Stories

The struggle to evaluate drugs

Illustration of a tree shedding pills instead of leaves.
Illustration: Aïda Amer/Axios

The Institute for Clinical and Economic Review and the pharmaceutical industry don’t even agree on what should be considered relevant evidence when deciding if a drug is safe and effective.

Why it matters: "This debate on what constitutes high-quality, 'real-world' evidence is not going away," said Walid Gellad, a pharmaceutical expert at the University of Pittsburgh.

Driving the news: Pharmaceutical companies that were singled out in ICER’s report yesterday on "unjustified" price hikes sent along hundreds of studies for ICER to consider in its analysis. ICER rejected almost all of them.

  • Many of the drug company studies were observational and funded by the companies, and ICER made it clear from the outset that it would only consider observational studies "that were high quality and comparative."

What we're watching: The FDA wants to use more simple trials and observational data in drug evaluations, similar to what drug companies submitted to ICER. 

  • But as Joseph Ross, a professor of medicine at Yale, wrote several years ago, it’s important to assess "the methodological rigor of observational studies before interpreting real-world effects."

Our thought bubble, via Bob: ICER's report is transparent, telling readers that none of the funding for its report came from any part of the industry and outlining clearly what it considered to be acceptable research.

Go deeper: Concerns rise about generic drugs from over