… specifically, the Black Knight from Monty Python, apparently reduced to nibbling impotently at the feet of pharmaceutical corporations as they sail through the approval process.
This study in JAMA reviews the characteristics of novel therapeutics approved by the FDA between 2005 and 2012. These authors identified 188 novel agents approved for 206 indications, and describe an entire host of fascinating data regarding the trials supporting approval. A few of the most damning pearls:
- 37% of indications were approved on the basis of a single trial.
- The median number of patients per trial was 760.
- 49% of trials used only surrogate outcomes.
- Surrogate outcome trials constitute sole basis of approval for 91 indications.
- Only 48% of cancer trials were randomized, and only 27% were double-blinded.
- 40 trials were part of accelerated approval, 39 of which used surrogate outcomes, with a median number of patients of 157.
The data go on and on.
Considering many landmark trials could not be independently reproduced, even with the help of the original researchers; most published research findings are false; and half of what you know is wrong – we might as well just dump poison in our water supply. It’s cheaper than suffering the next blockbuster drug for which pharmaceutical companies engineer an indication through distorted trial design.
“Clinical Trial Evidence Supporting FDA Approval of Novel Therapeutic Agents, 2005-2012“
http://jama.jamanetwork.com/article.aspx?articleid=1817794 (open access)
“Author Insights: Quality of Evidence Supporting FDA Approval Varies”
http://newsatjama.jama.com/2014/01/21/author-insights-quality-of-evidence-supporting-fda-approval-varies/