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

Abstract

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.

Multi-Criteria Decision Analysis for Benefit-Risk Analysis by National Regulatory Authorities

“…The FDA uses a structured benefit-risk framework based on an analysis of the evidence and uncertainties for four dimensions: analysis of the condition, current treatment options, benefit, risk and risk management ( 31 – 33 ). The framework will be supported by a guidance document on benefit-risk assessment for new drug and biological products, which is currently undergoing consultation ( 18 , 34 ).…”

Headache Reporting in Oncology Trials Depends on the Demographics of the Study Population

“…The overall benefit of oncology medicines can be seen as the net after side effects have been subtracted from efficacy. During oncology drug development, the therapeutic index in preclinical experiments ( 1) is translated into a formal benefit risk evaluation in clinical trials (2). Both compare the findings of the drug with those without the drug.…”