Small is Big.
Bob Temple said he "would like to see more" comparative effectiveness trials for symptomatic treatments to help identify potential responders. In particular, Temple said trials that randomize non-responders to a drug to receive both that drug and an alternative drug have "a lot of potential" but are almost never done.
"For example, you could take people who don't respond to a migraine drug and randomize them back to a new migraine drug, the old migraine drug the one they didn't respond to, and a placebo. If it's true that there are individual differences that are important, then the new drug ought to be able to show its advantage over the drug that didn't work," he wrote in a post on FDA's From a Clinical Perspective blog.
When asked whether institutional review boards (IRBs) would approve such trials, Temple said there is no ethical issue in an informed patient. He added that such trials would not be done for indications where "not working would be dangerous."
This seems to jive with what Dr. Joe Selby (PCORI’s executive director) told me the other day – that he believes PCORI should focus on funding “narrow” clinical trials aimed at smaller, more targeted populations, that PCORI's mission is to ensure that CER helps to advance the cause of personalized medicine and “the right treatment for the right patient at the right time.” Bravo.
If Joe Selby can be the Good Witch of Comparative Effectiveness Research, so much the better. I think he’d look lovely in ruby slippers.
As would Bob Temple.
