A Benefit-Risk Analysis Approach to Capture Regulatory Decision-Making: Multiple Myeloma.

ABSTRACT: Drug regulators around the world make decisions about drug approvability based on qualitative benefit-risk analysis. In this work, a quantitative benefit-risk analysis approach captures regulatory decision-making about new drugs to treat multiple myeloma (MM). MM assessments have been based on endpoints such as time to progression (TTP), progression-free survival (PFS), and objective response rate (ORR) which are different than benefit-risk analysis based on overall survival (OS). Twenty-three FDA decisions on MM drugs submitted to FDA between 2003 and 2016 were identified and analyzed. The benefits and risks were quantified relative to comparators (typically the control arm of the clinical trial) to estimate whether the median benefit-risk was positive or negative. A sensitivity analysis

SUBMITTER: Raju GK 

PROVIDER: S-EPMC7418461 | biostudies-literature | 2018 Jan

REPOSITORIES: biostudies-literature