FDA officials explain how to ensure registries generate fit-for-purpose data

The US Food and Drug Administration (FDA) considers several factors when evaluating whether registry data are fit for purpose for regulatory decision-making, according to Pallavi Mishra-Kalyani, deputy director of the Division of Biometrics at the Center for Drug Evaluation and Research (CDER). These factors include whether the data are relevant and reliable.

Mishra-Kalyani shared this information during a workshop focused on the use of registries in oncology drug development, which touched on efforts to bolster the use of real-world data (RWD) in regulatory decision making, especially for diseases with a significant unmet medical need.

Elizabeth Duke, of the Office of Oncologic Diseases (OOD) in the Center for Drug Evaluation and Research (CDER), kicked off the discussion by noting that the meeting “is not a forum to set policy. It is really an educational forum, we are here to learn from each other and hopefully come away with a clearer view of how oncology registry data can best be collected, validated and evaluated. We all share a common goal of having safer and effective therapies for patients with cancer, and high-quality data is at the heart of that goal.”

Donna Rivera, former assistant director at the Oncology Center of Excellence (OCE), said that registries are just one potential source of RWD. Other sources include electronic health records (EHRs), administrative claims, and patient-generated health data. FDA defines a registry as an organized system that collects clinical and other data in a standardized format, refined by a particular disease or conditions.

Mishra-Kalyani provided an example of how the agency approved Bristol Myers Squibb’s Orencia (abatacept) for treating acute graft-versus-host disease (aGVHD) in combination with a calcineurin inhibitor (CNI) and methotrexate. This approval was granted for both adult and pediatric patients and was based, in part, on registry data.

The registry utilized information from the Center for International Blood and Marrow Transplant Research (CIBMTR), which the FDA deemed fit for use because CIBMTR collects data on every allogeneic transplant conducted in the United States. Each year, the registry collects information on 23,000 new transplants and encompasses data from over 175,000 patients. The registry is managed by the Medical College of Wisconsin under a contract with the Department of Health and Human Services (HHS).

Mishra-Kalyani said that “it is a complete registry and a pretty representative of the US population regarding these transplants.”

She also mentioned that the study utilized comprehensive patient-level demographic information, which included details about patients' socioeconomic status, gender, age, and race and ethnicity. Additionally, the clinical information was thorough, encompassing factors such as disease sub-classification or histology, the stage at transplant, and molecular markers.

“Looking at the questions on using RWD, was the trial design that was used to generate the RWD able to provide adequate scientific evidence to help answer the regulatory questions? In this case we felt yes,” Mishra-Kalyani said.

She added that “the quality of the RWD study using the CIBMTR was clear, and the study was carefully conducted and documented. I think there was a lot of comfort in relying on this evidence for decision-making. Did the study meet the FDA requirements? The answer is yes.”

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FDA officials explain how to ensure registries generate fit-for-purpose data

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