The new rules are scheduled to take effect Thursday. They will have a big impact on how research is conducted, especially in fields like psychology and neuroscience. NIH distributes more than $32 billion each year, making it the largest public funder of biomedical and health research in the world, and the rules apply to any NIH-supported work that studies human subjects and is evaluating the effects of interventions on health or behavior.
In the biggest change, many studies that investigators previously considered basic research will now be considered clinical trials. That means those studies will be subject to the same stringent rules and reporting requirements demanded of traditional clinical trials, such as those that test the efficacy and dangers of a new drug or medical procedure.
“A lot of research that most of us have never thought of as being clinical trials is going to suddenly to fall under that rubric,” said Ellen Wright Clayton, a bioethicist and law professor. For years she has conducted research on how testing patients’ DNA for warning signs of future health problems would influence patients' or their doctors' behavior. “It has to do with health, but I’ve never thought of it as a clinical trial.”
The goal of the new rules, NIH officials said, is to increase transparency and accountability for its publicly funded research. NIH officials said reporting requirements in the past were so lax that sometimes they couldn’t even readily answer how many clinical trials they were funding in a given year.
“We needed a systemic way to keep track of the research we’re funding,” said NIH acting chief of staff Carrie Wolinetz. “Especially because this is human-related research, we have an ethical responsibility to people.”
The policies aim to solve a problem science has struggled with for decades. Scientists call it the “file drawer” problem, in which many results are never published or released publicly from studies that didn’t yield interesting findings. Instead, those results just get filed away somewhere.
“It’s a shocking waste of resources,” said Evan Mayo-Wilson, an expert on clinical trials at Johns Hopkins Bloomberg School of Public Health. “Knowing the answer to a question keeps us from asking it over and over again. So when results aren’t shared, all the funding that came from taxpayers is wasted.”
Beyond the financial cost, Mayo-Wilson points out, is the ethical responsibility to the human subjects who volunteer their time and in some cases their bodies and potentially risk harm to participate in the research.
“Interventions to human health is among the most important research out there, so it’s important that we know the results of those studies,” said Mayo-Wilson, who supports NIH’s changes but says they may require further tweaking.
In 2000, the U.S. government and NIH created a website, clinicaltrials.gov, that was designed to, among other things, capture more study results. Since then, all clinical trials funded by NIH have been required to be registered on the website and all the results logged in there. Not all researchers fulfill this requirement — another shortcoming NIH’s new rules are designed to address.
Many studies that weren’t previously considered clinical trials now have to register and log their results on the website.
Not all research covered by the rules, however, fits neatly into NIH format and requirements for clinical trials, argues Jeremy Wolfe, a cognitive scientist at Harvard University, who helped organized several letter campaigns last year to bring scientists’ concerns to NIH.
Wolfe, for example, has studied how people sort through chaotic piles of things to find a specific object — like sifting through a Lego bin to find a specific block. The research has implications for understanding how radiologists searching an X-ray for one specific disease could completely miss signs of an equally threatening disease. “Typically this is not the kind of work you think of as a clinical trial,” Wolfe said. “But because this involves humans it may fall under these rules.”
Responding to the surprise and anxiety among many researchers, NIH released a series of updated case studies to help researchers figure out whether their research would now qualify as a clinical trial. One example offered, that many researchers would have previously thought as basic behavioral science, is a study looking into what factors could help a sedentary person to become more active. That research would now be considered a clinical trial.
In recent weeks, Wolfe said NIH has worked with researchers like him to modify their rules and address their main concerns — like finding ways for behavioral researchers to perform initial research or pilot studies without having all such work fall under the onerous full-scale clinical trial requirements.
“We didn’t solve everything, but it’s become less a disaster and more of a nuisance,” Wolfe said. “The end-of-our-scientific-world scenarios and concerns have been taken care of, and that’s real progress.”
Some scientists gearing up for the changes still have complaints about the policies. Some worry a mistake in following them could kill their grant applications. Others note that while the administrative red tape and work will increase, the money NIH pays out for clinical trials doesn’t look like it will increase, forcing researchers and their institutions to absorb the cost of the new requirements.
“Most researchers understand why they're doing this. It's a reasonable request to say we need the results of publicly funded research,” Wolfe said. “But to squeeze every kind of research into this model of clinical trials is like trying to put on a set of clothes that just don't fit.”