How Can Precision Medicine Change Our Approach To Clinical Trials?

October 28, 2016

By Angela Woodall, Healthcare and Environmental Reporter.

It was Jan. 20, 2015 and about 30 minutes into Barack Obama’s State of the Union Address when the president announced a program that had been two decades in the making and whose scope could exceed any that have come before.

Wedged in between remarks about trade and net neutrality, President Obama told listeners that he was green-lighting the launch of the Precision Medicine Initiative, which would begin where the groundbreaking Human Genome Project had left off.

“I want the country that eliminated polio and mapped the human genome to lead a new era of medicine — one that delivers the right treatment at the right time,” Obama said.

“So tonight, I’m launching a new Precision Medicine Initiative to bring us closer to curing diseases like cancer and diabetes, and to give all of us access to the personalized information we need to keep ourselves and our families healthier.  We can do this.”

More importantly Obama also called for $215 million in fiscal year 2016 to support the initiative, which supporters say promises to dramatically transform the way healthcare is delivered and received.

The heart of the study is a close examination of the individual variations in our genes, our lifestyles and our environment — the nurture and nature side of our health but on a much more granular level.

“It’s a causal story that tells us about ourselves,” said James Tabery, a professor at the University of Utah who studies the ethical, social and legal sides of science.  “And clinical trials are what generate that information.”

Supporters are hoping a new approach to medicine will be accompanied by a new approach to clinical trials.

Conventional trials are strictly controlled and gather select measurements from thousands of people. They ignore the factors like income, lifestyle, genetics and diet, wrote Nicholas Schork, director of human biology at the J. Craig Venter Institute in La Jolla, Calif., in a 2015 issue of Nature.

“Precision medicine requires different ways of testing interventions,” Schork said. “Researchers need to probe the myriad factors — genetic and environmental, among others — that shape a person’s response to a particular treatment.”

In fact, one of the first near-term goals listed on the PMI website is the “innovative clinical trials of targeted drugs for adult, pediatric cancers.” That will begin with the PMI for Oncology led by the National Cancer Institute.

The NCI will continue what it calls a first-of-its-kind precision medicine trial: NCI-MATCH. In this clinical trial, adult patients are assigned to targeted treatments based on the genetic abnormalities in their tumors, regardless of the type of cancer they have. The PMI will also make it possible to accelerate planning for the NCI Pediatric MATCH trial, which is expected to open in late 2016.

Eventually the plan is to expand the PMI cohort to include 1 million Americans, an unprecedented number requiring an unprecedented level of coordination among healthcare professionals, as well as cooperation from the public. It’s their data that will be collected. But first they have to be recruited, sign consents, be examined, queried about everything from their grandmother’s health to what they ate that morning, and convinced to give samples of urine, blood and saliva.

Teams of researchers then have to work together to understand the fine details provided by a million individuals, who could reach beyond airplane altitudes if they made a human ladder.

It won’t be easy, of course. “There will be a lot of learning going on,” David Delaney,  the chief medical officer of SAP, said. “It’s going to force us to deal with a lot of these mechanics as we bring all of these issues together.”

Of course, this is where contract research organizations are at home. The payoff is being on the front lines of redesigning clinical trials.

Angela Woodall, Healthcare and Environmental Reporter.

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