The Within Our Reach campaign has been tremendously successful, and one of the benefits has been the development of a diverse donor base. The campaign has received tremendous support from the physicians, patients, and our industry partners, with nearly $28 million raised to date. Over $9.7 million has been raised from nonindustry sources, including the initial $5-million commitment from the ACR, which provided the early campaign momentum needed for success, and $3.3 million from lay donors who had not previously been a focus of the REF. Initial discussions about expanding this targeted research initiative are ongoing, and the REF will likely expand this program to continue funding critical RA research and possibly expand into other inflammatory arthritis research areas.
Novel Clinical Trials
Despite improvements in therapeutic options, RA remains a serious and life-threatening disease. About 30% to 50% of patients continue to have active disease and how best to manage these treatment-resistant patients remains a challenge. Basic research efforts have identified a plethora of molecules that could be targeted.
RA clinical trial design came of age in the mid 1990s when the Food and Drug Administration (FDA), with help from many of our members, published a guidance document on conducting RA randomized clinical trials. This has served our community well, resulting in the approval of several new biologic therapies during the last 15 years. Nevertheless, conducting trials in 2010 is different than it was in 1995, when few therapeutic interventions were available other than methotrexate. There is now significant interest in improving the clinical trial design for drug development to enhance patient recruitment and reduce patient exposure to placebo. There are a number of questions in RA that need to be addressed regarding effective intervention. The movement to evaluate the “comparative effectiveness” of therapeutics will stimulate a new generation of clinical trials. Investigator-initiated trials, including those traditionally sponsored by the NIH, often address key questions related to mechanisms of action, personalized medicine including pharmacogenomics, active comparator trials, or patient-centered outcomes with an emphasis on quality-of-life measures, and can be complemented with translational research into genetic, gene expression, and metabolic biomarkers.
It has been clear to most in the RA clinical research arena that it is time to reevaluate this process to enhance our ability to conduct trials and most efficiently address the numerous clinical questions that have arisen over the last decade. Last month, the ACR held a strategy meeting in Washington, D.C., with key stakeholders to discuss how we can move the RA clinical trials field forward. The goal of this meeting was to identify unmet needs and priorities for future investigator-initiated and industry-sponsored clinical trials in RA. Attendees included investigators from across the country, as well as representatives from the various NIH institutes, the FDA, AF, and REF. This meeting hopefully will serve as the catalyst to stimulate innovative trial design and influence the NIH agenda.