Reassessing Pediatric Gender Care: Evidence, Transparency, and the Path Forward

The prevailing consensus from organized medicine currently favors pediatric medical transition (PMT). This comes in contrast to mainstream America where 71% of respondents to a New York Times/Ipsos poll earlier this year agreed that “no one under age 18 should have access” to puberty-blocking medications or hormone therapy. Similarly, lawmakers in 27 states have enacted laws – of varying degrees – banning PMT. The difference between the medical establishment and the American public on the care of minors with gender dysphoria is real.

This divide was only further highlighted by the response to the recently published report from the Department of Health and Human Services (HHS), “Treatment for Pediatric Gender Dysphoria – Review of Evidence and Best Practice.” The American Academy of Pediatrics (AAP), on the same day as the report’s release, chided the authors for not consulting the organization and “[misrepresenting] the current medical consensus.” Also on that same day, five additional physician groups, along with the AAP, issued a joint statement echoing their collective support for the “full spectrum of evidence-based health care.”

Interestingly, the HHS report does just that – assess the evidence. It offers an umbrella review, thereby evaluating systematic reviews themselves. To conduct the overview, the report’s authors followed the Cochrane Handbook for Systematic Reviews of Interventions. The risk of bias for each systematic review was assessed using a standard tool. Then, for each outcome, the authors used a standard methodology to determine the certainty or quality of the evidence. After assessing 286 studies for eligibility, seventeen systematic reviews met the inclusion criteria, of which ten were determined to have a low risk of bias.

Yet, the AAP argues the HHS report “must consider the totality of available data.” A series of questions naturally follows: What data might the HHS review have omitted that the AAP would have included? At the risk of lowering the quality of evidence, should the HHS have incorporated select, individual studies beyond systematic reviews? And, of the systematic reviews deemed eligible, should those focused primarily on adults have been included?

One such excluded review was from Dr. Kellan Baker and colleagues, which has been cited 305 times according to Google Scholar and was conducted to inform the World Professional Association for Transgender Health’s (WPATH’s) Standards of Care for the Health of Transgender and Gender Diverse People, Version 8 (SOC-8). Yet, any definitive conclusions related to the care of minors should be taken with caution. For example, Baker included just one trial of 50 adolescents which showed “no difference in [quality of life] scores after a year of endocrine interventions.” Furthermore, while less than a handful of studies on depression showed some improvement, the risk of bias was determined to be either “moderate” or “serious.” The abstract itself noted a “high risk of bias in study designs, small sample sizes, and confounding with other interventions.” The study’s authors “could not draw any conclusions about death by suicide.”

The dearth of high-quality evidence unfortunately extends throughout this area of medicine. HHS’s review confirmed what our colleagues overseas had previously reported. Simply, HHS’s report noted, “the overall quality of evidence concerning the effects of any intervention on psychological outcomes, quality of life, regret, or long-term health, is very low.” Studies related to PMT are often of low quality given small sample sizes, significant attrition of enrollees, and relatively short follow up periods. Selection bias, uncontrolled confounding, and lack of a comparison group also weigh heavily on many of these trials. All of this leads to low quality evidence in the face of life altering interventions.

Moreover, data has not always been published in a timely manner. This past fall The New York Times reported that Dr. Johanna Olson-Kennedy’s “long-awaited study of puberty-blocking drugs” remained unpublished. This study began nearly a decade ago as part of a National Institutes of Health grant. As per the Times’ article, Olson-Kennedy’s study of 95 children did not find improvements in mental health from puberty blockers. After many years of waiting a preprint was posted last month confirming this to be the case. Similarly, findings from England’s youth gender clinic in 2011 showed that puberty blockers had “not changed volunteers’ well-being, including rates of self harm.” It was not until 2020 that those results were made public.

Within this climate, unwavering transparency, critical engagement, and intellectual rigor are needed now more than ever from both individual physicians and organized medicine. Physicians – particularly those caring for youth with gender dysphoria – must come to read and understand the HHS report. The duty to be informed is paramount, especially when the well-being of America’s children is at stake and irreversible interventions are in question.

Similarly, medical societies like the AAP and the Endocrine Society must review their clinical practice guidelines for PMT independent of external influence. Uncritically accepting WPATH’s recommendations, for example, will not serve this purpose, particularly when WPATH itself suppressed its own evidence reviews and allowed political pressures to influence its standards of care. Fortunately, the AAP opened the door to a new direction when its Board of Directors nearly two years ago called for a “systematic review of the evidence.” Now is the time to deliver on this directive.

As part of any reevaluation, American medicine must place a renewed focus on psychosocial support and psychotherapy as a legitimate and essential modality of care – one that carries no physical harms and prioritizes long-term well-being. Medicine in general, and pediatrics in particular, needs to move past the consumerist model, where minors – who are not of age to give informed consent – direct their own medical treatment absent valued clinical input. We cannot neglect our duty as physicians. In each clinical encounter patients and parents look to us as highly educated and well-trained professionals who must offer more than technical prowess. Physicians bear great moral responsibility, making it imperative that we act as thoughtful practitioners who weigh the totality of evidence along with our ethical duty to more than just blind allegiance to patient autonomy and little else. Ultimately, we must first do no harm.

To that end, the American Medical Association (AMA) must act, recognizing its unique role as America’s largest medical association with representation from numerous specialty societies at its House of Delegates. The AMA should ensure coordination of efforts among America’s medical societies to increase transparency, promote open debate, and ensure rigorous scientific scrutiny in the reevaluation of clinical practice guidelines related to pediatric gender medicine. The broader medical community, rather than a select group of individuals, must be involved. This entails reading and understanding the HHS’s review, as well as engaging with its contributors once the post-publication peer review process is complete.

The time has come for the medical community to have an honest conversation about PMT. It is of the utmost importance for the health and wellbeing of our nation’s youth that we, as physicians, have this dialogue and take the necessary appropriate steps. It is also critical to the integrity of our profession if we are to maintain the trust of the American people. The public is watching, and our actions must speak louder than the words we have thus far offered.