The use of mechanistic evidence in drug approval

June 11, 2018 Jon Williamson

This is the title of a new paper, out now in the Journal of Evaluation in Clinical Practice, written by Jeffrey K. Aronson, Adam La Caze, Michael P. Kelly, Veli‐Pekka Parkkinen and Jon Williamson.

Here’s the abstract:

“The role of mechanistic evidence tends to be under‐appreciated in current evidence‐based medicine (EBM), which focusses on clinical studies, tending to restrict attention to randomized controlled studies (RCTs) when they are available. The EBM+ programme seeks to redress this imbalance, by suggesting methods for evaluating mechanistic studies alongside clinical studies. Drug approval is a problematic case for the view that mechanistic evidence should be taken into account, because RCTs are almost always available. Nevertheless, we argue that mechanistic evidence is central to all the key tasks in the drug approval process: in drug discovery and development; assessing pharmaceutical quality; devising dosage regimens; assessing efficacy, harms, external validity, and cost‐effectiveness; evaluating adherence; and extending product licences. We recommend that, when preparing for meetings in which any aspect of drug approval is to be discussed, mechanistic evidence should be systematically analysed and presented to the committee members alongside analyses of clinical studies.”

The paper is available open access here.

Jon Williamson
Centre for Reasoning, University of Kent