3652 - Patterns of FDA Approval for Novel Oncology Drugs

Y. Gladkiy¹, and B. D. Facer²; ¹Boonshoft School of Medicine, Wright State University, Dayton, OH, ²Department of Radiation Oncology, The Ohio State University Wexner Medical Center, Columbus, OH

Purpose/Objective(s): Developing new therapies for oncologic indications is resource intensive. The US Food and Drug Administration (FDA) provides several pathways for expedited review and approval so sponsors with promising novel therapies can efficiently evaluate clinical efficacy. Efficacy data is demonstrated in clinical trials, which require significant time and resources. Proper trial design is critical, as a poorly designed trial can contribute to resource waste and prolonged unmet needs for patients with cancer. An improved understanding of current FDA approval patterns can influence trial design and regulatory strategies. The aim of this study is to detail the profile of recently approved novel oncology drugs, including the utilization of expedited FDA pathways and successful clinical trial designs. Materials/

Methods: All FDA approvals for novel drugs were reviewed from 2015 – 2024. Drugs were screened for therapeutic indications in oncology settings. Using the original label and FDA filings, variables were extracted regarding the drug sponsor, FDA review process (e.g., orphan status, accelerated approval, etc.), first approved indication(s), registration trial design, and drug efficacy. No subsequent indication approvals for any drug were included, only the original indication(s) approved by the FDA.
Results: From 2015 – 2024, 112 novel drugs were approved for the treatment of 122 oncologic indications. Almost all drugs (92%) were approved for one indication. Fifty-nine sponsors were represented in this cohort; the most drugs approved for one sponsor was 8. Regarding the FDA review process, 81 drugs (72.3%) had orphan drug status and 65 (58.0%) had priority review status. Accelerated approval (AA) was granted for 60 indications (49.2%) and breakthrough designation was granted for 61 indications (50.0%). The most common targeted malignancies were hematologic (36.1%), thoracic (13.9%) and breast (10.7%) malignancies. Seventy-two approvals (59.0%) were based on studies without a placebo or active comparator arm. Placebo controls were involved in 21 (17.2%) approvals. Data from a phase 3 trial was present in 48 (39.3%) approvals. For indications based on AA, objective response rate was the most common primary endpoint (90.0%); otherwise, the most common primary endpoint was progression-free survival (PFS) (40.3%). Approval was based on overall survival in 10 (8.2%) indications. Approvals granted via the AA process have been confirmed for 21(35.0%) indications and withdrawn in 9 (15.0%).
Conclusion: In this analysis, we highlight patterns of recent FDA approvals for novel cancer therapies. Most approvals are granted to drugs with orphan drug and priority review status. The most common primary endpoint is PFS for traditional and ORR for accelerated approval. These patterns can inform drug developers and clinical trialists who seek FDA approval for novel cancer drugs.