Real-world Data for Clinical Evidence Generation in Oncology

TLDR

Conventional cancer trials are slow, costly, and limited in external validity, but recent technological and policy advances enable real‑world data (RWD) from electronic health records, claims, registries, and digital solutions to improve evidence generation, with RWD defined by point‑of‑care intent and offering efficient experimental designs that balance internal and external validity, potentially expanding patient participation through pragmatic clinical trials. The study aims to show that prospective collection of RWD can enable evidence generation through pragmatic clinical trials that support randomized designs and broaden research to the point of care. RWD can be generated using experimental designs akin to conventional trials when data collection is research‑oriented, offering efficient execution and balanced validity, while valid real‑world studies require auditable data abstraction and incentives for electronic capture of clinically relevant data at the point of care. RWD supports active pharmacovigilance, provides insights into disease natural history, and enables development of external control arms.

Abstract

Conventional cancer clinical trials can be slow and costly, often produce results with limited external validity, and are difficult for patients to participate in. Recent technological advances and a dynamic policy landscape in the United States have created a fertile ground for the use of real-world data (RWD) to improve current methods of clinical evidence generation. Sources of RWD include electronic health records, insurance claims, patient registries, and digital health solutions outside of conventional clinical trials. A definition focused on the original intent of data collected at the point of care can distinguish RWD from conventional clinical trial data. When the intent of data collection at the point of care is research, RWD can be generated using experimental designs similar to those employed in conventional clinical trials, but with several advantages that include gains in efficient execution of studies with an appropriate balance between internal and external validity. RWD can support active pharmacovigilance, insights into the natural history of disease, and the development of external control arms. Prospective collection of RWD can enable evidence generation based on pragmatic clinical trials (PCTs) that support randomized study designs and expand clinical research to the point of care. PCTs may help address the growing demands for access to experimental therapies while increasing patient participation in cancer clinical trials. Conducting valid real-world studies requires data quality assurance through auditable data abstraction methods and new incentives to drive electronic capture of clinically relevant data at the point of care.