A field at a crossroads: Pediatric rheumatology struggling to meet demand, fill trials
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The field of pediatric rheumatology is at a crossroads.
Current projections based on available data predict that the demand for pediatric rheumatologists will far outstrip — possibly even double — the supply of working providers in less than a decade. Meanwhile, pediatric patients are not being enrolled in clinical trials at robust enough rates to keep pace with therapeutic advances.
Source: Colleen Correll, MD
Understanding and addressing these twin hurdles will be critical to the future of pediatric rheumatology as a sub-specialty, and essential to improving the current state of suboptimal access to patient care in many parts of the country.
Jay Mehta, MD, MS, attending physician within the division of rheumatology, and director of the pediatric rheumatology fellowship program, at the Children’s Hospital of Philadelphia, was blunt when asked about the workforce shortage in his field.
“We still have one,” he told Healio Rheumatology.
Some sobering numbers underscore this point.
According to a recently published workforce study from the American College of Rheumatology’s Pediatric Committee, barely more than 300 pediatric rheumatology providers were working in patient care in the United States as of 2015.
“The study predicted that by 2030, this number would decrease to approximately 260,” Colleen Correll, MD, chair of the ACR Pediatric Committee, who also serves as a liaison to the ACR’s Workforce Solutions Committee, said in an interview.
Meanwhile, further data showed that the projected demand for pediatric rheumatologists would increase from 382 providers to 461 during that same timeframe, according to Correll.
“Therefore, according to our model’s prediction, by 2030, the demand for pediatric rheumatologists will be twice the supply,” she said.
Recent data from the ACR’s National Residents Matching Program are similarly sobering. Findings show that just 62.8% of available pediatric rheumatology program slots were filled for the 2023 appointment year.
Although these statistics are ominous for myriad reasons — not least of which being stymied access to care — one key implication that may be overlooked is that with fewer doctors subspecializing in pediatric rheumatology, and thus becoming familiar with its specific treatment landscape, it may be increasingly difficult to safely enroll large numbers of patients in clinical trials.
However, this is just one explanation for the ongoing issue of low clinical trial enrollment in pediatric rheumatology studies, according to Brett Smith, DO, of Blount Memorial Physicians Group and East Tennessee Children’s Hospital.
“Recruitment is likely lower than expected due to high levels of clinical and administrative demands leaving less room for research interests, low volumes of participating patients/families and institutional barriers, in addition to the declining workforce,” he told Healio Rheumatology.
But it doesn’t have to be this way.
For Mehta, what is so surprising about the current situation is that many trainees who are exposed to pediatric rheumatology ultimately are sold on the specialty.
“I take any opportunity I can to speak to medical students or residents about the joys of a career in pediatric rheumatology,” he said. “We create amazing long-term relationships. We think about our diseases creatively. We use knowledge of immunology and work in high-functioning multidisciplinary teams.”
If this message could reach more trainees, it could have a beneficial impact on both the workforce and clinical trial enrollment. However, a deeper understanding of the issues is necessary to reach those goals.
Making a Pediatric Rheumatologist
According to Randy Q. Cron, MD, PhD, director of pediatric rheumatology at the University of Alabama at Birmingham, the factors causing the workforce shortage are multifactorial and complex.
“There are more than 30 pediatric rheumatology training programs in the United States, but all slots are not always available, often due to lack of funding,” Cron said in an interview. “Also, not all slots fill, often due to low numbers of applicants.”
Even those who do graduate may not contribute to the U.S. workforce.
“Many graduating fellows often return to their countries of origin, which is good for the planet, but not necessarily the workforce in the United States,” Cron said.
A companion point is that significant resources are required to train a pediatric rheumatologist, according to Cron.
“The funding often comes from the department of pediatrics of the children’s hospital in the area, and it is a fair amount of money,” he said.
Cron noted that at UAB, there is an endowment that allows for a 3-year fellow each year.
“But very few places have these endowments,” he added.
In the absence of this kind of funding, the other alternative is to apply for grants from organizations like the Rheumatology Research Foundation or the Arthritis Foundation, or from pharmaceutical companies.
“But these are one-offs you have to keep writing for, and it is difficult to keep pace with so many other pressing needs,” Cron said.
This is why interventions by organizations like the ACR is so critical.
“The ACR recognized this as a major issue in the field and subsequently created a Workforce Solutions Committee,” Correll said. “The committee is making concrete efforts to improve the workforce through a multi-pronged approach.”
One approach is to increase awareness of the field by emphasizing visiting professorships to medical schools that do not have a pediatric rheumatologist, according to Correll. However, this too comes with structural roadblocks.
