Using a Benefit-Risk Analysis Approach to Capture Regulatory Decision Making: Renal Cell Carcinoma

Clin Pharmacol Ther. 2020 Mar;107(3):495-506. doi: 10.1002/cpt.1589. Epub 2019 Sep 27.


Drug regulators such as the US Food and Drug Administration (FDA) make decisions about drug approvals based on benefit-risk analysis. In this work, a quantitative benefit-risk analysis approach captures regulatory decision making about new drugs to treat renal cell carcinoma (RCC). Fifteen FDA decisions on RCC drugs based on clinical trials whose results were published from 2005 to 2018 were identified and analyzed. The benefits and risks of the new drug in each clinical trial were quantified relative to comparators (typically the control arm of the same clinical trial) to estimate whether the benefit-risk was positive or negative. A sensitivity analysis was demonstrated using pazopanib to explore the magnitude of uncertainty. FDA approval decision outcomes for the clinical trials assessed were consistent and logical using this benefit-risk framework.

Publication types

  • Research Support, U.S. Gov't, P.H.S.
  • Review

MeSH terms

  • Antineoplastic Agents / administration & dosage
  • Antineoplastic Agents / adverse effects
  • Carcinoma, Renal Cell / drug therapy*
  • Decision Making
  • Drug Approval / legislation & jurisprudence*
  • Humans
  • Indazoles
  • Kidney Neoplasms / drug therapy*
  • Pyrimidines / administration & dosage
  • Pyrimidines / adverse effects
  • Risk Assessment
  • Sulfonamides / administration & dosage
  • Sulfonamides / adverse effects
  • United States
  • United States Food and Drug Administration


  • Antineoplastic Agents
  • Indazoles
  • Pyrimidines
  • Sulfonamides
  • pazopanib