Reporting of Drug Benefit in FDA-Approved Prescription Drug Labeling
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studies (e.g., phase 3 trials), but the peer-reviewed literature describing these studies may be incomplete (e.g., with unpublished trials), and systematic reviews often have yet to be conducted.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;1however, 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.,...
All data we extracted and coded, as well as copies of original drug labels used in our study, are freely available in the Zenodo repository ( https://doi.org/10.5281/zenodo.2562183).
All authors had full access to all of the data in the study and take responsibility of the integrity of the data and the accuracy of the data analysis.
Study concept and design: Desai, Doshi
Acquisition of data: All authors
Analysis and interpretation of data: All authors
Drafting of the manuscript: Desai, Doshi, Powers
Critical revision of the manuscript for important intellectual content: All authors
Statistical analysis: Desai
Administrative, technical, or material support: Doshi
Study supervision: Doshi
This study was funded with nonspecific faculty research funds provided by the University of Maryland, Baltimore, to Dr. Doshi.
Compliance with Ethical Standards
Conflict of Interest
Dr. Doshi has received travel funds from the European Respiratory Society (2012) and Uppsala Monitoring Center (2018) and grants from the Laura and John Arnold Foundation (2017–2021), American Association of Colleges of Pharmacy (2015), Patient-Centered Outcomes Research Institute (2014–2016), Cochrane Methods Innovations Fund (2016–2018), and UK National Institute for Health Research (2011–2014) and is an editor at The BMJ and an unpaid member of the Reagan-Udall Foundation for the FDA. Dr. Powers reports personal fees from Abbvie, Cardeas, Cempra, Contrafect, Gilead, Johnson & Johnson, MedImmune, Novartis, Otsuka, Pfizer, Roche, Romark, and Trius outside the submitted work. Dr. Desai and Dr. Hong have no interests to declare.
- 2.Clinical Studies Section of Labeling for Human Prescription Drug and Biological Products—Content and Format. Rockville, MD: U.S. Food and Drug Administration; 2006:25. https://www.fda.gov/media/72140/download. Accessed 17 Sept 2019.
- 3.Food and Drug Administration. New Drugs at FDA: CDER’s New Molecular Entities and New Therapeutic Biological Products. https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/default.htm. Accessed 17 Sept 2019.
- 4.AHFS Classification - Drug Assignments and Reassignments. http://ahfs.ashp.org/drug-assignments.aspx. Accessed 17 Sept 2019.