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A Benefit–Risk Analysis Approach to Capture Regulatory Decision‐Making: Multiple Myeloma
Author(s) -
Raju G.K.,
Gurumurthi Karthik,
Domike Reuben,
Kazandjian Dickran,
Landgren Ola,
Blumenthal Gideon M.,
Farrell Ann,
Pazdur Richard,
Woodcock Janet
Publication year - 2018
Publication title -
clinical pharmacology and therapeutics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.941
H-Index - 188
eISSN - 1532-6535
pISSN - 0009-9236
DOI - 10.1002/cpt.871
Subject(s) - multiple myeloma , medicine , decision analysis , risk assessment , drug , clinical trial , risk analysis (engineering) , oncology , ixazomib , intensive care medicine , pharmacology , computer science , statistics , bortezomib , mathematics , computer security , carfilzomib
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 was demonstrated using ixazomib to explore the magnitude of uncertainty. FDA approval decision outcomes were consistent and logical using this benefit–risk framework.