NIAMS is also sponsoring two placebo-phased studies to address unmet needs in patients with rare diseases. The unique designs used seek to minimize patient exposure to placebo because all patients receive the intervention while maintaining the statistical rigor of a well-controlled, randomized clinical trial. RIM is an international, multicenter clinical trial examining the effectiveness of rituximab for treating myositis in adults and children. The RAPPORT trial is evaluating Rilonacept, an IL-1 blocker/trap, in the systemic form of JIA. More information about these trials and other NIAMS-supported efforts is available through the NIH’s ClinicalTrials.gov Web site (www.clinicaltrials.gov).
Basic research is the backbone of NIH’s mission to acquire new knowledge to help prevent, detect, diagnose, and treat disease and disability.
The Osteoarthritis Initiative
Osteoarthritis (OA) is the most common form of arthritis, affecting an estimated 27 million adults in the United States. Today, 35 million people—13% of the U.S. population—are 65 or older. By 2030, 20% of Americans (approximately 70 million people) will have passed their 65th birthday and will be at risk for OA. One of the barriers to the development of drugs that block joint degradation, the underlying cause of painful and disabling OA symptoms, is the lack of objective and measurable standards for disease progression by which new drugs can be evaluated. To overcome this problem, the NIH—with input from the U.S. Food and Drug Administration—has partnered with private sponsors to create the Osteoarthritis Initiative (OAI) that is delivering a publicly available research resource that investigators can use to identify and evaluate OA biomarkers. In this case, the biomarker would be a physical sign, biological substance, or imaging finding that could be used in clinical studies to monitor changes in joint health.
The OAI is currently collecting OA patient data, radiological information, and biospecimens and will make these data widely available so that investigators around the world can utilize the information. The study, which uses a prospective, natural history cohort, tracks nearly 5,000 subjects over time. It aims to increase understanding and ultimately improve treatment of a major cause of disability among Americans. So far, the initiative has released baseline clinical data on all participants, as well as 12- and 18-month follow-up data and images on many. When the study is complete, the information will document the natural progression of the disease and help identify biomarkers associated with it, ideally hastening research and therapies. All OAI data and images are available to researchers worldwide through the initiative’s Web site (www.oai.ucsf.edu/datarelease), along with tools to interpret them.
New Standards for Patient-Reported Outcomes
The NIAMS continues to help lead the paradigm-shifting NIH Roadmap initiative. The Patient-Reported Outcomes Measurement Information System (PROMIS) seeks to advance the science of patient-reported outcomes in clinical research and care by applying state-of-the-art item response theory (IRT) approaches. Calibration of items and questions by IRT methods improves the precision, lessens the patient burden to gather this information, and allows comparisons of these clinically important outcomes not possible to date. Long term, PROMIS offers unique opportunities to learn more about how interventions, biologic or behavioral, affect individual patients either from an efficacy or adverse event perspective. Much more is available about PROMIS, including a demonstration of computerized-adaptive testing, through its Web site (www.nihpromis.org).
