Outdated consent rules a barrier to improving children’s health care

The evidence to support much of children’s healthcare is limited.Ten years ago, randomized controlled trials in adults were increasing ten times faster than pediatric ones. Unfortunately, it doesn’t seem like the trend has changed and the gap in evidence between adult and children’s healthcare continues to widen. Randomized controlled trials are the best ‘fair tests’ we have in medicine when the choice between treatments is unclear and help provide clarity whether new treatments are better or worse than routine ones.

When there is uncertainty in the practice of medicine, some argue that it is unethical to not enroll patients in randomized trials. Not doing so is wasteful, and actively maintains uncertainty rather than using such opportunities to improve patient care. Yet, traditional randomized controlled trials are technically difficult, costly, time-consuming, complicated, and often do not apply to average patients outside the study. Trials in children are especially hard because of parental reluctance and small numbers.

What happens when two or more treatments are considered routine? For example, several different steroid regimens are believed to prevent complications in children with asthma attacks, but it is unclear if one works better than the other. Comparative effectiveness research is the term coined for trials that compare such routine treatments. They are essentially large-scale randomized controlled trials that are inserted into the everyday practice of medicine; by doing so, they minimize the barriers faced by traditional trials, and others have proven they are possible.

Comparative effectiveness research is important. The basic assumption that ‘routine’ treatments are harmless may be false. Treatments that have been routinely used for decades are seldom based on robust studies, or held to a similar standard if they were unveiled today. For example, the widespread use of oxygen for babies in the nursery led to serious eye problems including blindness. What treatments used routinely today may have similar harms?

But comparative effectiveness studies challenge the ethical framework that is steadfastly defended by Institutional Review Boards. This is particularly true when studies involve children. These regulatory committees require excruciatingly detailed consent documents, which outline every possible risk and benefit of enrolling in the study. These requirements make it difficult to recruit patients and act as a barrier to get doctors involved in research. While these consents make perfect sense for traditional trials where new therapies with unknown benefits and risks are being tested, they make much less sense when the therapies being tested are already considered the standard of care. The end result: failure to advance medical knowledge around uncertainty and maintaining the minimal evidence base available to guide children’s healthcare.

Here lies the rub. If there are two readily acceptable treatments used in routine practice, doctors decide a particular treatment based on various reasons like personal preference, anecdotal experience, pharmaceutical industry influence, or cost. Yet, if the same doctor wants to formally study the treatment to determine if it does more good than harm, a costly, and burdensome system awaits. Pediatrician Richard Smithells summarized this conundrum: “I need permission to give a drug to half of my patients, but not to give it to them all.”

Several authors have proposed changing the current Institutional Review Boards’ informed consent process to better reflect the actual risk of comparative effectiveness research studies and to reduce the barriers to conducting such research in children.

In certain situations, where risks to patients are minimal, or no greater than usual care, informed consent may not be required at all. Such studies would require ethical review and approval, but not consent for individuals enrolling in the study.

Others have argued for a ‘streamlined’ consent process which aims to better reflect the way actual decisions about treatments are made by doctors and patients. When the choice between two equally acceptable treatments is unclear, a conversation could be had between the doctor and parent describing this uncertainty. Verbal informed consent for study enrolment and randomization would be documented in the patient record the same way we already discuss risks and benefits of initiating these treatments. Such a system would avoid exaggerating the actual risk of the research which creates unnecessary deterrents to comparative effectiveness research participation.

There is a real tension between Institutional Review Boards’ necessary role in protecting the vulnerable on one hand (particularly in research involving children) and the inability to move the evidence base for children’s healthcare forward. But changes to how we conduct research involving children are needed to improve the quality of children’s healthcare.

We suggest that where clinical uncertainty is unresolved, we may be unintentionally causing harm to our young patients. While parents, clinicians and policy makers may find this unacceptable, the current Institutional Review Board framework for informed consent needs to be modified to reflect the actual risks of comparative effectiveness studies for the quality of children’s healthcare to improve.

Peter Gill is a paediatric resident at The Hospital for Sick Children in Toronto, Ontario and an Honorary Fellow at the Centre for Evidence-Based Medicine at the University of Oxford. Follow Peter on Twitter @peterjgill Jonathan Maguire is a paediatrician and Research Scientist at St. Michael’s Hospital in Toronto, Ontario.

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  1. H Khan

    Why is it essential for the clinician to conduct a formal clinical trial? The physician can measure outcomes associated with different therapies and over the long-term use those data to support using one form of treatment over another. This can replace, and in some ways better than, a clinical trial because it uses a random population and is conducted in a ‘real life’ (i.e. uncontrolled) setting. I find many physicians are reluctant to measure patient health outcomes because they view it as a measure of their own performance and thereby will lead to increased accountability, which is inaccurate.

    When it comes to patients, especially children, the consent process should be thorough and the risks associated with a trial treatment should not be left for the parent to understand. Many parents have a hard time understanding the care process, let alone understand risks to their child during a time when their child could be very ill. Suggesting experimental therapies to these vulnerable parents without a regulatory body review to me is unethical. I agree that this may delay the use of some innovative and effective therapies, however, I do not believe we need to neglect the safety of current patients for the benefit of the future ones.

    • Peter Gill

      Thank you for your thoughtful comment on our blog post. To clarify, we are not proposing that studies are conducted without Institutional Review Boards’ approval. This process is important to ensure independent assessment of the study in question, and to ensure research complies with appropriate ethical conduct. We suggest that the level of detail required in informed consent documents to enrol patients in clinical trials is revised to be more consistent with actual clinical practice, particularly for comparative effectiveness research where treatments are already part of the everyday practice of medicine. Parents are not required to read and sign multi-page consent document outlining every possible risk and benefit of routine treatments but they are if researchers want to study these everyday treatments. We hope to see a system that facilitates research in child health to reduce uncertainty and improve the evidence base for children.

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