Reporting of Drug Benefit in FDA-Approved Prescription Drug Labeling

Abstract

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 o n t h e A m e r i c a n H o s p i t a l F o r m u l a r y S e r v i c e classification. 4

Consensus on Language for Advance Informed Consent in Health Care–Associated Pneumonia Clinical Trials Using a Delphi Process

“…Others have documented a lack of varied understandings of noninferiority trials, including the trade-offs. 16 We also found that whether and how to explain a noninferiority trial in consent text eludes easy consensus. Ultimately, this confusion and concern might reflect that the noninferiority design is not an intuitive statistical or clinical concept, whereas superiority designs are likely more readily understandable or more familiar.…”

“…While there were concerns by stakeholders that patients might not understand the noninferiority concept, some investigators, study coordinators, and IRB members also had their own misconceptions. Others have documented a lack of varied understandings of noninferiority trials, including the trade-offs . We also found that whether and how to explain a noninferiority trial in consent text eludes easy consensus.…”

Did the FDA break its own rules in approving the antibiotic Recarbrio?

“…Even the drug’s 17 page prescribing information for healthcare providers had little to say about the randomised trial results. In the section of the labelling where such efficacy data are typically presented,6 Recarbrio’s label offered the same information about the cUTI and cIAI studies, saying, “These trials provided only limited efficacy and safety information.”7…”