October 31, 2015
An academically trained skeptic, this single project has turned me into a raving optimist.
Imagine you are a big drug company, testing out a new drug. You pick a group of people who you think are most likely to respond to the drug. If you give the new drug to ten cancer patients and nine of them have progressive disease — their tumors continue to grow — then you’re probably going to shelve that drug. You wouldn’t possibly want to give a drug to a group of patients when 90 percent of them will continue to get worse. But what about that one patient?
While the other nine progressed, one patient improved remarkably. You’re probably still going to have to shelve the drug. A 90 percent failure rate is unacceptable. And yet 10 percent of the patient population might be missing out on a phenomenal treatment for their specific tumor.
That one patient is the extraordinary responder — her response was different than the expected, ordinary response based on statistics. If researchers could figure out what made her extraordinary — why she responded to the drug and the others didn’t — perhaps they could tailor a trial to patients with only that “extraordinary” characteristic. By better selecting a target patient population, they could take a drug with a 90 percent failure rate and turn it into a drug with an exceptional success rate!