The Value of Pharmaceutical Industry-Sponsored Patient Registries in Oncology Clinical Research

In May 2019, the US Food and Drug Administration (FDA) released the Framework for FDA's Real-World Evidence (RWE) Program, a draft guidance to evaluate the potential use of real-world data in facilitating regulatory decisions. As a result, pharmaceutical companies and medical communities see patient registries, which are large, prospective, noninterventional cohort studies, as becoming increasingly important in providing evidence of treatment effectiveness and safety in clinical practice. Patient registries are designed to collect longitudinal clinical data on a broad population to address critical medical questions over time. With their large sample sizes and broad inclusion criteria, patient registries are often used to generate RWE in the general and underrepresented patient populations that are less likely to be studied in controlled clinical trials. Here, we describe the value of industry-sponsored patient registries in oncology/hematology settings to healthcare stakeholders, in drug development, and in fostering scientific collaboration. Patient registries provide real-world evidence of treatment effectiveness and safety in clinical practice. This review discusses the characteristics and use of patient registries in oncology and hematology settings and describes the value of industry-sponsored oncology registries for healthcare stakeholders and for fostering scientific collaboration.

Automated summary

In May 2019, the FDA released guidelines on the potential use of real-world data in regulatory decisions. Patient registries, particularly in oncology/hematology settings, are seen as crucial in providing evidence of treatment effectiveness and safety. These registries collect large-scale clinical data to address important medical questions and are often used to generate real-world evidence.