Treatment Efficacy and Toxicity in Rheumatoid Arthritis Database and Repository

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DESCRIPTION (provided by applicant): Treatment of rheumatoid arthritis (RA) has improved dramatically in the last 10 years. No single drug, however, is effective in every patient, and there is great variability in toxicity and price, ranging from ~$400/year for methotrexate (MTX) to up to $15,000/year for biologic agents. This proposal is based on the premise that the next major advance needed in the treatment of RA is not additional drugs, but rather a dramatic improvement in the efficiency and cost-effectiveness of the use of drugs for individual patients with RA. One of the major obstacles to identifying clinically useful markers of treatment response in RA is the lack of cohorts with prospectively collected treatment response data coupled with biological samples. To date, there has been no attempt to harmonize the efforts of the US academic RA research community to create a public resource. Our proposal will thus fill a critical need: establishing an infrastructure of academic investigators to lay the foundation for future collaborative large-scale registries; and to align and unite the efforts of many organizations with the common goal of improving care of RA patients. By unifying the efforts of academic researchers, we can create resources that would not otherwise be available, such as a bank of cryopreserved blood cells to allow sophisticated immunologic research to dissect molecular signals of successful treatment of RA. Our specific aims are: 1) To create a collaborative network of leading academic investigators committed to collecting treatment response data and biologic samples in RA patients starting MTX or biologic agents; 2) To implement a protocol to facilitate uniform data and specimen collection; 3a) To perform a "proof of concept" study to determine the feasibility of this infrastructure by enrolling a small number of RA patients at 10 sites; 3b) To utilize financial and in-kind contributions from the Arthritis Foundation and corporate partners to perform pilot projects to facilitate future grant funding; 4) To stimulate collaborative efforts of federal funding agencies, voluntary health agencies, professional organizations and industry partners to enable creation of a large, sustainable database and repository to better understand the molecular basis of treatment and rapidly accelerate translational research in RA.