Assessing Patient Risk, Benefit, and Outcomes in Drug Development: A Decade of Sorafenib Clinical Trials

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Abstract

Background: Pharmaceutical companies often investigate previously approved cancer drugs against other cancer indications. This application of drugs to new indications may pose excessive risk to trial participants receiving treatment with non-indicated pharmaceuticals. This study investigates sorafenib's risk-benefit profile in clinical trials published in the past decade, including over 32 non-FDA approved indications.

Objective: To assess sorafenib's drug portfolio by reviewing clinical trials evaluating its efficacy over the last decade.

Methods: On May 25, 2023, we conducted a literature search of bibliographic databases to retrieve clinical trials regarding sorafenib's efficacy as cancer treatment. Included studies were adult clinical trials published in the last 10 years using response criteria. Inclusion screening and data extraction occurred in a masked, duplicate fashion. From this dataset, risk-benefit profiles were evaluated by extracting trial characteristics, overall response rate, adverse event rates, median progression-free survival, and median overall survival in months. The primary endpoint for each trial was categorized based on whether it was met (positive), not met (negative), or indeterminate.

Results: After sorafenib's most recent FDA approval for differentiated thyroid carcinoma in 2013, clinical trials have further explored its efficacy in more than 32 off-label indications. Cumulative trends over time demonstrated consistently low objective response rate and increased toxicity for off-label indications. Of a total of 12,226 evaluable participants for adverse event grading across all 154 clinical trials, 11,003 grade 3-5 adverse events were reported.

Conclusion: With few positive trial endpoints and low objective response rates for sorafenib monotherapy in non-FDA approved indications, trialists should review the risk-benefit profile before using sorafenib monotherapy in the reviewed off-label indications. We also recommend a more transparent practice in reporting outcomes in oncology drug trials due to the findings of the current study.
Original languageAmerican English
StatePublished - 21 Jul 2023
Event7th Annual Joint Research Meeting: Biomedical, Biological, Neuroscience, Physiology, Forensics - Tandy Conference Center, Tulsa, United States
Duration: 21 Jul 202321 Jul 2023

Conference

Conference7th Annual Joint Research Meeting: Biomedical, Biological, Neuroscience, Physiology, Forensics
Abbreviated title7th Joint Annual Research Meeting
Country/TerritoryUnited States
CityTulsa
Period21/07/2321/07/23

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