There are also numerous program and operational challenges associated with subject recruitment. “Once you define eligibility criteria for a particular trial, it may turn out that because of prior therapy or disease stage, the number of patients at a given site is too small to power a study. This is especially true with the less common diseases,” Dr. Rotrosen explained. “In such cases, we have to do multisite studies, recruiting patients from around the country, and sometimes we have to go overseas, which introduces regulatory barriers and financial constraints.”
To alleviate some of the challenges, the NIAID provides support for multidisciplinary clinical research networks in order to help facilitate long-term, multisite clinical trials, said Dr. Rotrosen said. In autoimmune disease research, this helps investigators explore the mechanisms of disease progression and get a sense of the impact of therapies over time. “This is especially important with relapsing and remitting diseases. Stable support over time is critical in order to see improvement against this background,” he said. Additionally, the NIAID offers generous support for mechanistic studies and biomarker discovery, “which hopefully will lead to earlier, more accurate diagnoses, and will allow us to stratify subjects based on stage of disease, family history, genetic background, and so forth, so we can ultimately simplify trial designs and make them shorter and less costly.”
Dr. Rose and Dr. Rotrosen did not report conflicts of interest related to their presentations.