The NIH, in pursuit of precision medicine, tries to avoid ghosts of its past

WASHINGTON — The National Institutes of Health is preparing to recruit 1 million volunteers for one of its most significant research projects in years, one meant to develop personalized medical treatments.

As it does so, officials are trying to dodge the ghosts of a similarly ambitious research initiative, one that ended with a whimper: the National Children’s Study.

Launched with great fanfare in 2000, the congressionally mandated children’s study was touted as an effort to understand the impact of children’s environments on their health and development. Fourteen years and $1.3 billion later, the effort was shut down, the victim of internal agency conflicts and problems with the design of the study.

The question now is whether the NIH can avoid going down a similar road with the Precision Medicine Initiative, which is expected to enlist 10 times the number of volunteers as the children’s study sought — but failed — to enroll. Some researchers have already suggested the initiative has been poorly designed and will face some of the same problems.

“I read the request for applications and I also read the advisory report to NIH, and I thought, if anybody in one of my epidemiology classes presented this as a study design, it would get an F,” said Dr. Nigel Paneth, a pediatrician and epidemiologist at Michigan State University who led a research site for the children’s study.

The Precision Medicine Initiative is designed to help researchers learn more about early signs of disease, information that could make it easier to develop better, more individualized cures for patients. It was launched in January 2015 by President Obama, just a few weeks after NIH Director Francis Collins ended the children’s study.

As part of the new initiative, researchers will collect medical records and biological specimens from participants, and well as data from any smartphones or wearable sensor devices, and information about diet, substance abuse, and disease symptoms. The NIH estimates it will take three to four years to secure 1 million volunteers for the study.

With so much at stake, officials have pledged to learn from the children’s study and not repeat the same mistakes.

“Believe me, it has occurred to us that we better not go down that same path,” Collins told STAT.

Read more: An interview with NIH Director Francis Collins

Officials said the children’s study was beset by problems from the start. The initial plan was to enroll 100,000 pregnant women and to track their children from birth to age 21, measuring exposure to chemical pollutants as well as other factors and ascertaining their impact on ADHD, autism, asthma, and other disorders.

But it took far too long to get the study underway — more than 10 years, NIH officials said.

“Science changed drastically during that time, and so the design that had been put together didn’t really fit the scientific opportunities by the time it was ready to get started,” Collins said.

There were other intractable problems, among them disagreements over how best to enlist volunteers and even arguments about how to measure research outcomes.

And then there were money issues. The study had trouble retaining enough experienced epidemiologists, researchers said, because funding for the initiative had to be reauthorized by Congress each year, making the job unstable.

The Precision Medicine Initiative has a fiscal 2016 budget of $200 million, and the NIH is hoping to avoid funding issues problems, in part because lawmakers have expressed such eagerness for the development of “faster cures.”

But Paneth, the epidemiologist at Michigan State University, said the project still might not be sufficiently funded.

“If you do the math, there’s only a few hundred dollars for each person enrolled, and that will have to cover the lab tests, getting informed consent and other expenses,” he said. “It is likely that all that will be covered is clinical data already collected. There’s just enough money to transfer the data.’’

Paneth also questioned the validity of the design, saying that recruiting people from so many different research centers, including those who “self-select” will not yield valuable information.

NIH Deputy Director Kathy Hudson, who planned and created the Precision Medicine Initiative, insisted the failures of the children’s study have not been forgotten. She was a member of the NIH advisory committee that recommended it be discontinued.

“We tried to define the seven deadly sins of the children’s study,” Hudson said, “and we, in fact, spent some time in our working group discussing what exactly we needed to know about that undertaking and what went wrong in order to learn from that in building this program.”

Among the lessons: the importance of consistent leadership. The children’s study went through numerous directors, officials said, creating confusion and frustrating advisory panels created to straighten it out. It also tried to do too much.

That, too, is a problem researchers with the Precision Medicine Initiative hope to avoid.

The children’s study “tried to be too many things to too many people, and we learned lessons about focus and making sure that we define a few questions that can definitely be answered,” said Dr. Russ Altman, professor at Stanford Medical School and the co-chair of the children’s study working group.

Collins agreed that the Precision Medicine Initiative must be focused — and to have leadership that is “fully empowered to make decisions without a lot of second-guessing.”

“We aim to keep it rapidly moving, to attach itself to today’s science, and to get that implemented quickly,” he said.