The Childhood Arthritis and Rheumatology Research Alliance (CARRA), the largest North American research collaboration among pediatric rheumatologists, recently reviewed their use of consensus treatment plans to foster an environment that emphasizes evaluation of the comparative effectiveness of therapeutic regimens. Consensus treatment plans have been detailed and implemented for 7 rheumatologic disease states — juvenile localized scleroderma, juvenile lupus nephritis, systemic juvenile idiopathic arthritis (JIA), polyarticular JIA, and 3 categories of juvenile dermatomyositis — and have been successful at identifying effective treatment options and improving clinical outcomes, according to the review published in Arthritis and Rheumatology.
Although randomized controlled trials are generally preferred for evidentiary guidance, they also have certain limitations — including restricted eligibility, excessive cost, and large sample size requirements — that make them less suited to provide answers to some of the most urgent problems in pediatric rheumatology. Consensus treatment plans offer a complementary avenue with wider patient contact and more generalizability than randomized controlled trials.
The authors describe consensus treatment plans as treatment strategies that aim to “reduce variation in treatment approaches, standardize outcome measurements, and allow for comparison of the effectiveness of different approaches, with the goal of improving disease outcomes.” The consensus treatment plans are based on a combination of available literature and expert consensus and should be considered research tools rather than actual treatment guidelines. The overall approach is modeled after the work of other pediatric networks such as the Children’s Oncology Group, which has enhanced patient outcomes and improved lives.
A total of 5 pilot programs have been completed thus far, deploying consensus treatment plans for juvenile dermatomyositis, juvenile localized scleroderma, juvenile lupus nephritis, systemic JIA, and polyarticular JIA. In addition, there are 2 ongoing pilot studies currently examining JIA: Start Time Optimization in Polyarticular JIA (STOP-JIA), which compares 3 treatment strategies for the initial treatment of polyarticular JIA to help patients achieve inactive disease, and First Line Options for Systemic JIA Treatment (FROST), which compares the effectiveness of biologic vs non-biologic therapies in achieving clinically inactive disease.
An essential aspect of CARRA’s efforts to standardize care through consensus treatment plans is the use of stakeholder involvement, whereby input is solicited from patients, parents, and healthcare providers in an attempt to promote an iterative process that continuously replaces older, less effective therapies with those demonstrating better results. Healthcare providers are then asked to choose from a few agreed on treatment options that cover the majority of clinical presentations, in accordance with usual practices and thoughts from other stakeholders.
The goal of consensus treatment plans and comparative effectiveness research is to drive a cycle of innovation and replacement that allows the addition of new treatments and the removal of unused or ineffective strategies in an effort to fine-tune the treatment of rare diseases. As more evidence is published and expert consensus evolves, consensus treatment plans are amended to reflect the standard of care. Although the immediate objective of CARRA is to improve the lives of children with rheumatic diseases, the future success with consensus treatment plan studies in pediatric rheumatology could be applied to other rare disorders.
Ringold S, Nigrovic PA, Feldman BM, et al. The Childhood Arthritis & Rheumatology Research Alliance consensus treatment plans: towards comparative effectiveness in the pediatric rheumatic diseases [published online January 15, 2018]. Arthritis Rheumatol. doi:10.1002/art.40395