research needs, both in general and within their specific disease area.
According to the authors, the experts developed a series of overarching themes common across all disease states, including the need to innovate clinical trial designs with an emphasis on patients with refractory disease. The experts also stressed the need to develop trials that acknowledge disease endotypes and patients with overlapping inflammatory diseases, as well as well as the need to improve understanding of the prevalence and incidence of such conditions in developing regions of the world. Ultimately, Winthrop and colleagues wrote, researchers must develop therapies that can cure inflammatory autoimmune diseases.
“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,” Winthrop and colleagues wrote. “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 [conventional synthetic] DMARDs or [biologic] DMARDs.”
The researchers added that, “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.” – by Jason Laday
Disclosure: Winthrop reports personal fees AbbVie, Bristol-Myers Squibb, UCB, Eli Lilly, Galapagos, GlaxoSmithKline Pfizer, Roche and UCB, as well as grants from Bristol-Myers Squibb. Please see the study for all other authors’ relevant financial disclosures.
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