Study: FDA approved five drugs to treat rare diseases using RWE in 2020-2022

Between fiscal years 2020 and 2022, the US Food and Drug Administration (FDA) approved five drug and biologics for which real-world evidence (RWE) played a role in demonstrating effectiveness. All of these approvals were to treat rare diseases and had orphan drug designation, according to a new study published in the journal Clinical Pharmacology & Therapeutics.

The article was co-authored by Gabriel Innes, a health science policy analyst at FDA’s Office of Medical Policy within the Center for Drug Evaluation and Research, and other current and former FDA employees.

The evaluation examined whether the primary focus of the study was on effectiveness or safety. It considered real-world data sources, such as medical claims, medical records, and registries. Additionally, it assessed the study design, including whether the trial was randomized, externally controlled, or non-interventional. The evaluation also looked at "regulatory intent," determining whether the application was for a new indication, population, or dosing regimen. The survey found there were no studies conducted solely to provide the safety data required for approval.

At least two authors reviewed the studies, and if there were disagreements, a third author was brought in for further review.

The study revealed that between FY 2020 and 2022, CDER approved five drug and biological product applications where RWE “contributed to substantial evidence of effectiveness.” There were no such approvals in FY 2020, while there were two approvals in FY 2021 and three in FY 2022.

The applications were reviewed by different offices, including the Office of Oncologic Diseases, the Office of Cardiology, Hematology, Endocrinology, and Nephrology, the Office of Immunology and Inflammation, and the Office of Rare Diseases, Pediatrics, Urology, and Reproductive Medicine.

The approved drugs included Nulibry (fosdenopterin) and Prograf (tacrolimus), both of which were approved in FY 2021; as well as Orencia (abatacept), Voxzogo (vosoritide), and Vijoice (alpelisib) approved in FY 2022.
Nulibry was approved to reduce the risk of mortality in patients with molybdenum cofactor deficiency, while Prograf was indicated as a prophylaxis of organ rejection in adult and pediatric patients receiving allogeneic liver, kidney, heart, or lung transplants.

Voxzogo was approved to increase linear growth in pediatric patients with achondroplasia with open epiphyses; Orencia was approved as a prophylaxis for treating acute graft versus host disease (aGVHD), in combination with a calcineurin inhibitor and methotrexate in adults and pediatric patients undergoing hematopoietic stem cell transplantation (HSCT); and Vijoice was approved for adults and pediatric patients with severe manifestations of PIK3CA-Related Overgrowth Spectrum (PROS).

The five approvals referenced a total of six studies classified as RWE. Among these, three were non-interventional cohort studies, two were externally controlled trials, and one study included a treatment arm that combined real-world data (RWD) with clinical trial data, which was then compared to an external control arm derived from RWD.

Four of the studies utilized RWD from registries, while two relied on RWD from medical records.

The authors noted that the goal of the report was to describe applications to CDER containing real-world data to generate RWE in pivotal studies that resulted in regulatory approvals. This followed the reauthorization of the Prescription Drug User Fee Act (PDUFA VII), which committed FDA to publish annual reports describing drug and biologic submissions containing RWD to generate RWE.

Clinical Pharmacology & Therapeutics

Study: FDA approved five drugs to treat rare diseases using RWE in 2020-2022

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