Life Years Gained From the FDA Accelerated Approval Program in Oncology: A Portfolio Model
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Background: Although the FDA Accelerated Approval Program (AAP) has come under scrutiny, the population-level health benefit of the program has not been quantified. Therefore, the objective of this study was to estimate the number of life years gained among patients with cancer that can be attributable to the therapies receiving FDA accelerated approvals in oncology between 2006 and 2022 in the United States. Methods: The data sources used were FDA listings, FDA approval letters and labels, published clinical trial data and other publications including relative effectiveness estimates, and the Ipsos Oncology Uptake Tool for product uptake. Data for 130 oncology treatments approved by the FDA under the AAP were extracted and validated. We developed a decision analytic model to estimate the survival gain for each indication and to accumulate life years gained for consecutive cohorts of patients receiving the therapies. Life year gains were estimated with and without the AAP, and the incremental life years gained were attributed to the program. Results: The analysis estimated that through December 2022 in the United States, the program gained approximately 263,000 life years across 69 products for which overall survival data were available, for approximately 911,000 patients with cancer. Conclusions: Policy discussions about the evaluation of AAP cannot be complete without assessing its impact on its most important target outcome: patient survival. To date, there has been no estimation of the life year gain delivered by the AAP. Our research shows that substantial number of life years were gained for patients with high unmet need by the cancer therapies approved through the program.
Submitted July 28, 2023; final revision received December 11, 2023; accepted for publication January 16, 2024. Published online April 22, 2024.
Author contributions: Conceptualization: All authors. Data curation: Benedict, Szabó, Marczell, Doherty. Formal analysis: Benedict, Szabó, Marczell. Methodology: All authors. Writing—original draft: All authors. Writing—review & editing: All authors.
Disclosures: Á. Benedict, G. Szabó, and K. Marczell have disclosed being employed by Evidera, which received research support from Janssen Scientific Affairs/Johnson & Johnson Innovative Medicine. B. Doherty and S. Martin have disclosed being employed by Janssen Scientific Affairs; and owning stock in Johnson & Johnson.
Funding: Research reported in this publication was partially funded by Janssen Scientific Affairs.
Supplementary material: Supplementary material associated with this article is available online at https://doi.org/10.6004/jnccn.2024.7010. The supplementary material has been supplied by the author(s) and appears in its originally submitted form. It has not been edited or vetted by JNCCN. All contents and opinions are solely those of the author. Any comments or questions related to the supplementary materials should be directed to the corresponding author.
Supplementary Materials
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