‘Urgent Action’ on Puberty Blockers Sought

A group of five doctors and eight organizations have submitted a citizen petition to FDA calling for urgent action on the off-label use of GnRH agonists, a class of drugs also known as puberty blockers. “We are concerned by the dramatic increase in the off-label use of puberty blockers to treat children with gender dysphoria,” the petition says. “Despite the widespread, and rapidly growing, use of these drugs in this population, and despite serious known and potential risks from these drugs, this use has never been evaluated for safety and effectiveness by FDA.”

The National Institutes of Health says that approved indications for these drugs, depending on the specific agent, include advanced prostate cancer, endometriosis, and precocious puberty.

The petitioners say that although the use of the drugs to treat gender dysphoria has not been approved by FDA, the agency “must not turn its back on the potential harm to children from continued use of a drug with known risks and unproven benefits. We are asking FDA to fulfill its public health mission by commissioning a comprehensive analysis of existing studies concerning this use, by seeking new, long-term studies of this use, by promoting awareness of the state of evidence concerning this use and that this use is off-label, and by alerting manufacturers and healthcare providers that because this use is unapproved, it is unlawful to promote this use, directly or indirectly.”

The petition says such actions are needed to understand the long-term risks and outcomes associated with the use of puberty blockers in children with gender dysphoria, to promote public awareness that this use has not been shown to be safe or effective, and to prevent further harm to this vulnerable population.

The specific actions requested in the petition are:

• commission a systematic review of the off-label use by the National Academy of Sciences, Engineering, and Medicine;
• issue written requests under the Best Pharmaceuticals for Children Act for long-term registry studies of the off-label use;
• create a dedicated Web page on the off-label use; and
• alert manufacturers and providers to the consequences of unlawful promotion of the off-label use.

The five doctors who signed the petition are a family physician, a child and adolescent psychiatrist, a pediatric urologist and ethicist, an endocrinologist, and a pediatric endocrinologist. The groups signing the petition are Detrans Help, FAIR in Medicine, Gender Dysphoria Alliance, Gender Exploratory Therapy Association, Genspect, International Partners for Ethical Care, Our Duty USA, and Rethink Identity Medicine Ethics. The Web sites for several of the organizations oppose gender identity care or exploration.