It takes a great deal of time and money to produce clinical practice guidelines for rheumatic diseases. No matter how well a treatment inhibits inflammatory cytokines, it won’t lower disease activity without one essential factor: patient compliance.
“You can’t propose a treatment algorithm in your research that no patient would actually use,” says Veena Ranganath, MD, a rheumatologist who conducts rheumatoid arthritis (RA) research at the Ronald Reagan Medical Center at the University of California, Los Angeles. Patient input in the grant-development stage, as well as during clinical trials, can help shape rheumatology research and lead to cohesive physician–patient-oriented research, she notes. “If researchers propose major changes to the standard-of-care manner of treating rheumatology patients, it is important to consider feasibility and patient buy-in to the research concept.”
The Lived Experience
Patients are playing a more active role in rheumatology research than ever before. As highly valued stakeholders, patient advocates serve on grant review panels, guidelines development task forces and committees that craft meaningful outcomes measurement tools.
The American College of Rheumatology (ACR) Policy and Procedures Manual for Clinical Practice Guidelines, published in January 2015, states that at least one knowledgeable consumer and one patient advocate must be included in guideline project development groups.
In 2010, the European League Against Rheumatism (EULAR) passed recommendations to include patients in rheumatology research and published a set of reference cards to help researchers and patients work together more effectively. The introduction to the cards states that patient stakeholder engagement “improves both the methodology and outcomes of the research, and offers invaluable insights. Contributions by patients to the design, implementation and evaluation of research leads to effectiveness, credibility and often to more cost efficiency as well.”
It is patients’ “lived experience” with rheumatic diseases that helps steer research in practical directions, says leading patient advocate Amye Leong, an RA patient who has become extensively involved in rheumatology research through such organizations as Outcome Measures in Rheumatology (OMERACT), the American College of Rheumatology, the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), and the Patient-Centered Outcomes Research Institute (PCORI).
“I’m stuck with this disease for the rest of my life,” says Ms. Leong, who is the founder and chief executive officer of Healthy Motivation, a health education and advocacy consulting firm based in Santa Barbara, Calif., and Paris, France. She was diagnosed with RA at 18, and went from living on Social Security disability to becoming a respected arthritis speaker and educator, and serving on the NIAMS Advisory Council and its Clinical Trials Grant Review Panel. “Patients can help researchers better understand the orders of importance of measurable outcomes. Every decision that researchers make could change the course of my disease.”
When Ms. Leong first became involved in arthritis advocacy in the 1980s, it was purely in the patient sphere. She helped to organize the first support groups for young patients with rheumatic diseases in Southern California. Arthritis patients may have been subjects of study in clinical trials, but they were not invited to high-level discussions about rheumatology research strategic directions.
Ms. Leong was invited to OMERACT’s 2002 meeting in Brisbane, Australia, where she met with global rheumatology researchers to discuss measurable outcomes in rheumatic disease. When patients share their firsthand knowledge of living with RA, they can shift researchers’ views on what matters most in RA disease activity or treatment, she says.
“It is still a challenge sometimes, because some researchers assume that patients ‘don’t know what I know.’ So we have an opportunity to teach researchers to refrain from dismissing the importance of patients’ experience,” says Ms. Leong.
At the 2002 OMERACT meeting, Ms. Leong and other RA patients shared their opinions on the importance of such outcomes as sleep and fatigue. OMERACT’s Patient Research Partners program continues to engage patients with researchers in developing new tools and recognizing areas of concern for rheumatic disease patients, such as tolerability of MRI scanning duration.
At the organization’s 2014 meeting, psoriatic arthritis patients participated in a working group to refine core responder indices for this disease. Across the research spectrum, patient stakeholder engagement is becoming more prevalent. The Food and Drug Administration Safety and Innovation Act (FDASIA), passed in 2012, requires patients to be involved in drug development. The FDA’s Patient-Focused Drug Development initiative includes a series of five meetings with patients to discuss the impact of disease on their lives. Patient-reported outcomes are now included in the current ACR/EULAR RA remission criteria, developed by a committee that included RA patients.
Both patients and researchers can learn from each other, says Ms. Leong. “There is room for developing an infrastructure or process for new researchers to learn how to engage patients and vice versa,” she says. “I have learned that so much good can come from articulating
the patient’s experience in the research discussion. We can influence how a researcher understands concepts based on lived experience.”
Guiding Emerging Researchers
Patient stakeholders continue to influence new research, says Jennifer Leese, a research trainee and doctoral student at Arthritis Research Canada in Vancouver, B.C., Canada. Her work includes evaluating new tools to support self-management by patients with musculoskeletal disease.
“Involving patients as active partners in research is important because it opens opportunities for research to be more relevant, credible and accountable to issues that matter most to patients. Involving patients from the early stages of the research process can enhance the quality of research evidence, clinical practice and health outcomes,” says Ms. Leese. “In recent decades, an increasing body of evidence has shown discrepancies between what arthritis patients and health professionals prioritize as important outcomes.”
Ms. Leese is one of several researchers working with her institution’s Arthritis Patient Advisory Board launched in 2001. Both patients and researchers collaborate on a program called “It IS About Us,” and they “aim to develop a how-to manual that can guide patients and research teams or organizations in developing a practice of patient engagement in research based on patients’ perspectives,” says Ms. Leese. The patient participants mentor emerging researchers to develop better communication skills with patients in their work, she adds.
