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Accelerated approval (AA) is a US Food and Drug Administration (FDA) expedited program intended to speed the approval of drugs and biologics that may demonstrate a meaningful advantage over available therapies for diseases that are serious or life-threatening.
This review describes all malignant hematology and oncology AAs from inception of the program on December 11, 1992, to May 31, 2017. During this period, the FDA granted AA to 64 malignant hematology and oncology products for 93 new indications. Of these AAs, 53 were for new molecular entities. Overall, the end point of response rate, including hematologic response rates, accounted for most AAs (81 [87%]), followed by time-to-event end points of progression-free survival or time to progression (8 [9%]) and disease-free survival or recurrence-free survival (4 [4%]). Single-arm trial designs provided the data for 67 (72%) of the initial AA indications. Of the 93 AAs, 51 (55%) have fulfilled their postmarketing requirement and verified benefit in a median of 3.4 years after their initial AA. Thirty-seven (40%) indications have not yet completed confirmatory trial(s) or verified benefit, and 5 indications receiving AA (5%) have been withdrawn from the market.
Conclusions and Relevance
The use of the AA program during the past 25 years has increased over time, and only a small portion of indications under the AA program fail to verify clinical benefit. For patients with serious or life-threatening oncologic diseases, AA brings products to the market years before confirmatory trials are typically completed.
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Accepted for Publication: November 28, 2017.
Corresponding Author: Julia A. Beaver, MD, Office of Hematology and Oncology Products, US Food and Drug Administration, 10903 New Hampshire Ave, Building 22, Room 2100, Silver Spring, MD 20993 (firstname.lastname@example.org).
Published Online: March 1, 2018. doi:10.1001/jamaoncol.2017.5618
Author Contributions: Drs Beaver, Howie, and Pelosof contributed equally to this work. Drs Beaver and Kluetz had full access to all the data in the study and take responsibility for the integrity of the data and the accuracy of the data analysis.
Study concept and design: Beaver, Kim, Blumenthal, Pazdur, Kluetz.
Acquisition, analysis, or interpretation of data: Beaver, Howie, Pelosof, Kim, Liu, Goldberg, Sridhara, Blumenthal, Farrell, Keegan, Kluetz.
Drafting of the manuscript: Beaver, Howie, Pelosof, Liu, Blumenthal, Farrell, Pazdur, Kluetz.
Critical revision of the manuscript for important intellectual content: Beaver, Howie, Pelosof, Kim, Goldberg, Sridhara, Blumenthal, Farrell, Keegan, Kluetz.
Statistical analysis: Howie, Liu.
Administrative, technical, or material support: Beaver, Howie, Kim, Goldberg, Sridhara, Keegan, Pazdur.
Study supervision: Beaver, Kim, Blumenthal, Farrell, Pazdur, Kluetz.
Conflict of Interest Disclosures: The authors conducted all work on the manuscript during their work at the US Food and Drug Administration. No additional funds were used for this activity.
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