Reporting of Drug Benefit in FDA-Approved Prescription Drug Labeling
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Department of Pharmaceutical Health Services Research , University of Maryland School of Pharmacy , Baltimore , MD , USA; 2George Washington University School of Medicine , Washington , DC , USA.
J Gen Intern Med DOI: 10.1007/s11606-019-05460-2 © Society of General Internal Medicine 2019
INTRODUCTION
Obtaining an accurate understanding of the known benefits and risks of drugs is difficult, particularly when drugs first come to market. The most robust evidence is largely limited to pre-marketing studies (e.g., phase 3 trials), but the peer-reviewed literature describing these studies may be incomplete (e.g., with unpublished trials), and systematic reviews often have yet to be conducted. Despite their reputation as unhelpful, FDA-approved prescribing information (also known as “package inserts” or “drug labels”) should provide clinicians with a trustworthy source of information about new drugs that has been independently vetted. Previous research has assessed the quality of clinical trial evidence supporting recent FDA-approved therapeutic agents;1 however, how this information is reported to prescribers through drug labels has not been evaluated. Per guidance, labels are supposed to include key efficacy information including the magnitude of treatment benefit, i.e., “the effect that can be attributed to the drug” (measured as the difference between observed effects in new drug and control groups, presented as absolute and/or relative differences), and measures of precision and random error (confidence intervals and p values).2 We sought to assess the degree to which labels actually provided this information.
METHODS
Original drug labels for all new molecular entities (NMEs) approved by the FDA 3 between January 2015 and March 2018 were downloaded from the Drugs@FDA online database. We excluded NMEs not treating or preventing diseases or conditions (e.g., diagnostic products) and not tested in controlled trials (e.g., single-arm
studies). Text, tables, and figures in each label’s “Clinical Studies” section were independently evaluated by two authors, and disagreements were resolved through consensus with an additional author. For each indication, we extracted the primary efficacy outcome (either explicitly stated or implicitly defined) and recorded the presence or absence of the following attributes describing treatment effects for primary outcomes: point estimates, confidence intervals, and p values. Primary efficacy outcomes were categorized as either a surrogate endpoint or direct measure of patient benefit. We classified presentation of treatment effects as absolute and relative differences and whether results for new drug and control groups were presented separately. Drug therapeutic class was based on the American Hospital Formulary Service classification.4
RESULTS
One hundred thirty indications (from 119 NMEs) were approved between January 2015 and March 2018. One hundred two indications met inclusion criteria. Forty-five percent (46/ 102) of primary outcomes were surrogate endpoints, not direct measu
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