Posted by Michael Wonder on 18 Apr 2018
Post-marketing modifications of drug labels for cancer drugs approved by the US FDA between 2006 and 2016 with and without supporting randomized controlled trials
11 April 2018 - Modifications in cancer drug indications, dosing, and related toxicities after FDA approval are common. It is unclear whether drug approval without a supporting randomised controlled trial influences the probability of such modifications.
Shepshelovich et al. searched the Drugs@FDA Web site for new drug indications for solid tumors approved between January 2006 and December 2016. Study characteristics, regulatory pathways, and label modifications from approval to October 2017 were collected from drug labels. Label modifications were considered to be major if defined as such in the drug label. Indications approved with and without supporting RCTs were compared using logistic regression. The Benjamini-Hochberg false discovery rate method was used to adjust for multiplicity.
They identified 59 individual drugs for 109 solid tumour indications. Of these, 17 indications (15.6%) were not supported by an RCT, with no change over time. Indications not supported by RCTs were more likely to require companion diagnostic tests (odds ratio [OR], 3.90; P = .02), to include surrogate end points as primary outcomes (OR, 7.88; P < .001), and to receive breakthrough therapy designation (OR, 7.62; P = .006) or accelerated approval (OR, 17.67; P < .001).
