FDA official discusses use of RWE in cancer research and approvals

Editor's Note: The article has been updated to correct that FDA approved Vijoice (alpelisib) to treat PIK3CA Related Overgrowth Spectrum (PROS) based on RWE and not Piqray for breast cancer.

Use of real-world evidence (RWE) is progressing in the oncology area and was used as the basis for approving two new therapies, both of which fulfilled an unmet medical need, an official with the US Food and Drug Administration (FDA) said at a 28 September meeting convened by Duke-Margolis Center for Health Policy.

Donna Rivera, associate director for pharmacoepidemiology in the Oncology Center for Excellence (OCE), noted that RWE was used in the successful application for accelerated approval of Vijoice (alpelisib) on 5 April 2022. The treatment is indicated for PIK3CA Related Overgrowth Spectrum.

FDA approved Orencia (abatacept) in December 2021 for prophylaxis of acute graft-versus-host disease (GVHD). The treatment is used in combination with other drugs for patients undergoing hematopoietic stem cell transplantation (HSCT) from a matched or mismatched donor. This treatment was approved based on data from the Center for International Blood and Marrow Transplant Research (CIBMTR), which includes data on every allogenic transplant performed and analyzed the demographics of the treatment population,

To get these treatments approved, Rivera stressed that the sponsors had to predefine the essential elements of their studies, use two real-world data (RWD) sources to describe how each element was identified, address issues that are essential for determining the reliability and relevance of the data in the protocols, and confer with FDA before starting to ensure the acceptability of the evidence sources used.

Rivera also highlighted some extramural work to understand RWD and enhance its regulatory review. One example is a grant to the University of Oklahoma to analyze clinical outcomes and genomic data of Native American patients treated with immune checkpoint inhibitors for various cancers.

Rivera also discussed another initiative, ENCORE (Emulation of Interventional Trials in Clinical Oncology with Real World Evidence), which uses electronic health records (EHR) to support regulatory approvals in the oncology area. The project is being led by Harvard and Brigham Women’s Hospital.

Other efforts to increase the uptake of RWD were highlighted at the meeting, including the release of a new white paper by the Duke-Margolis Center that explores how master protocols could apply to RWD/RWE studies. The paper was informed by a 2022 collaborative workstream on RWE, according to Rachele Hendricks-Sturrop of the center.

Her takeaway: Master protocols improve consistency and efficiency in RWD/RWE studies, and the standardization mechanism in these protocols “may reduce variation and produce more reliable and comparable real-world evidence.”

Duke-Margolis Center for Health Policy meeting

FDA official discusses use of RWE in cancer research and approvals

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