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Assessing patient risk, benefit and outcomes in drug development: an observational study of regorafenib clinical

Brody Dennis1, Chance Bratten1, Griffin K Hughes1

  • 1Oklahoma State University Center for Health Sciences, Tulsa, Oklahoma, USA.

BMJ Oncology
|January 31, 2025
PubMed
Abstract

Insights

Regorafenib trials show increasing patient risk and low objective response rates over time, especially in colorectal cancer. Higher quality research is needed to clarify its true risk/benefit profile.

Area of Science:

  • Oncology
  • Clinical Pharmacology
  • Drug Repurposing

Background:

  • Drug development is costly, prompting pharmaceutical companies to repurpose existing medications for new uses through clinical trials.
  • A comprehensive pooled analysis is essential to evaluate the risk-benefit profile against a drug's efficacy.

Purpose of the Study:

  • To conduct a comprehensive analysis of all oncological clinical trials involving regorafenib.
  • To establish a complete risk-benefit profile for regorafenib in cancer treatment.

Main Methods:

  • Systematic screening of PubMed, Embase, Cochrane (CENTRAL), and ClinicalTrials.gov for regorafenib trials in solid cancers.
  • Extraction of key data including progression-free survival, overall survival, adverse event rates, and objective response rate (ORR).
  • Classification of studies as positive, negative, or indeterminate based on pre-specified endpoints and tolerability.

Main Results:

  • Analysis included 56 trials with 4960 participants across 13 indications; 78.75% were non-blinded and 73.21% were non-randomized.
  • Colorectal cancer trials initially showed positive outcomes but trended negative over time, with increasing patient risk and consistently low ORR.
  • Cumulative patient risk rose over time, while objective response rates remained consistently low across studies.

Conclusions:

  • Regorafenib's risk profile has increased since its initial FDA approval for its primary indication.
  • A significant proportion of non-randomized, single-arm trials were identified, highlighting the need for higher-quality research.
  • Regorafenib's efficacy and safety may be more favorable in cancers beyond its currently approved indications.
Keywords:
Colorectal cancerGastric cancerLiver cancer

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