Understanding when real world data can be used to replicate a clinical trial: A cross-sectional study of medications approved in 2011

Pharmacoepidemiol Drug Saf. 2020 Oct;29(10):1273-1278. doi: 10.1002/pds.5086. Epub 2020 Aug 14.


Purpose: To determine how commonly pre-approval clinical trials could potentially be replicated using real-world data from insurance claims databases.

Methods: We conducted a cross-sectional study of medications approved by the FDA in 2011. For each medication, we reviewed the drug's label and the details of the pivotal clinical trials supporting its approval. We assessed whether each clinical trial could be replicated using an insurance claims databases by determining whether the following pivotal trial features could be reliably captured in claims data: study outcome, inclusion criteria, exclusion criteria, and the presence of an appropriate active comparator.

Results: In 2011, 28 new medications were approved. The most common disease areas were oncology (N = 8, 29%), infectious disease (N = 5, 18%), and neurology (N = 4, 14%). The primary outcome of pre-approval clinical trials was identifiable in claims databases for six (21%) of the medications. Two (ticagrelor and linagliptin) had at least 80% of inclusion and exclusion criteria that could be identified in claims databases and had an available active comparator. The non-identifiable primary outcomes were related to patient-reported symptoms (N = 9, 32%), imaging findings (N = 5, 18%), laboratory values (N = 5, 18%), or other measurements (eg, blood pressure) not typically available in insurance claims databases (N = 4, 14%).

Conclusions: Among drugs FDA-approved in 2011, two (7%) had a pre-approval trial that could be replicated using insurance claims databases. In such qualifying trials, replication using claims databases could be useful in assessing whether they provide concordant results.

Keywords: epidemiology; pharmacoepidemiology; real-world.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Aged, 80 and over
  • Child
  • Clinical Trials as Topic / methods*
  • Cross-Sectional Studies
  • Data Collection / methods
  • Databases, Factual / statistics & numerical data*
  • Drug Approval*
  • Female
  • Humans
  • Insurance, Health
  • Male
  • Middle Aged
  • United States
  • United States Food and Drug Administration
  • Young Adult

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