Pioneering the Path for FDA Approval of Gender-Affirming Hormone Therapies: The Research Institute for Gender Therapeutics’ Groundbreaking Initiative

The Research Institute for Gender Therapeutics (RIGT), a nonprofit organization, is pioneering research to gain FDA approval for the use of estrogen as a gender-affirming treatment for transgender and gender-diverse patients. Historically, hormone replacement therapies like estrogen have been approved for cisgender patients, such as menopausal women, but have been prescribed off-label for transgender healthcare. RIGT, established to legitimize gender-affirming hormones, submitted a plan for a Phase 3 clinical trial focusing on estradiol, a common form of estrogen in hormone treatments, and received the FDA’s feedback on November 17.

The FDA’s response was unexpectedly supportive, advising against a placebo-controlled trial and recommending the inclusion of adolescents as young as 13. This feedback is significant at a time when many states are restricting hormone treatment and puberty blockers for youth. Brad Sippy, RIGT’s president, expressed that this guidance from the FDA validates the treatment of gender incongruence and provides a clear path to federal approval for gender-affirming care.

RIGT aims to expand its research beyond estradiol to include testosterone and puberty blockers. Initially, RIGT proposed a double-blind placebo-controlled study for adults, but the FDA advised excluding placebo treatments. Experts in gender-affirming care support this, citing ethical concerns and the impracticality of blinding due to the evident physical effects of hormone treatments, such as breast development and changes in hair growth.

The FDA’s feedback also reflects a shift in the perception of transgender and gender-diverse identities, supporting the classification of gender incongruence as a sexual health condition rather than a mental health issue, aligning with the World Health Organization’s classification. This is a significant step away from the term “gender dysphoria” used in the Diagnostic and Statistical Manual of Mental Disorders.

Before initiating the study, RIGT needs to finalize its study design, gain FDA approval for safety, and receive approval from an institutional review board. The organization also funds the purchase of estradiol and may collaborate with established researchers. The study is anticipated to start in 2025.

Critics of gender-affirming care often cite the lack of FDA approval for hormone treatments. However, Alex Keuroghlian, a Harvard Medical School associate professor and LGBTQ+ health expert, doubts FDA approval will quell opposition, comparing it to the politicized battle over abortion rights.

RIGT’s founders, including Nicole LaRocque, are motivated by personal connections to the trans and nonbinary community. LaRocque, a parent of a transgender child, emphasizes the importance and availability of quality medical care for this group. They aim to ensure broader acceptance and legitimacy for gender-affirming treatments through official medical endorsement.

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[Source: STAT, November 28th, 2023]