Many rheumatology researchers share concerns about the landscape for future opportunities, given recent pressures from the current presidential administration. These may make an already challenging environment even more difficult for academic physicians to successfully navigate. But many in the field will persevere despite obstacles, driven by the need to deepen understanding of these conditions and ultimately improve long-term quality of life for people with rheumatic diseases.
Value of Rheumatology Research
The ACR’s core mission is empowering rheumatology professionals to excel in their specialty. A key component of that is promoting efforts to continually advance the field and improve patient care. This is not possible without, among other things, new insights and guidance gleaned from high-quality research initiatives.
Research needs are even more apparent for rheumatic diseases that have been less studied than RA, with consequently fewer approved therapies, such as spondyloarthritis. And in some rheumatic diseases, such as systemic sclerosis, some patients still die directly from their disease.
Sonali Bracken, MD, PhD, a research fellow in the Division of Rheumatology at Duke University, notes, “We’ve improved quality of life and longevity across many rheumatologic diseases, but we still have a long way to go.”
Ed Behrens, MD, chief of the Division of Rheumatology at Children’s Hospital of Philadelphia, underscores the ongoing need to develop more targeted treatments to address the underlying pathophysiology of a given condition. “When you get drugs more specific to the biology at hand, you get more effective cures with less side effects,” he says.
Relatedly, Dr. Bracken points out, a major long-term goal in the field is the development of therapies that can achieve disease control without the degree of immunosuppression that is currently needed, eliminating current concerns such as increased risks of infection or cancer.
However, developing such targeted therapies requires a sophisticated understanding of underlying pathophysiology. This is part of why basic science research about immunology, autoimmunity, inflammation, tissue repair and fundamental disease topics is also so essential.
Dr. Behrens points out that research progress is often slow, requiring many incremental steps along the way. “Sometimes there are big innovations that happen, like the [tumor necrosis factor] inhibitors, but they’re not coming out of nowhere when that happens,” he adds. “We’ve got to be willing to invest in that slow pace to make those big things happen.”
In addition to developing new therapies, research is essential for diagnostic improvements, developing better biomarkers and identifying environmental and lifestyle risk factors. Research is also necessary for solidifying evidence-based medicine approaches, promoting the development of sounder clinical practice guidelines to support high-quality care. Although not yet realized, researchers are also working to understand what approaches might actually prevent full-blown disease.
Rheumatology research on complex disease mechanisms can also yield data critically important for other fields, such as cancer or infectious diseases. Previous innovations in rheumatology, such as the tumor necrosis factor inhibitors, are now also commonly used in other fields, such as gastroenterology and dermatology. Research in rheumatology has also heavily influenced other fields with respect to such areas as precision medicine and patient-reported outcomes.
Even beyond the personal impact of rheumatic diseases on patients and their families, these diseases substantially impact society. As leading causes of workplace disability and decreased productivity, the social and economic burdens of rheumatic diseases are hard to underestimate.
Challenges in Rheumatology Research
Given just the scope and complexity of both rheumatic diseases and new research tools, making real research progress is demanding. But additional practical constraints can make pursuing a career in academic rheumatology even more challenging.
Broad Funding Challenges
Dr. Kuhn sees obtaining sufficient grant funding as the number one barrier to making important discoveries in rheumatology research.
The National Institutes of Health (NIH), the largest public funder of biomedical research in the world, funds less than 1 in 10 grant applications. Even when a grant is obtained, Dr. Kuhn explains that impactful studies often require a larger sum than many grants can provide. Many grants don’t allow for the kind of risk-taking that is necessary for paradigm-shifting studies, she notes. If grant reviewers aren’t convinced that a study can be completed on time for a certain amount of money, they may be unlikely to choose to fund it.
“I think it’s discouraging for people, not being able to do the research you want to do with the amount of funding you get,” says Dr. Kuhn.
Rheumatology is also relatively underfunded compared to some other areas. For example, funding to the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) has remained low compared to other institutes of the NIH.1
“If you look at what we spend and what gets raised, for instance, to fight the war on cancer, it dwarfs what we’re spending for autoimmune research,” notes Dr. Behrens. “We should be fighting a war on autoimmunity.”
Dr. Bracken argues that the available research funding has not kept pace with increasing expenses in science. Labor and other resources have become more expensive, but publishing expectations have also changed, often requiring highly sophisticated and expensive methods. “Young researchers often do not have access to the funds required to do these experiments, so it can be especially challenging for new investigators to get high-quality papers published at a fast enough rate to establish a track record that invites funding,” she says.
Awards exist specifically to help launch early career researchers, but Dr. Bracken notes that although these often cover salary, they may not provide a lot of money to cover additional costs necessary to advance the research. For early career researchers, Dr. Bracken points out how essential it is to have a well-resourced mentor to help navigate this. But that can sometimes also create a challenge for young researchers, as dependence on a mentor and ownership issues can slow the process of becoming an independent scientist.
For some, the pressures of finding funding ultimately drive the choice to pivot to other types of more secure career paths (e.g., purely clinical jobs or employment with industry, even if they’d rather have kept pursuing research).
Although young researchers face particular challenges, Dr. Kuhn notes that funding remains a stress and a challenge throughout one’s career. She advises investigators to diversify their sources of funding, seeking multiple sources such as the NIH, industry and private medical foundations.
“Funding challenges happen to everyone, and obtaining funding is hard for everyone,” says Dr. Kuhn. “You really have to keep pushing yourself along and surround yourself with a lot of positive energy from colleagues so that you can maintain resilience.”
