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Assessing the Comparative Effectiveness of Newly Marketed Medications: Methodological Challenges and Implications for Drug Development
Author(s) -
Schneeweiss S,
Gagne J J,
Glynn R J,
Ruhl M,
Rassen J A
Publication year - 2011
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 - 10.1038/clpt.2011.235
Subject(s) - comparative effectiveness research , clinical pharmacology , psychological intervention , clinical trial , medicine , drug development , alternative medicine , placebo , cohort , drug , intensive care medicine , family medicine , pharmacology , nursing , pathology
Comparative‐effectiveness research (CER) aims to produce actionable evidence regarding the effectiveness and safety of medical products and interventions as they are used outside of controlled research settings. Although CER evidence regarding medications is particularly needed shortly after market approval, key methodological challenges include (i) potential bias due to channeling of patients to the newly marketed medication because of various patient‐, physician‐, and system‐related factors; (ii) rapid changes in the characteristics of the user population during the early phase of marketing; and (iii) lack of timely data and the often small number of users in the first few months of marketing. We propose a mix of approaches to generate comparative‐effectiveness data in the early marketing period, including sequential cohort monitoring with secondary health‐care data and propensity score (PS) balancing, as well as extended follow‐up of phase III and phase IV trials, indirect comparisons of placebo‐controlled trials, and modeling and simulation of virtual trials. Clinical Pharmacology & Therapeutics (2011); 90 6, 777‐790. doi: 10.1038/clpt.2011.235