A Benefit-Risk Analysis Approach to Capture Regulatory Decision-Making: Multiple Myeloma
| Autor: | Karthik Gurumurthi, Reuben Domike, Ann T. Farrell, Gideon M. Blumenthal, G. K. Raju, Dickran Kazandjian, Janet Woodcock, Richard Pazdur, Ola Landgren |
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| Rok vydání: | 2017 |
| Předmět: |
Drug
Oncology medicine.medical_specialty Comparative Effectiveness Research media_common.quotation_subject Comparative effectiveness research Decision Making Antineoplastic Agents computer.software_genre Risk Assessment Disease-Free Survival Article Ixazomib 03 medical and health sciences chemistry.chemical_compound 0302 clinical medicine Internal medicine medicine Humans Pharmacology (medical) 030212 general & internal medicine Adverse effect Drug Approval Multiple myeloma media_common Pharmacology business.industry Surrogate endpoint medicine.disease Clinical trial chemistry 030220 oncology & carcinogenesis Drug and Narcotic Control Data mining business Risk assessment Multiple Myeloma computer |
| Zdroj: | Clin Pharmacol Ther |
| ISSN: | 1532-6535 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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