Did newborn babies participating in two recent clinical studies face health risks that their parent's weren't informed about? Experts debate.
Credit: Newborn baby photo via Shutterstock
WASHINGTON — Two families that participated in a government-funded study of oxygen levels in premature infants say they felt they were not fully informed of the health risks their babies faced in participating in the study.
One mother, Sharissa Cook, whose son was less than 2 lbs. when he was born, said she did not realize she had entered her newborn in a medical study. The name of the study — SUPPORT, which stands for Surfactant Positive Pressure and Oxygenation Randomized Trial and was conducted between 2004 and 2009 — led her to believe that it would provide emotional support to her and her family, she said, speaking here at meeting on Wednesday.
Other parents, Shawn and Carrie Pratt, said that although they knowingly entered their premature daughter, Dagen, in the SUPPORT study, they thought the researchers would only be collecting data about their daughter's condition. Only later did they realize the study was attempting to find an optimal level of oxygen to give to premature babies, and that Dagen had been randomly assigned to receive one of two treatments (either a "low" or "high" oxygen level). Dagen required surgery early in life for an eye disease, and was diagnosed with cerebral palsy at age two. These two conditions are linked with prematurity, and it is impossible to know whether enrollment in the SUPPORT study increased the risk of either condition. [7 Medical Myths Even Doctors Believe]
Both families spoke during a meeting of the Office for Human Research and Protections, which was spurred by criticism of the SUPPORT study. Earlier this year, the OHRP said it found faults with the SUPPORT study consent forms, although no action has been taken as a result of the office's findings.
At the meeting, experts debated the question of exactly how doctors should go about informing patients of the potential harms of participating in medical studies, particularly if that research involves treatments that are already widely used.
Some argued that consent forms need to be more explicit about the possible risks of such studies, including how risks for participants might differ from risks faced during usual patient care. But other experts argued that doctors already do a significant amount of guessing when they care for patients every day, and highlighting the risks of a given medical study may give patients a skewed perception of how risky the study really is.
Although some of the discussion focused on what to include in a consent form, the stories of these two families highlight that consenting to take part in medical research goes far beyond words on a form.
"The consent form is only part of the process of consent," said Dr. Jerry Menikoff, director of OHRP. Although it will be a challenge, experts are working toward improving the process so that people who enter studies have an adequate understanding of what the study involves, Menikoff said.
SUPPORT study criticisms
In the SUPPORT study, more than 1,000 babies were randomly assigned to receive either a "high" or "low" oxygen level, but babies in both arms of the study received oxygen levels that were within the range of those acceptable by doctors today.
One criticism of the SUPPORT study was that it did not make clear that the risk of side effects — which included blindness and death — might differ depending on which group the babies are assigned to, according to Public Citizen, a consumer advocacy group.
Instead, the consent forms state the following (or something very similar): "because all of the treatments proposed in this study are standard of care, there is no predictable increase in risk for your baby."
Lois Shepherd, a professor of biomedical ethics at the University of Virginia Health System, said at the meeting that it is important for researchers to be transparent about the reasons they are doing a study, what they know right now and what they hope to find out. Researchers should explain that the purpose of the trial is to find out if there's a difference between treatments, Shepherd said.
"If the intervention affects the health of the individual in a certain way, then any difference in that is going to be important for the individual to know about," Shepherd said.
In the SUPPORT study, the consent forms stated that high oxygen levels are known to increase the risk of blindness, but did not mention that low oxygen levels might increase the risk of death, which was ultimately a finding of the study. Researchers involved in the SUPPORT study said that, at the study's start, there was no way of knowing that oxygen levels within the range already used today could increase the risk of death. Public Citizen says that studies done in the past suggested that low oxygen levels might increase the risk of death.
Many experts who spoke at the meeting agreed that a similar study conducted in New Zealand, called BOOST, did a better job than the U.S. study of explaining the purpose of the oxygen research. The New Zealand consent forms explained that the purpose of the study was to see if oxygen levels on the lower extreme of what is used in care today could reduce the risk of blindness without increasing the risk of death and disability.
Drawbacks of "standard of care"
Others who spoke at the meeting stressed that studies of the standard of care — the care doctors already use today — are important because, in many situations, doctors don't know which method of care is the best. They use their best judgment, but this may be based on their experience or preference, rather than hard evidence.
In situations like these, "Your doctor is guessing. When you let doctors guess, they make more mistakes. They need data, we need data, to drive our decision-making," Dr. Jeffrey Drazen, editor-in-chief of The New England Journal of Medicine, told LiveScience in an interview before the meeting.
What's more, when doctors recommend a treatment, they don't always discuss all the risks of that treatment, or mention alternatives, said Nancy Kass, a bioethics professor at the John Hopkins Bloomberg School of Public Health. Thus, patients who enter a research trial, in which all the risks are thoroughly laid out, may think that the trial is risker than their regular care, Kass said. But in fact, some studies suggest that patients enrolled in clinical trials do better overall than patients who aren’t. [10 Medical Myths that Just Won't Go Away]
Researchers should state the risks of a given study, but "We also want to say, had you not been in this study, you would have gotten one [treatment] or the other, without really knowing which one is better," Kass said.
Menikoff said the agency will consider the statements made during Wednesday's meeting as it develops more rigorous guidance for the informed consent process.