CreakyJoints®, the online, non-profit, arthritis patient support community with more than 80,000 members, today announced the publication of “When patients write the guidelines: Patient panel recommendations for the treatment of rheumatoid arthritis,” in Arthritis Care & Research (AC&R), an official journal of the American College of Rheumatology (ACR).
Summarizing an innovative pilot program to evaluate more integral participation of patients in the development of clinical practice guidelines (CPGs), the article was published simultaneously with the new 2015 ACR Guideline for the Treatment of Rheumatoid Arthritis because the evidence report and drafted recommendation statements used in the pilot program were developed first for use in the RA guideline project.
A collaborative effort with lead author Liana Fraenkel, MD, of the Yale University School of Medicine, co-authors of the pilot project paper include W. Benjamin Nowell, PhD, Director of Patient-Centered Research at CreakyJoints, and several Arthritis Power Patient Governors who participated in the project. Arthritis Power is the first ever patient-led, patient-generated, patient-centered research registry for arthritis.
“Once published, the ACR’s clinical practice guidelines define standards of rheumatology practice for at least three years, and while most recommendations are grounded in published evidence, some are primarily based on professional judgment due to a lack of high quality evidence,” said Dr. Nowell.
“In general, when developing guidelines, it’s vital to incorporate the patient perspective as much as possible, because the priorities and risk/benefit valuations of patients may differ from those of health care professionals.”
Although many guideline developers, including the ACR, already include patients in their guideline development work, only 1-2 patients or patient advocates are usually involved in the clinician-dominated voting panels that determine final guideline recommendations.
This pilot study demonstrated the feasibility of more integral patient involvement by developing CPG recommendations based on a voting panel composed entirely of patients. Notably, the project showed that patients developed nearly the same recommendations as physician-dominated panels for questions where there was evidence warranting moderate to high confidence.
However, in some cases patients and physicians differed in the weight they gave to the impact of side effects when evaluating treatment scenarios.
“The insights gained help us to better understand rheumatology patients’ viewpoints and priorities, especially as they relate to guideline development,” said Amy S. Miller, Senior Director of Quality at the American College of Rheumatology, who oversees ACR guideline efforts. “We were glad to have the opportunity to partner in this successful pilot program.”
Patients Trained to Evaluate Evidence
The pilot study included 10 rheumatoid arthritis patients, identified primarily from the Patient Governor group of the Arthritis Patient Partnership With Comparative Effectiveness Researchers (AR-PoWER) Patient Powered Research Network (PPRN), now referred to as Arthritis Power.
The panel included three men (all Caucasian) and seven women (five Caucasian, one African American, one biracial) with a mean (SD) age of 38.1 years (range 29-56 years). All were college educated and six were currently employed.
The average duration since diagnosis was 10.5 years; four were on a traditional disease-modifying antirheumatic drug (DMARD) only, and six were on a biologic with or without a traditional DMARD.
Prior to arriving at the in-person meeting to evaluate and vote on sample CPGs, the panel completed an 8-hour Collaborative Institutional Training Initiative (CITI) Human Subjects Protection training and the Cochrane Collaboration’s Understanding Evidence-Based Healthcare and Serving on a Clinical Practice Guideline modules in order to implement the Grading of Recommendations, Assessment, Development and Evaluation (GRADE) method to develop CPGs, which is also used by ACR guideline voting panels.
The project was facilitated by the same two ACR staff people who facilitated the ACR physician-dominated CPG development project (which took place two weeks after the patient in-person meeting), as well as a rheumatologist.
“We designed the study to determine the feasibility of developing guidelines using voting panels composed entirely of patients. We were also interested in examining whether patients and physicians develop similar recommendations when presented with the same evidence,” said Dr. Fraenkel.
“It was interesting to note, that while patients and physicians reached the same conclusions for the majority of clinical scenarios, there were some notable differences in the risk benefit trade-offs between the two groups.”
For this pilot study, only a subset of RA-specific questions (n=18) that were supported by moderate to high quality evidence were presented to the panel, based on the assumption that patients would not feel prepared to make judgments in the absence of sufficiently robust data.
Patients and Physicians Contrast in How to Weigh Side Effects
The patient panel developed recommendations for 16 out of the 18 questions and chose not to vote on two questions because they felt that they did not have enough direct data to support a recommendation.
For 13 of the remaining 16 questions, the patient panel voted in the same direction as the physician-dominated panel. The strength of the recommendation (conditional vs. strong) was the same across both panels for 10 out of these 13 recommendations.
Dr. Fraenkel observed a few notable differences between the patient and physician panels. In certain cases, patients placed more emphasis on quality of life (e.g., minimizing gastrointestinal side effects) and the potential benefits of a treatment.
For example, the physician-dominated panel voted conditionally against triple (versus mono-DMARD) therapy whereas patients voted conditionally for using triple therapy for DMARD-naïve RA patients with at least moderate disease activity.
The patient panel concluded that the increased chance of significant improvements (e.g., remission) associated with triple therapy and the lack of significant added toxicity found in studies justified the use of three medications.
Even though the patients differed from physicians in their recommendation, they labeled their recommendation only as conditional to reflect that some patients would differ when weighing the practical and psychological burdens of taking three medications.
The two panels also differed in the direction of their recommendations in one case. The patient panel voted for using tofacitinib over methotrexate in DMARD-naïve RA patients, whereas the physician-dominated panel voted against using tofacitinib in this population.
Ultimately patients voted in favor of tofacitinib because of the statistically significant incremental benefits associated with tofacitinib and its lower risk of gastrointestinal side effects (a side effect felt to have a significant impact on quality of life) compared with methotrexate.
“Our patient panel demonstrates that—with proper preparation—people living with RA are well equipped to meaningfully contribute to the recommendations that will influence our future treatment,” said Kelly Clayton, a patient participant. “The patient perspective is vital to capture.”
Patient Representatives Contribute to New ACR Guidelines
Currently, the American College of Rheumatology routinely invites 1-2 patients to join its clinical practice guideline voting panels. Seth Ginsberg, president and co-founder of CreakyJoints, sat on this year’s RA guideline voting panel as a patient and as a representative of the arthritis patient community.
“We appreciate that the American College of Rheumatology recognizes the importance of incorporating the patient perspective into its newly published guidelines,” said Mr. Ginsberg. “CPGs have a huge impact on the practice of rheumatology – from both a clinical and insurance coverage perspective – therefore, understanding patient preferences and priorities is critical to ensuring access to the full range of treatment options.”
“Patient-centered care is predicated on the idea that patient and physician voices carry equal weight in health care decision making and this study demonstrates the validity of that premise,” added Louis Tharp, co-founder and Executive Director of Global Healthy Living Foundation, the parent organization of CreakyJoints.
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