Are research and medicine catered to only a certain type of people?

Are research and medicine catered to only a certain type of people?
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Ideally, medical research should benefit everyone in society. Unfortunately this has not historically been the case. Populations such as children, women, and minorities have been consistently excluded from research, often due to unconscious or implicit bias or difficulty in adjusting research design to be more inclusive.

The 2018 government funding bill increased the budget for medical research at the National Institutes of Health (NIH) by $3 billion to a total of $37 billion.

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The NIH is the largest public funding of biomedical research in the world and its mission is to enhance health, lengthen life, and reduce illness and disability. One way the NIH accomplishes this goal is by funding cutting-edge research in order to “acquire new knowledge to help prevent, detect, diagnose, and treat disease and disability, from the rarest genetic disorder to the common cold.”

 

What we are not discussing enough is that bias persists with regards to who has the opportunity to participate in and ultimately benefit from research.  

As pediatric subspecialists at a tertiary care hospital where we care for the sickest children, inclusion of infants and children in research becomes particularly important if the goal is to provide evidence-based innovative care.    

The NIH recognized this problem in the 1990s and released guidelines to increase the representation of women and minorities as subjects in clinical research. The NIH also mandates a requirement that children must be included, or a justification must be provided for exclusion.  

In addition, the NIH defines vulnerable populations as pregnant women, human fetus, and neonates, prisoners, children, person at risk for suicidality, and persons with impaired decision making. Investigators must put in extra protections when conducting research in these populations.   

To be sure, there is a historical framework for this requirement. Research atrocities conducted during World War II by Nazi Germany led to the creation of the Nuremberg Code in 1949 that codified the conditions under which research must be conducted. Specifically, research participants must give informed consent and participate voluntarily, there must be a favorable risk-to-benefit profile, and they must have a right to withdraw without repercussions.

Despite this, multiple studies were conducted in vulnerable populations during the last century that violated these ethical rules. The most famous is the Tuskegee study that was conducted without proper informed consent when black men were told they were being treated for “bad blood” and ultimately denied proper treatment for syphilis. In the 1950s, thalidomide continued to be used in pregnant women with leprosy despite knowledge that it caused malformation of limbs in children. And finally, the Willowbrook study intentionally exposed mentally disabled children to hepatitis A in an attempt to track the development of the viral infection.  

The response to these atrocities was the creation of the Belmont Report which summarized the ethical principles that must be adhered to when conducting research that involves human subjects — respect for persons, beneficence, and justice.   

So, what we have today is a tension between protecting our vulnerable populations and advocating for fair participation so everyone can benefit from NIH research and funding.  

In reality, when we care for our pediatric patients, we use therapies that are not grounded in empirical evidence. From the drugs we use to the technology we employ, much is extrapolated from adult data and has never been tested in children. We rely on anecdotal evidence and case reports and rarely have randomized, double-blinded, controlled trials as a basis for management of disease. We are, in fact, experimenting daily in our medical practice.

Certainly there has been movement over the last 30 years to have women be more fully represented in clinical trials and biomedical research. Similarly, in 2000, the Children’s Health Act established the Pediatric Research Initiative to “conduct and support research that is directly related to diseases, disorders, and other conditions in children." 

But, despite the best of intentions, participation of infants and children in research still lags behind and remains woefully underfunded by NIH dollars. The estimated total NIH funded research grants and projects specifically for pediatric research is estimated to be $3.8 billion in 2018, approximately 10 percent of the total budget.  

We acknowledge that children are vulnerable, pediatric study design can be more complicated, and informed consent can be ethically challenging.   

But, let’s also remember that unequal representation in research entrenched in bias leads to disparities in health care. Our children deserve more. 

Angira Patel, MD, MPH, is assistant professor of pediatrics and medical education and a member of the Center for Bioethics and Medical Humanities at Northwestern University Feinberg School of Medicine, and a pediatric cardiologist at Ann & Robert H. Lurie Children’s Hospital of Chicago. Nana Matoba, MD,MPH, is an assistant professor of pediatrics in Northwestern University’s Feinberg School of Medicine, a neonatologist at Ann & Robert H. Lurie Children’s Hospital of Chicago. Both are Public Voices Fellows through The OpEd Project.