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Background <p>The United States Food and Drug Administration’s (USFDA) accelerated approval process enables the rapid introduction of biologics based on surrogate endpoints for unmet medical needs, particularly in cancer treatment. Biologics play a vital role in precision medicine by enhancing the immune system’s ability to fight cancer or targeting malignancies directly. Since its inception in 1992, the program has significantly increased the approval of cancer biologics.</p> Method <p>This study analysed the regulatory status of accelerated biologics approved for cancer over 30 years, based on the USFDA “Drug and Biologic Accelerated Approvals Based on a Surrogate Endpoint” report (as of September 30, 2024). The time to full approval, withdrawal trends, and the submission of Biologics License Application (BLA) and supplements were evaluated.</p> Results <p>Out of the 106 Biologics License Applications (BLAs), 97 (91.50%) were for cancer and 9 (8.49%) for non-oncology indications. Among 37 original BLAs, 22 (59.45%) achieved full approval, 11 (29.72%) were not converted, and 4 (10.81%) were withdrawn. For 60 supplements, 38 (63.33%) gained full approval, 14 (23.33%) were not converted, and 8 (13.33%) were withdrawn. Accelerated approval of BLAs for cancer took an average of 4.44 years to reach full approval, while supplements required 3.10 years. Withdrawals averaged 3.64 years for BLAs and 4.14 years for supplements.</p> Conclusion <p>The study highlights the dominance of BLA supplements over original BLAs, their faster conversion to full approval, and greater time for withdrawal. These findings underscore the significance of supplement applications in enhancing the regulatory and clinical value of previously approved biologics.</p> Graphical Abstract <p></p>

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The Evolution of Turbocharged Cancer Therapeutics - How Regulatory Flexibility has Driven Accelerated Cancer Biologics Approvals in the United States (1993–2023)

  • 期刊论文

  • 发表日期：

    2025年7月26日

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  • Fathima Musthafa,
  • Simran Simran,
  • Vishal Sachin Gangawane,
  • Vaishnavi Milind Kalokhe,
  • Aftab Ahmad,
  • Saurabh Srivastava

摘要

Background

The United States Food and Drug Administration’s (USFDA) accelerated approval process enables the rapid introduction of biologics based on surrogate endpoints for unmet medical needs, particularly in cancer treatment. Biologics play a vital role in precision medicine by enhancing the immune system’s ability to fight cancer or targeting malignancies directly. Since its inception in 1992, the program has significantly increased the approval of cancer biologics.

Method

This study analysed the regulatory status of accelerated biologics approved for cancer over 30 years, based on the USFDA “Drug and Biologic Accelerated Approvals Based on a Surrogate Endpoint” report (as of September 30, 2024). The time to full approval, withdrawal trends, and the submission of Biologics License Application (BLA) and supplements were evaluated.

Results

Out of the 106 Biologics License Applications (BLAs), 97 (91.50%) were for cancer and 9 (8.49%) for non-oncology indications. Among 37 original BLAs, 22 (59.45%) achieved full approval, 11 (29.72%) were not converted, and 4 (10.81%) were withdrawn. For 60 supplements, 38 (63.33%) gained full approval, 14 (23.33%) were not converted, and 8 (13.33%) were withdrawn. Accelerated approval of BLAs for cancer took an average of 4.44 years to reach full approval, while supplements required 3.10 years. Withdrawals averaged 3.64 years for BLAs and 4.14 years for supplements.

Conclusion

The study highlights the dominance of BLA supplements over original BLAs, their faster conversion to full approval, and greater time for withdrawal. These findings underscore the significance of supplement applications in enhancing the regulatory and clinical value of previously approved biologics.

Graphical Abstract