Additional Current Funding Concerns
Concerns about funding, already present, have heightened with choices made by the new political administration. On Feb. 7, the NIH announced that it planned to drastically cut payments to cover institutions’ indirect research costs, a percentage of the overall grant money. These indirect costs, the percentage of which vary by institution, are meant to go toward broad research needs, such as equipment and facilities maintenance, information technology and administrative costs.
Many research groups and universities criticized the move, arguing that it would force universities to shoulder large unbudgeted expenses, slowing research progress. In a later court filing, the NIH claimed that the total grant spending would not change under the new policy, but that grant spending would be directed more toward direct research funding as opposed to indirect costs. At present, implementation of the new policy has been temporarily blocked by a court order currently undergoing a legal appeal.
Dr. Behrens points out that reasonable people might argue that this course of action and the speed of its potential implementation is not for the best, as grant cuts to these institutions may impact scientific output. But he points out that discussions about the percentage of grants devoted to indirect costs are not new, and the best way to resource science research is a debate that shifts over time.
“Resources are not unlimited, so I think it’s right for us to discuss how much goes in what bucket. Let’s be open to having an open dialogue about the right model for enabling this research,” Dr. Behrens adds. “The only wrong answer is to stop doing it.”
Many have also voiced concerns about the administration’s choice to freeze the review of new grant applications at the NIH, although this has now been partially lifted. Another related concern applies to researchers whose work intersects with issues of racial differences, socioeconomic disparities or related factors that can influence health. In response to an executive order on diversity, equity and inclusion (DEI), funding for research with any applicability to this topic may not receive funding, including some previously approved projects, and a significant number of projects have been terminated. The administration has tried to pressure universities, such as Columbia, to make ideologically based policy changes in order to receive funding, which may limit their ability to provide bridge funding to individuals’ whose NIH funding status is now uncertain. Moreover, the administration has also implemented massive funding freezes and cutbacks at the NIH itself.
Dr. Bracken sees cutting funding as the wrong approach. Rheumatic diseases are influenced by genetics, environmental and socioeconomic factors, and work related to DEI has important implications for patient care, she points out. “But there is an opportunity here to reform the way that we fund scientists. There are a lot of inefficiencies in the way that we currently do things, and there’s an opportunity for us to evaluate the system and improve it.”
“We continue to move ahead and pursue our research,” says Dr. Kuhn. “But we’re realistic, and we’re listening carefully. I think the key thing will be how we pivot in response to changes in the way funders want us to focus our research.”
Personnel Challenges
The resourcing challenges of rheumatology research extend beyond money. The rheumatologist shortage, projected to worsen over coming decades, may limit not just clinical availability but also the availability of MD/PhDs to perform important translational work in rheumatology. Even before the most recent research concerns, many people dropped out of their research career track between receipt of an NIH career development award (K series) and obtaining an R01-level project grant.2
The time pressures can be intense for academic physicians, as they balance tasks such as clinical responsibilities, teaching, direct research and other indirect research tasks such as administrative responsibilities. In addition to funding concerns, a survey found that poor institutional research infrastructure, local politics, lack of mentorship and unclear career developmental pathways were also associated with the choice to leave research.2
“One of my biggest concerns is that we’re going to see an exodus of individuals from research careers,” says Dr. Kuhn.
Challenges Inherent to Rheumatology
Researchers in rheumatology also face additional challenges from the nature of the topic itself. For example, many rheumatic conditions are relatively rare. Dr. Behrens points out that this sometimes makes it necessary to perform collaborative, multi-center studies to recruit enough patients. However, he also emphasizes that, taken as a group, rare diseases are the most common type of medical condition, so they definitely deserve scientific study.
Many rheumatic diseases display variable presentation, making them difficult to definitively classify and diagnose. Treatment response can also vary significantly, which can make it difficult to define the right trial end points. And some of the most important outcomes of rheumatic diseases, like pain and fatigue, are hard to objectively measure, all posing challenges for study design. Additionally, the chronic nature of these diseases often means that long-term trials are needed to fully evaluate response.
The heterogeneity of many of these conditions, which display variable clinical and molecular phenotypes, also poses both a challenge and an opportunity, as researchers learn how to better understand the underlying disease pathophysiology. Current disease classification systems may have contributed to multiple negative trials in diseases like systemic lupus erythematosus. By necessity, researchers across rheumatology have begun to define different disease endotypes to improve trials results and advance treatment options for patients.
Value of Communication and Advocacy
Despite these challenges, researchers continue their work, driven both by a fundamental intellectual curiosity and by their desire to decrease disease burden and improve patients’ lives.
Communicating the value of this rheumatology research is key, and not just to the individuals who make specific funding decisions. “It’s important that we as a field stay united and advocate for everything that we do,” says Dr. Bracken. “I think it is going to be more and more important for scientists and physicians alike to be able to communicate better with the lay public about why what we do is important.”
Dr. Behrens shares his experience working with the devastating condition macrophage activation syndrome, which was poorly understood and lacked specific treatments early in his career. Through fundamental research to understand the underlying science, the condition now has targeted treatments and a much improved prognosis. “It’s not a fairy tale to say that we can make huge strides and have real successes,” he says. “Now is not the time to back off of that.”
Ruth Jessen Hickman, MD, a graduate of the Indiana University School of Medicine, is a medical and science writer in Bloomington, Ind.
References
- Nguyen AT, Aris IM, Snyder BD, et al. Musculoskeletal health: An ecological study assessing disease burden and research funding. Lancet Reg Health Am. 2024; Jan 8;29:100661.
- Ogdie A, Shah AA, Makris UE, et al. Barriers to and facilitators of a career as a physician-scientist among rheumatologists in the US. Arthritis Care Res (Hoboken). 2015 Sep;67(9):1191–1201.