Interview conducted by April Cashin-Garbutt, MA (Cantab)
Please can you outline the recent pilot program that evaluated the integral participation of patients in the development of clinical practice guidelines (CPGs) for rheumatoid arthritis?
The study, “When patients write the guidelines: Patient panel recommendations for the treatment of RA,” published in Arthritis Research & Care, demonstrated the feasibility of developing CPG recommendations based on a voting panel composed entirely of patients.
Notably, it showed that patients developed nearly the same recommendations as physician-dominated panels for questions (n=18) where there was evidence warranting moderate to high confidence.
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.
Participating patients were recruited from the Arthritis Power Patient Governors group and trained in advance in the identical methods used by participants who create the official ACR patient treatment guidelines in order to conduct an accurate recreation of the guidelines approval process.
When there were differences between the patient-panel and physician-dominated panel (the typical make-up of the ACR CPG panel), they usually revolved around risk vs. benefit.
Patients weighed “non-serious” adverse events, such as gastrointestinal discomfort, more troublesome than physicians, for example.
On the other hand, they were also willing to inconveniently take more pills if there was a notable efficacy uptick with fewer side effect risks for a particular treatment option.
This study is the result of a close collaboration among lead author Liana Fraenkel, M.D. of the Yale University School of Medicine, the American College of Rheumatology, CreakyJoints, and many Arthritis Power Patient Governors who participated in the project and contributed to the analysis.
Arthritis Power is the first ever patient-led, patient-generated, patient-centered research registry for arthritis. It is a joint project between CreakyJoints, the University of Alabama at Birmingham and the Patient Centered Outcomes Research Institute.
What are the benefits of including patients in the development of CPGs?
While healthcare provider priorities often revolve around clinical outcomes or concerns, little has been done to explore which concerns are the most pressing to the rheumatic disease patient community.
As this study demonstrated, while the priorities and risk-benefit valuations made by patients may differ from physicians, patients have the ability to understand and evaluate evidence similar to physicians. Therefore, it is important that panels that develop and update CPGs include this perspective in order to address the needs of patients.
How did the pilot study differ in terms of the number of patients typically involved in determining the final guideline recommendations?
The American College of Rheumatology has been really forward thinking in including patients in the CPG updating process.
Currently, the ACR invites two patients to join their clinical practice guidelines evaluation panel. In fact, Seth Ginsberg, president and co-founder of CreakyJoints, sat on this year’s panel as a patient and as a representative of the arthritis patient community.
The patient panel pilot program included 10 patients and we worked directly with the ACR to implement this pilot study.
Representatives from the professional organization provided the patient training, ran the evaluation session, and managed the technology to record the recommendations.
The ACR recognizes the needs to include patient perspectives and to have them weigh in on key issues, particularly since the CPGs affect treatment decisions and, possibly, access to treatments.
Was the pilot a success?
Yes. The pilot study demonstrated that, with the same training, rheumatology patients are able to participate on a guidelines panel. Patients received a comprehensive training regarding how to evaluate evidence.
Patient recommendations mirrored the conclusions drawn by the physician-dominated panel working on the official 2015 ACR Guidelines for Treatment of RA, which was published in parallel with this study.
While we knew there was support from guideline developers to hear the patient voice, as evidenced by the ACR already including two representatives on the existing panel, there were questions regarding how effectively an all-patient panel could be.
The study demonstrates that patients are fully able to participate in an exercise that tangibly affects treatment and access to treatment of RA. Patient communities across other conditions have an important role to play in setting the standards for treatment.
How did patient recommendations compare to physician dominated panels for questions where there was evidence warranting moderate to high confidence?
In this study, the patient panel only evaluated 18 of the 74 questions evaluated by the official ACR panel. Of those, the patient panel determined that 16 had enough evidence in order to make recommendations.
For 13 of those 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.
What areas did patients and physicians differ in opinion?
