TY - JOUR AU - Miller, Kathleen L. AU - Mueller, Christine AU - Liu, Gumei AU - Miller Needleman, Katherine I. AU - Maynard, Janet PY - 2020 DA - 2020/09/03 TI - FDA orphan products clinical trial grants: assessment of outcomes and impact on rare disease product development JO - Orphanet Journal of Rare Diseases SP - 234 VL - 15 IS - 1 AB - The Office of Orphan Products Development (OOPD) of the United States (U.S.) Food and Drug Administration (FDA) has awarded over 700 grants to conduct clinical trials of medicals products for rare diseases since 1983, leading to over 70 marketing approvals. However, despite recent progress in rare disease product development, thousands of rare diseases still have no approved treatments. An assessment of this clinical trial grants program was undertaken to provide an in-depth analysis of the characteristics and outcomes of the program. Results of this analysis will be used to inform future goals of the program, as well as internal data collection to continue to maximize the program’s impact in supporting rare disease product development. SN - 1750-1172 UR - https://doi.org/10.1186/s13023-020-01514-5 DO - 10.1186/s13023-020-01514-5 ID - Miller2020 ER -