“In order for someone to end up in a pediatric rheumatology fellowship, they would probably have to have some knowledge of rheumatology in the first place,” Sangeeta Sule, MD, PhD, chief of the division of rheumatology at Children’s National Hospital, in Washington, D.C., and associate professor of pediatrics at the George Washington University School of Medicine and Health, said in an interview. “But we are mostly an outpatient field. Many of our patients do well and rarely are admitted to the hospital, so trainees on rounds just do not get exposed to our patients or the specialty in general.”
A further hurdle to exposure is that there are many regions of the United States with no pediatric rheumatologists at all. Again, the ACR is fighting the uphill battle to solve this problem.
“We are helping open new fellowship programs in geographically underserved areas that already have pediatric residency programs but not rheumatology fellowship programs,” Correll said. “We are working to pass loan forgiveness programs to help compensate for the relatively low salary for a pediatric rheumatologist.”
According to Smith, federal efforts have been made to offer loan forgiveness through the Pediatric Subspecialty Loan Repayment program, valued at $30 million per year.
“The ACR continues to advocate for additional funding for training slots to meet the needs of the U.S. population,” he said.
There is some optimism among experts that these incentives could address the dwindling number of pediatric practitioners in rheumatology. However, some argue that these incentives should also be paired with messaging about pediatric rheumatology as a specialty.
‘What a Wonderful Field It Is’
According to Sule, both the ACR and Childhood Arthritis and Rheumatology Research Alliance (CARRA) are inviting interested trainees to national conferences to see the benefits of a career in pediatric rheumatology.
“It would be appealing to a lot of people if they knew it existed,” she said. “A lot of residents do not know what a wonderful field it is.”
Mehta added that the Rheumatology Research Foundation and other such organizations are also working to expose more young scientists to the field using various types of media.
“One short video features the story of a patient with lupus,” he said.
This patient, like many others, experienced a long, difficult journey toward diagnosis and treatment, Mehta reported.
“She is now at college and living a happy, healthy life,” he added. “We see this kind of thing every day. This is why many people go into medical training in the first place.”
Like adult rheumatology, pediatric rheumatology offers “interesting and challenging” patient cases, which Mehta suggested is another reason young trainees choose medicine as a field.
“We have an increasing number of medications to treat those conditions, so we are starting to see a lot more successful outcomes,” he said.
However, perhaps the most satisfying component is the opportunity to build relationships.
“We get to see our patients longitudinally,” Sule said. “We get to know their families and we get to see our patients grow up.”
That said, the joy of watching patients grow is balanced by a unique set of clinical challenges they present. Clinicians have had to be creative to meet these challenges despite being short-handed.
‘Complicated Psychosocial Aspects’
Several types of health care professionals have been enlisted to make up for the lack of available pediatric rheumatologist in the workforce. However, by far the two most called upon groups are primary care providers and adult rheumatologists.
“A reliable pediatrician or primary care physician is extraordinarily valuable for comprehensive patient care, particularly with long wait times and complicated cases,” Smith said.
According to Correll, the ACR is working on ways to train general pediatricians and family practitioners to meet the specific needs of young patients with musculoskeletal diseases.
“The goal is for them to care for patients who have musculoskeletal complaints that are not rheumatic diseases,” she said.
Cron explained a common example of why training PCPs is necessary.
“We get a lot of referrals for children who have a positive ANA, but the largest chunk of those patients do not have a rheumatic disease,” he said. “Many of them have some other type of stress-related pain syndrome. Of course, it is important to treat those kids, but they do not necessarily require a rheumatologist.”
These patients create an “immense amount of work” for pediatric rheumatologists who are already overworked, according to Cron.
“If primary care can learn to recognize which kids need a rheumatologist and which kids need stress counseling and aerobic exercise, it would ease our workload,” he said.
A companion approach would be for pediatricians to focus on tasks that do not require the specific expertise of a pediatric rheumatologist.
“Pediatricians aiding in health maintenance, laboratory studies, vaccinations and compliance is a significant help to rheumatology practitioners,” Smith said.
However, if non-rheumatologists are going to handle these duties, it is important for them to have information and resources at their disposal.
“The ACR is working on a project to provide information to primary care physicians and advanced practice providers,” Correll said. “This online resource will be available through the ACR website. It will offer information on differential diagnoses, when to refer, appropriate testing before referrals, potential treatments the PCP or APP can provide, etc. We anticipate launching this project in mid-2024.”
That said, the ACR cannot — and should not — be the only source of information for primary care providers being called into rheumatology service. An increasing body of research will be critical in this regard, as well.
In a paper published in Current Problems in Pediatric & Adolescent Health Care, Spencer and Patwardhan offered a diagnostic approach for juvenile idiopathic arthritis.
“It is necessary to know the personalities of these JIA categories,” they wrote. “It is also crucial to be able to recognize the common infectious, orthopedic and mechanical, malignant, genetic, other rheumatic diseases, and other miscellaneous syndromes that can mimic JIA.”
