Summary: The FDA and the NIH National Center for Advancing Translational Sciences (NCATS)/Office of Rare Diseases Research (ORDR) conducted this needs assessment to better understand unmet medical device needs for rare diseases; generate meaningful data to inform patients, practitioners, policymakers, and device developers on the needs, barriers, and incentives related to medical device development for rare diseases; and increase public awareness of these needs. The assessment included a subfocus on pediatric rare disease patients. This report (also available as report only, without the appendices and executive summary) describes the results of that assessment, which offers key findings about device needs in adult and pediatric rare disease populations.
The survey results clearly document that patients with rare diseases face numerous unmet needs regarding diagnostic and therapeutic devices for the pediatric and adult population. This survey demonstrates the need for innovative medical devices to care for children and adults with rare diseases. FDA and NIH continue to support and seek opportunities to accelerate device development for rare disease patients. The figure above illustrates some of overall findings from the survey.
A companion manuscript Children and Adults with Rare Diseases Need Innovative Medical Devices was published in the Journal of Medical Devices, Vol 12, Issue 3 as an expert review. It summarized key findings of the needs assessment project, focusing on only physician respondents (inclusive of surgeons) with direct experience or knowledge regarding rare diseases. The manuscript contains statistical analysis that evaluated differences between physician groups in response to questions regarding the need for new or improved devices and satisfaction with current diagnostic and therapeutic devices. It also analyzes relationships between dissatisfaction with devices and physicians' characteristics and perceptions (e.g., impediment factors). Additionally, the manuscript discusses impediments as options for the lack of device development for rare diseases.