Universities and drug companies that use human volunteers for research face tough new rules designed to make sure that valuable information from these volunteers is widely available, not only to the volunteers themselves but to scientists trying to advance medical science.
The rules currently on the books are confusing and often ignored.
Since 2007, scientists have been required to post results of experiments on a government website, ClinicalTrials.gov. But many top universities and drug companies have failed to meet those standards, according to academic studies and investigative journalists.
"We as a community have had a disappointing track record," said Francis Collins, director of the National Institutes of Health. Collins and other officials are now hoping that will change with clearer, more expansive rules and potentially stinging new penalties put forth by the Department of Health and Human Services.
The new rules, published Friday in the Federal Register, are designed to make it easier for scientists, universities and corporations to understand what experiments must be included in the federal database. It also expands the list of studies that must be registered, to include experimental behavioral interventions — such as a study that compares different diets among diabetics — as well as early-stage (Phase 1) drug trials if they have NIH funding.
For the first time, these rules also require companies to post results of trials for drugs that don't make it to market, not just the ones that do. "This has been a very opaque world up until to now," Food and Drug Commissioner Robert Califf told reporters during an embargoed phone conference Thursday. "These are tremendous changes."
Researchers must also say in advance how they plan to analyze their results. That is designed to discourage researchers from changing the focus of a study after the fact and dredging through their data to highlight findings that may simply be coincidental, and which might not be robust enough to appear in a study that was specifically designed to look for them.
Clinical trials "are the gold standard for understanding the effect and effectiveness of medical interventions, understanding what works and what doesn't work," Collins said in the press conference.
If information from them is widely shared, it can help scientists identify potentially serious side effects, and it can help scientists avoid wasting federal tax dollars by conducting redundant studies. Right now, scientists often don't report their failures, so other researchers may try the same useless strategy.
Reporting results is also an ethical issue. Millions of patients volunteer to participate in studies, Collins said. "Sharing the results from clinical trials helps fulfill society's responsibility to those individuals who volunteered in these studies with an understanding that their participation will contribute in advancing medical knowledge."
For studies that are regulated by the FDA, companies can face fines of $10,000 a day if they don't comply with the existing rules. Those fines have not been imposed, however, and the FDA will not have additional funding to hire enforcement staff, Califf said. "I really believe that it won't take much to get people to comply with this once they realize how serious this is."
Collins said the NIH will cajole university scientists to comply as well. If they don't, the NIH would consider withholding funds on planned clinical trials at the offending university until scientists there are following the new rules. This would not apply to research that started before the rules take effect, and the NIH would not stop clinical trials that are already under way.
The new rules come into effect in January 2017. Researchers will have 90 days to comply.
KELLY MCEVERS, HOST:
Millions of Americans volunteer to be human guinea pigs. They offer to test new drugs, devices, even new diets, in the name of advancing medicine. And researchers who run medical trials are supposed to be clear about what they're doing, and they're supposed to share their results. They don't always do these things. So today, the federal government announced new rules to address this problem. NPR's Richard Harris reports.
RICHARD HARRIS, BYLINE: The rules involve human studies known as clinical trials, which are typically tests of drugs or medical devices on volunteers.
FRANCIS COLLINS: Clinical trials are how we find out what works and what doesn't work and make advances in medicine.
HARRIS: Francis Collins heads the National Institutes of Health, which, in addition to industry, funds many of these studies. In 2007, the government established rules requiring that, in many instances, researchers need to disclose the existence, design and results of these experiments. But last year, Dr. Monique Anderson and colleagues at the Duke University Medical School reviewed clinical trials registered on a government website, and they were startled to see how few experiments reported their results on time.
DR. MONIQUE ANDERSON: Legal reporting was really low. It was 13.4 percent overall for the mandatory 12 months.
HARRIS: More scientists reported results late. But still, many didn't comply with the law. Anderson and her colleagues struggled to figure out which studies fell under the rule to begin with.
ANDERSON: It's actually really hard.
HARRIS: She's glimpsed at the new rules and thinks they will be much easier to interpret. They will also cover more areas of research. For the first time, companies will have to report drug and device research even if their products don't end up on the market. Also, NIH-funded researchers will need to register studies that involve behavioral research, for example, studies that compare one diet with another for disease prevention. Again, Francis Collins.
COLLINS: You randomize people to one dietary intervention or another, and then you look to see what worked, we'd want to know the results of that as soon as possible. Right now, it wasn't clear that those were included. We have made it very clear today that they are.
HARRIS: One common problem is that scientists don't report their results if the drug or other intervention doesn't work out. But Dr. Collins says that information can be just as important. And if other scientists don't know something has been tried and failed, they might get a grant and try to find out for themselves.
COLLINS: We are very stressed for resources. And the idea that we might end up supporting a trial for something where it's already been tested and found not to be successful - that's a waste of resources.
HARRIS: When these studies are done as part of the FDA approval process, companies that violate the disclosure rules can face fines of $10,000 a day. But the FDA has not levied those fines and doesn't have the budget to expand its enforcement. Even so, FDA Commissioner Rob Califf is optimistic.
ROBERT CALIFF: Yeah, I have to say I really believe that it won't take much to get people to comply with this once they realize how serious this is.
HARRIS: And Collins says the NIH will up its game by warning a university that it could lose funding for future clinical trials if it's not complying with the new rules.
COLLINS: Imagine if you get that note and you're the president of Harvard, it's probably going to get your attention.
HARRIS: The rules don't look back at the many studies that are still out of compliance, but they will apply to every covered new clinical trial starting next January. Richard Harris, NPR News. Transcript provided by NPR, Copyright NPR.