In the paper, the authors outlined clinical patterns, information on conducting a thorough musculoskeletal and rheumatic history, recommendations for laboratory testing and guidance in following patients over time.
“Adult rheumatologists often do not have the training to understand some of the unique aspects of childhood and adolescents,” Sule said, noting that they can also benefit from such research.
The way young patients grow can have a significant impact on immune and musculoskeletal issues, according to Sule.
“There are also complicated psychosocial aspects of childhood and adolescence that need to be managed appropriately,” she said. “School is also obviously a big issue that often plays a role in our management of these patients.”
Beyond primary care and adult rheumatology, other practitioners, including — perhaps especially — nurses, have increasingly been called in to assist with the pediatric rheumatology workload.
‘Thinking Outside the Box’
In a paper published in BMC Health Services Research, Thomsen and colleagues reported on the Parents in Transition — A Nurse-led Support and Transfer Educational Program (ParTNerSTEPs), which included three parts: an informational website, online support for parents, and transfer consultations with providers in both pediatrics and adult care settings.
“The [UK Medical Research Council] framework was successfully applied to develop a comprehensive transfer program targeting parents of adolescents with chronic illness,” the researchers wrote. “By incorporating the principles of participatory design in the development phase, we ensured that both parents’ and adolescents’ needs were represented and addressed in the program.”
According to Sule, there is an increasing number of courses designed to train APPs and NPs in pediatric rheumatology and transitional care.
“Getting NPs and APPs involved is a great example of thinking outside the box,” she said. “The ACR and CARRA have made efforts during national and regional meetings to educate these practitioners on all aspects of pediatric rheumatology.”
However, transitional care is not the only way that nurses are called into service.
“Skilled nursing staff and APPs are increasingly utilized by rheumatology clinics due to the increasing demands from patient volumes, prior authorizations, EHRs and other administrative demands,” Smith said.
Nurses also tend to be effective communicators, according to Cron.
“Advanced practice nurses can be employed to diagnose and educate these patients and families, many of whom do not require follow up care,” he said.
On a more personal level, Sule said she believes that the attractive qualities of pediatric rheumatology could draw some of these other practitioners into the specialty full-time.
“There are many APPs and NPs who desire to provide the type of longitudinal care we provide, and pediatric rheumatology lends itself to that,” she said.
That said, even as these attempts to draw personnel to the field ramp up and potentially take hold, the issue of clinical trial enrollment remains a concern.
Rethinking the Clinical Trial Design
Heather A. Van Mater, MD, MS, a pediatric rheumatologist at Duke University Medical Center, sees logistical hurdles as a significant barrier to clinical trial enrollment.
“Our patients already have to travel great distances for office visits,” she said. “When you are in a trial, you are often required to have more visits than the standard of care.”
However, Van Mater sees an opportunity to move the field forward in creative ways.
“One thing to consider is to have nurses come to the homes of patients in clinical trials,” she said. “By seeing patients in their homes, we can better assess our patients, their disease and why the treatments are working or not working.”
This model could also be combined with telehealth in clinical trial designs, she added.
“The physician would see the patient as a video visit, and a nurse could do a home visit to draw blood, get vitals, or other aspects that would need to be in person,” Van Mater said, adding that a few of her colleagues are now conducting a study into using telehealth and in-home visits to make trials more accessible to patients. She described the technology’s increased use across the spectrum of rheumatology care as one of the few “silver linings” of the COVID-19 pandemic.
According to Van Mater, the next natural step would be to use remote technology to increase clinical trial participation.
“We need to rethink clinical trial design across the board,” she said.
Another way, according to Van Mater, to update pediatric rheumatology clinical trials is to rethink the necessity of the washout period.
“It is difficult to ask a child with active arthritis or lupus to stop taking any further treatment until they can get on a trial,” she said. “We will be asking them to go through a period of time when they will feel worse before they can get on this new medication that might make them feel better. So, it is not surprising many families decline to participate.”
Van Mater additionally encouraged rheumatologists of all stripes to make their voice heard regarding pediatric trials.
“If you have the opportunity to participate at a site for a pediatric trial, you should give feedback as often as you can,” she said.
However, although many pediatric practitioners would, in fact, participate in a trial, the opportunities to do so are limited.
“Many rheumatic diseases, particularly in pediatrics, are rare, such as the ANCA vasculitides, but even for JIA there are multiple subtypes, not all of which are part of the inclusion criteria for clinical trials,” Cron said. “There are also rarely if ever clinical trials that compare new therapies, such as biologics, to established therapies like TNF inhibitors.”
According to Correll, there are, at times, also multiple studies for the same disease, so eligible patients may have already enrolled in one study and cannot enroll in another.
