The initial CARRA strategy was to support members by identifying grant opportunities and providing both a ready group of collaborators with patient populations and the scientific endorsement of the appropriate experts within CARRA. A critical step forward was the first two-year contract from the AF, “to find out if we could to go from our blueprints to a working organization,” says Dr. Mellins. The AF has been a consistent source of core financial support for CARRA since 2002.
CARRA Today
“CARRA is important because the diseases we are interested in are relatively rare. One center can’t do a definitive study with the limited number of patients there. CARRA enables 300 investigators at 92 sites to pool resources, synergize, recruit the required number of patients, and do rigorous research,” says Norman T. Ilowite, MD, chair of CARRA and professor of pediatrics at the Albert Einstein College of Medicine in the Bronx, N.Y. While PRCSG continues to do industry-sponsored studies, he says, CARRA concentrates on investigator-initiated research.
“These diseases have analogues in adult medicine, but others are distinct and restricted to children,” Dr. Ilowite says. “And the patients are different, with developmental and nutritional factors that we need to account for. Most of the time, a treatment that is safe and effective in adults is also safe and effective in kids, but the dosing, pharmacokinetics, and pharmacodynamics can be quite different. You can’t always extrapolate from adult data very well.” He also explains that because pediatric rheumatologic diseases are relatively rare, pharmaceutical companies have little financial incentive to do definitive studies in children in diseases other than polyarticular juvenile idiopathic arthritis (JIA).
“CARRA was formed to fill a void in investigator-initiated research, which was quite limited 10 years ago. CARRA has a very democratic and transparent philosophy and is very inclusive. We can do the research that we conceive of and can get funded,” Dr. Ilowite adds.
CARRA research currently focuses on six areas: juvenile arthritis, pediatric systemic lupus erythematosus (SLE), juvenile dermatomyositis (JDM), vasculitis, scleroderma, and pain and health-related quality of life. CARRA members have identified specific agendas for each of these areas. A small sampling of these agenda items includes
- Developing trials to determine the best treatment approaches for all JIA types and assessing the long-term safety and efficacy of the immune modulating, suppressive, and biologic agents used to treat JIA;
- Developing studies to understand the assessment and treatment of kidney involvement in pediatric SLE;
- Developing studies that will evaluate treatment approaches in JDM;
- Developing therapeutic interventions for children with chronic rheumatic and nonrheumatic musculoskeletal pain; and
- Identifying the most effective means of studying the course and outcomes of vasculitis and scleroderma in children.
“With CARRA, we are bringing research to more patients,” says Laura Schanberg, MD, professor of pediatrics at Duke University in Durham, N.C., and one of the physicians who has been with CARRA since its earliest days. CARRA has already successfully completed two major clinical trials and many other smaller studies supported by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and the AF. As Dr. Schanberg says, “we haven’t yet reached as many as we’d like, but our long-term impact is really greater than the studies we’ve done to date. We’ve developed the infrastructure to facilitate clinical research and to try to advance an agenda that is not formed by industry alone.”
