A new Gene Therapy Data Report is now available, offering researchers and the public timely insights into patients receiving gene therapies within healthcare settings across the PCORnet® infrastructure. As gene therapy development and approval rapidly advance, closing evidence gaps has become increasingly important to help patients make more informed healthcare decisions. This report provides a focused look at the rich, real-world patient data available through PCORnet® Clinical Research Networks—data that could potentially drive future comparative clinical effectiveness research on the long-term outcomes of approved gene therapies.
The Gene Therapy Data Report demonstrates that the PCORnet® infrastructure has the capacity to support national-scale studies on gene therapy. The report describes the characteristics of patient populations who received gene therapy for different health conditions between January 1, 2016 and June 30, 2024, including distinct cohorts for cancer patients and noncancer patients. Data from 72 sites participating in PCORnet are included in this report, reflecting the experience of more than 52,000 patients. These data are standardized from healthcare settings using the PCORnet® Common Data Model, meaning they are ready for researcher analysis.
Data reports are also available for other areas of research including maternal morbidity and mortality, telehealth, intellectual and developmental disabilities, mental and behavioral health, and pediatric population data. Most offer the largest national query of real-world data on health conditions, populations, or service utilization. Read them all.
The PCORnet infrastructure offers access to a broad range of patient populations and standardized data, streamlining the research process while maintaining rigorous privacy protections. Interested in conducting national-scale research in one of the areas highlighted in the data reports? PCORnet may be used by all interested investigators, regardless of affiliation or source of funding. Contact the PCORnet® Front Door to start the conversation.