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As detailed in , each of the partners has a unique role. For example, academic centers provide expertise in the design and conduct of clinical trials, and industry brings expertise in medicinal chemistry, preclinical pharmacologic models, toxicology, and regulatory science. The CFF supports more than 120 accredited care centers in the United States and a patient registry that longitudinally tracks demographic, clinical, and genotype data on more than 20,000 patients. In addition, in 1999 the TDP launched the Cystic Fibrosis Therapeutics Development Network (CF-TDN) that focuses on assisting industry partners through the therapeutic development process. Several strengths of the TDN and its partner, the European Cystic Fibrosis Society Clinical Trial Network, have been the establishment of uniform outcome measures and standard care practices, a common international protocol-review process, and continuous assessment of study-site metrics in order to improve the safety and efficiency of study conduct ().

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In the early 1980s, treatment strategies for type 1 diabetes entered a new era that was catalyzed by the understanding that this disease was an autoimmune disorder resulting in selective destruction of insulin-secreting pancreatic islet beta cells. Several clinical trials of immune intervention have shown partial interruption of disease progression. Such findings have served as proof of concept for therapy directed at the underlying autoimmunity, even though these beneficial effects have been transient and the search for new treatments continues. Although the autoimmunity in type 1 diabetes often evolves over months or years preceding overt hyperglycemia, the clinical diagnosis of the disease corresponds to a critical period of beta-cell loss, which constitutes a window of opportunity for therapeutic intervention. However, most pharmaceutical companies have chosen not to prioritize immune therapy to prevent or to halt islet-cell damage in early type 1 diabetes in their clinical-trial portfolios, since the prevalence of the condition is only 0.4% in the United States and Europe and current insulin-replacement therapy is effective at restoring partial metabolic control. To fill this gap, an efficient translational ecosystem has been established that encompasses public advocacy, academic science, federal initiatives, and disease-related foundations, a collaboration that has enabled the conduct of numerous trials of new therapeutic agents that are designed to interrupt disease progression by blocking autoimmunity.

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Every year in the United States, approximately 25,000 patients receive a diagnosis of multiple myeloma, with 60,000 to 70,000 prevalent cases at any given time. The relatively low number of cases (as compared with diseases such as lung, colon, prostate, and breast cancer) limits the attraction for industry and the National Cancer Institute to initiate research projects. In addition, the pathway for drug approval has been less clear because of a lack of consistent treatment approaches and, more recently, a plethora of new treatment options, which has led many investigators and pharmaceutical companies to avoid translational research in multiple myeloma in favor of more common approval approaches. The opportunity to bring together academia with industry, the FDA, patients, and researchers from other disciplines to help solve problems in multiple myeloma was readily apparent ().