At UCLA, Dr. Ranganath has worked on a number of clinical research studies involving RA patients, including use of ultrasonography and magnetic resonance imaging to assess structural damage and inflammation. Rheumatology research should be driven by what may benefit patients the most, she says.
“If research is not applicable to the patient sitting in your clinic, then why do it?” says Dr. Ranganath. Patients help drive rheumatology research where it needs to go, she says. “Patient enrollment in early tumor necrosis factor inhibitor trials led to FDA approval of the first biologic drugs in RA. Now, rheumatologists have several medications to choose from to treat RA. Patients are extremely important in the research process.”
Many sources of rheumatology research funding, such as NIH or PCORI in Washington, D.C., require patient involvement as part of their grant criteria, says Dr. Ranganath. Researchers need to treat patients in their clinical trials as valued equals in the process.
“It should be a collaborative effort between the physician and the patient,” she says. Patients should not feel that they are in a passive role. “It is critical for the successful conduct of clinical research that there [be] trust between the patient and the rheumatologist. There should always be two-way communication.”
Patient engagement at every level of rheumatology research, from clinical trials for new drugs to international retreats formulating measurable outcomes, has greatly improved the quality of life for people with rheumatic diseases, says Cheryl Koehn, who has had RA for 27 years. In 2000, Koehn founded Arthritis Consumer Experts, a national patient advocacy group headquartered in Vancouver, B.C., Canada. Ms. Koehn has chaired the Canadian Arthritis Network’s Consumer Advisory Council, among other national leadership roles.
“What I bring to the table may be the most important but least measurable factor in arthritis research. I bring my lived experience into the room,” says Ms. Koehn, who is also a consumer representative with and board member of the Arthritis Alliance of Canada. She and other patient advocates play an active role in rheumatology research by directly impacting project protocols and research methodologies. “The conversation changes and deepens. Suddenly, it is not just a bunch of researchers and scientists sitting in a room talking in the abstract, but they are talking with someone who is living with the disease. I share a perspective that they may not know otherwise. They may not know that on some days my RA is so bad that simply looking after daily hygiene is difficult.”
Patient input has made an important impact in the development of new therapies, says Ms. Koehn.
“When the first, Phase I papers on biologics were being presented at the ACR meeting, and at every plenary session for years, patient advocates like me kept asking, ‘When is it going to become available in a pill?’ Patient demand and input helped even if in a small way to develop something that patients didn’t mind taking. And now we have tofacitinib,” says Ms. Koehn. “That is one way we can contribute and impact the product stream. If you’re a patient, get involved in a clinical trial or study. It’s through the desire to get better and practicing altruism that science advances.”
Basic scientists, more than clinicians, may benefit most from patient feedback because they have little opportunity to talk to people living with rheumatic diseases, says Ms. Koehn.
However, recruiting patient volunteers to participate in clinical trials or policy-shaping meetings is challenging. Patients may be busy with work or family obligations and have little extra time for studies, Dr. Ranganath notes. They may not understand how their time and effort will contribute to better disease outcomes in the future.
Some funding organizations, like PCORI, require patients to be compensated financially for involvement in research projects, or patients may be paid as an incentive to participate in clinical trials.
‘Suddenly, it is not just a bunch of researchers & scientists sitting in a room talking in the abstract, but they are talking with someone who is living with the disease.’ —Cheryl Koehn, president, Arthritis Consumer Experts
Patients may support research in different ways according to their interest or experience. The Rheumatology Research Foundation, which has granted more than $131 million to research and training since 1985, recently created a strategic planning committee that includes patients who offer guidance as they plan future funding initiatives, says Mary Wheatley, the Foundation’s executive director.
“Our mission is to improve the health of people with rheumatic disease, so everything the Rheumatology Research Foundation does is focused on the patient,” says Ms. Wheatley. “Our fellowship grants were created to ensure patients have access to qualified, well-trained rheumatologists, and we fund innovative research that will one day lead us to cures for patients with rheumatic diseases. By engaging patients in goal setting, we can be sure we’re on the right track.”
Technology may be able to help bridge logistical gaps, and bring more patients into rheumatology research in meaningful ways, says Ms. Leese. In 2013, Arthritis Research Canada’s Arthritis Patient Advisory Board cohosted a public forum with University of British Columbia researchers. Patients could attend in person or through a live webcast, or swap comments on the social media network, Twitter.
“By using these technologies, a larger and more diverse audience was able to engage with ARC research than if only an in-person event had been held,” says Ms. Leese. Three patients who followed the event on Twitter have since joined the patient advisory board of the organization, she says.
Technology, such as online survey tools, makes it easier for researchers to collect data from patients and cast a wider net to include patients who may live far from the clinic, says Dr. Ranganath. Web- or smartphone-based apps may one day be able to help rheumatology researchers gather data from patients in large cohorts, she says.
Ms. Leong would like to see rheumatology organizations work together to create webinar training modules to help arthritis patients get involved in research efforts. “We could help patients understand what research is and why it is important, and attune researchers to the needs and issues that are important to us,” she says.
The rheumatology community should involve all important stakeholders in driving research, says Ms. Leong.
“Let’s create a culture of thorough, patient-centered, realistic and cost-effective research,” Ms. Leong says. “It is important to not just have a token patient on a committee, but to create a research environment that captures the breadth of disease experience for researchers to create meaningful, measurable progress and outcomes. Patients win when researchers win.”
Susan Bernstein is a freelance medical journalist based in Atlanta.