Christopher Bellonci, MD, Judge Baker Children’s Center, Harvard Medical School
If you were to learn that 79% of a population had serious thoughts about suicide in their lifetime[1], 69% with depression symptoms and 75% with significant anxiety symptoms[2], would you consider this an urgent public health crisis for this population? For many transgender, gender expansive, and intersex young people who do not have access to affirming care, this is a reality[3]. Gender affirming care (GAC) is a life-saving treatment approach to address and prevent this health crisis and help young people live fulfilling lives. Yet, the recent Cass review out of the UK raised questions about the state of the research regarding the potential benefits and harms of gender affirming care for youth[4]. Noting that the quality of existing studies showing positive outcomes for youth receiving GAC was poor, the Cass report recommended that access to GAC for youth in the UK be limited to approved research studies which will result in severely limiting access to what are life-saving treatments. How did we get here?
When asking the question, “what does the science tell us about…?” we understandably take a rigorous review of existing studies and following the best scientific principles we rate the level of evidence including potential biases in the methodology or by potential conflicts of interest of the researchers[5]. This is a very high scientific standard and one that many areas of scientific inquiry, not just GAC research, struggle to attain. When I was on the American Academy of Child and Adolescent Psychiatry’s Quality Issues Committee, we authored clinical practice guidelines based on systematic reviews done by the US Agency for Healthcare Research and Quality (AHRQ). In one study focused on the benefits and risks of using antipsychotic medications for children and adolescents, the evidence was surprisingly sparse and the risks potentially significant, leaving the committee in a dilemma about whether we could justify recommending their use versus merely suggesting they be considered. There was no one on the committee (and likely in the field of child psychiatry) who doubted the wisdom of treating psychotic disorders in adolescents with antipsychotic medications. So, what do we do when the science may not be of the highest quality (i.e., not randomized controlled trials [RCTs]) but we have patients we know have benefited from an intervention with many studies of less scientific rigor showing benefits? Does evidence-based medicine directs us to forego an intervention until we achieve the research gold-standard?
The answer to this question is a resounding, “NO!” When the Institutes of Medicine published their seminal document, Crossing the Quality Chasm in 2001, they understood the importance of ensuring that the best available science should be used to inform medical decisions for patients. It also emphasized that healthcare should be safe, effective, patient-centered, timely, efficient, and equitable. One way of thinking about the components of evidence-based clinical decision-making is that there are three legs to the stool: 1) what the existing science tells us works, 2) clinician experience and 3) patient values and preferences. If we stop with just what the science affirms to be true, we fail to consider clinical experience and, most importantly, patient preferences. This is not how evidence-based medicine is intended to work.
So, what do we do when we have emerging evidence that an intervention, such as GAC, shows promise in addressing a vital health concern such as the significant behavioral health burdens of gender dysphoria. Are we supposed to wait until there are a sufficient number of randomized control trials before we provide care for these youth? What about the cost of our inaction? How much suffering must these youth endure and how many lives will be lost while we create the evidence? It takes an average of 17 years for new knowledge generated by randomized controlled trials to be incorporated into practice, and even then, application is highly uneven5. 17 years represents an entire generation of young people who will be denied care while we wait for RCTs. And why is GAC being held to a standard that almost no other behavioral health intervention for children and adolescents is required to meet?
This is a distortion of the importance of using science to guide our clinical decision-making. Yes, we need more research on outcomes. While building that research, we should also be providing necessary care in a transparent way to determine what is best for transgender and nonbinary young people and their families, because GAC saves lives.
“The hormones didn’t change my brain; they changed my body to finally match my brain. And the more I saw the real me in the mirror, the more my anguish faded. Gender affirming health care literally saved my life.”[6]
Ultimately our responsibility is to offer compassionate, evidence-informed treatment to patients so that they get to decide what is best for them, given what is known and unknown about the risks and benefits of an intervention. To do otherwise would be failing in our scientific mission to ensure that gender affirming care is safe, effective, patient-centered, timely, efficient, and equitable, just as the Institutes of Medicine has called for.
[1] Marquez-Velarde, G., Miller, G. H., Shircliff, J. E., & Suárez, M. I. (2023). The Impact of Family Support and Rejection on Suicide Ideation and Attempt among Transgender Adults in the U.S. LGBTQ+ Family: An Interdisciplinary Journal, 19(4), 275–287. https://doi.org/10.1080/27703371.2023.2192177
[2] 2022 National Survey on LGBTQ Youth Mental Health. The Trevor Project. Accessed June 12, 2024. https://www.thetrevorproject.org/survey-2022
[3] It is important to note, transgender, gender expansive and intersex youth experience higher rates of behavioral health issues primarily because of minority stressors including discrimination, prejudice, violence, and lack of affirming and responsive care.
[4] https://cass.independent-review.uk/home/publications/final-report/
[5] Institute of Medicine (US) Committee on Quality of Health Care in America. Crossing the Quality Chasm: A New Health System for the 21st Century. Washington (DC): National Academies Press (US); 2001. Executive Summary. Available from: https://www.ncbi.nlm.nih.gov/books/NBK222271/
5 Ibid
[6] Alexa. (2023). Gender affirming care saved my life. Everyone should have access to it. ACLU Indiana Blog Post. Retrieved on June 12, 2024 at https://www.aclu-in.org/en/news/gender-affirming-care-saved-my-life-everyone-should-have-access-it#:~:text=The%20hormones%20didn’t%20change,care%20literally%20saved%20my%20life.
This resource was prepared for the Substance Abuse and Mental Health Services Administration (SAMHSA) under contract number H79FG000583 with SAMHSA, U.S. Department of Health and Human Services (HHS). The views, opinions, and content of this publication are those of the author and do not necessarily reflect the views, opinions, or policies of SAMHSA or HHS.