If the time for precision medicine is now, could the massive databases of health and drug plan claims systems be a starting point?
Among the top ten drugs sold in the United States, patients actually benefit at best around a third of the time, according to a study in the journal Nature. For other top medications, only one in 25 patients, or 4 percent, will be helped.
"Every day, millions of people are taking medications that will not help them," writes Nicholas Schork, a Professor and Director of Human Biology at the J. Craig Venter Institute. For statins, as few as 1 in 50 may benefit, according to Nature.
The problem, Schork argues, could aptly be called "imprecision medicine."
(Source: Nature.)
The current model of drug development and clinical trial is "inefficient at best," Schork argues. "Because scant data are collected on factors such as genetics, lifestyles and diets, the results of these trials often indicate the need for yet another study to validate the effectiveness of the intervention among the apparent responders and to establish the underlying mechanisms."
To achieve the kind of personalized, precision medicine that President Obama wants to pursue with federal research funding, "a crucial part of the mix" will the the N-of-1, studies that focus on one person, according to Schork.
"Physicians have long done these in an ad hoc way. For instance, a doctor may prescribe one drug for hypertension and monitor its effect on a person's blood pressure before trying a different one. But few clinicians or researchers have formalized this approach into well-designed trials--usually just a handful of measurements are taken, and only during treatment."
With big data, that approach can be scaled. "Aggregated results of many N-of-1 trials (all carried out in the same way) will offer information about how to better treat subsets of the population or even the population at large," according to Schork's vision.
"These could save the millions of U.S. dollars that are spent on inappropriate interventions, the management and treatment of persistent or recurring diseases, and on conventional phase III trials (which can cost between $100 million and $700 million per drug)."
Dipping their toes into comparative effectiveness Amid a debate about the cost and value of drugs, a big payer and a big pharma company are combing through their collective evidence.