“Most clinical studies require a multicenter approach for participation to reach adequate sample sizes,” she said. “Mainly the issue with getting children to enroll in studies is that pediatric diseases are all rare diseases, so we just don’t have large sample sizes.”
That said, even when the field can move past these structural barriers to clinical trial participation, there are still personal issues to be considered between doctor, patient and parent.
Looking for ‘Creative’ Solutions
Conventional wisdom states that pediatric patients — or their parents or guardians — are reluctant to join clinical trials due to concerns about risk.
“It can be hard to enroll kids in clinical trials for this reason,” Mehta said.
However, Correll is not necessarily convinced of this.
“Many pediatric patients are motivated to participate in clinical trials, particularly when their disease is poorly controlled,” she said.
That said, if the perceived issue is that of parent or guardian hesitancy, the obvious solution is education, according to Sule.
“We need people to understand why clinical trials are so necessary,” she said. “They need to know that this is how drugs are developed, and that if we have more drugs available, it will benefit the lives of their children.”
It is similarly important to communicate to parents that by the time drugs are being used in patients of any kind, they have passed through a rigorous safety assessment.
“Parents and patients need to know about monitoring boards and other safeguards,” Sule said. “These kinds of procedures are as safe as we can possibly make them.”
The conversation should be personal and address the parents’ or guardians’ concerns as directly as possible, according to Mehta.
“We need to help parents understand that we are not just experimenting on their children,” he said. “I do not know if this message always comes across.”
An organization like CARRA — which, according to their website, has “become a major driver of advances in evidence-based medicine and career development in pediatric rheumatology” — is critical to advancing these educational goals.
However, educating parents is just one part of the equation. Van Mater, for her part, described “pushback” from the FDA about certain requirements of clinical trial design.
“We need to take advantage of opportunities to work with the ACR and FDA and be thoughtful about how we approach safety and monitoring,” she said.
The goal would be to gather information on drugs in a “more practical way that could be more attractive and useful for families,” Van Mater said.
Another consideration is that the FDA has made allowances so that companies that have marketed drugs to adults do not have to perform studies in children, according to Mehta.
“The result is a much more limited range of DMARDs, biologic DMARDs and oral small molecules available for pediatric use compared with adult use,” Smith said. “Additional interest from the pharmaceutical industry would be beneficial to the global pediatric rheumatology community.”
Mehta agreed but also offered a counterpoint from the pharmaceutical perspective.
“Adding children to an indication for a drug does not add much market value,” he said.
Although many adult rheumatology drugs are indeed used effectively and relatively safely, by FDA standards, in pediatric patients, Sule believes that targeted research must be ongoing.
“Kids are very different biologically from adults in the way they may respond to the drugs and the adverse events they experience,” she said. “We cannot continue to treat children based on results in adult populations.”
That said, although pediatric rheumatology providers know that it is a “difficult conversation” when discussing clinical trial enrollment with a parent, some are relieved to know that the drug has been vetted in an adult population, Sule added.
“Parents are most scared about using drugs that are new on the market,” she said. “They ask what may happen to their child years from now. There are a lot of uncertainties in pediatric rheumatology care.”
However, appealing to the altruism of parents with sick children can be useful, according to Sule.
“Of course, parents want their children to get better,” she said. “But they also want other children to get better. We need to let them know that clinical trial enrollment is a way to accomplish both of these goals.”
For this and for the other challenges facing pediatric rheumatology, Mehta offered an over-arching final point: “It is going to take some pretty creative solutions.”
- References:
- ACR Match report: Press Release: 2022 Specialty Match Day (rheumatology.org)
- Spencer CH, Patwardhan A. Curr Probl Pediatr Adolesc Health Care. 2015; doi:10.1016/j.cppeds.2015.04.002.
- Thomsen EL, et al. BMC Health Serv Res. 2022;doi:10.1186/s12913-022-07888-5.
- For more information:
- Colleen Correll, MD, can be reached at 2450 Riverside Ave., Minneapolis, MN 55454; email: mmcdonald@rheumatology.org.
- Randall Q. Cron, MD, can be reached at 1600 7th Ave. S # Acc64, Birmingham, AL 35233; email: randycron@uabmc.edu.
- Jay Mehta, MD, MS, can be reached at 3405 Civic Center Blvd., Philadelphia, PA 19104; email: mehtaj@email.chop.edu.
- Brett Smith, DO, can be reached at 232 Associates Blvd. Alcoa, TN 37701; email: blsmith@protonmail.com.
- Sangeeta Sule, MD, PhD, can be reached at 111 Michigan Ave. NW, Washington, DC 20010; email: ssule@childrensnational.org.
- Heather A. Van Mater, MD, can be reached at 2301 Erwin Rd., Durham, NC 27710; email: heather.vanmater@duke.edu.
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