Notable differences typically came down to how patients rated quality of life (e.g., minimizing gastrointestinal side effects), convenience and potential treatment benefit.
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 labelled their recommendation only as conditional to reflect that some patients would differ when weighing the practical and psychological burdens of taking three medications.
Another difference, 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.
What impact do you think this study will have on future panels?
CPGs have a huge impact on the practice of rheumatology – from both a clinical and access perspective – therefore, understanding patient preferences and priorities is vital.
This study demonstrates that patients can effectively contribute to a process that directly impacts the practice of RA treatment.
Given the ACR’s direct involvement with the study, we’re hopeful that patients will continue to be part of their CPG panel and in greater numbers in the future.
What are CreakyJoints plans for the future and how do you think patient recommendations will influence rheumatoid arthritis treatments going forwards?
CreakyJoints, part of the not-for-profit Global Healthy Living Foundation, is a dynamic education, support, advocacy and patient-centered research organization for people with all forms of arthritis and rheumatic disease. We have over 80,000 members worldwide and we offer all of them the opportunity to participate in research and influence the future of arthritis treatments.
Specifically, CreakyJoints, in collaboration with the University of Alabama at Birmingham, and supported by the Patient-Centered Outcomes Research Institute (PCORI), created Arthritis Power, the first ever patient-led, patient-generated, patient-centered research registry for arthritis.
Arthritis Power is part of PCORnet, the National Patient-Centered Clinical Research Network, a large, highly representative, national network for conducting clinical outcomes research.
Using a web-based and mobile application, and focusing on rheumatoid arthritis as well as other musculoskeletal conditions, the goal of Arthritis Power is to collect PRO health data from tens of thousands of arthritis patients to support future comparative treatment research.
Arthritis Power includes a committee of people living with forms of autoimmune arthritis called Patient Governors, who identify research needs for study development and prioritize research requests from the CreakyJoints patient community around the world.
It’s from that group that we primarily recruited for this particular guidelines study. Looking ahead to 2016, Arthritis Power will undergo a relaunch as we enhance the interface to be even easier to use.
We’ll also continue to offer patients the opportunity to track their health (and send that data directly to their health care team) and participate in research studies via notifications on the web-based and mobile platform.
Also, with support from PCORI and in partnership with the RAND Corporation, CreakyJoints is currently conducting a training initiative focused on research into joint replacement implant device safety and selection.
The title of the initiative is Bringing Stakeholders Together for Engagement in Research for the Selection of Arthroplasty Implant Devices (BeTTER SAID). CreakyJoints continues evolving to be both a patient support, advocacy and research organization.
Where can readers find more information?
Arthritis Power is the first ever patient-led, patient-generated, patient-centered research registry for arthritis. Focusing on rheumatoid arthritis as well as other musculoskeletal conditions, the goal of Arthritis Power is to collect health data from tens of thousands of arthritis patients to support future research to compare treatments, identify new ones, and, perhaps, find elusive cures. To learn more and join Arthritis Power, visit www.ArthritisPower.org
About Dr Nowell
W. Benjamin Nowell, PhD, is Director, Patient-Centered Research for CreakyJoints. After graduating with a BA from Gonzaga University in 1994, Ben served as the Pacific Region’s regional team leader for AmeriCorps*VISTA Integrated Training and consulted as a core curriculum writer to redesign AmeriCorps*VISTA training across the U.S.
He co-founded a leadership program for AmeriCorps members in Washington and Oregon that recently graduated its twelfth class of service leaders.
Prior receiving his PhD from Columbia University School of Social Work in 2014, Ben earned his Masters in Social Welfare Management and Planning (M.S.W.) from the University of California, Berkeley in 2004.
He spent the next three years helping develop the Regional Stroke Centre at The Ottawa Hospital in Ottawa. Since moving to New York City in 2007, he has been involved in the community as a New York City chapter leader of AmeriCorps Alums and a partner with UN representatives to train staff on HIV policy and preventive health.