Why Was the Puberty Blockers Trial Paused by the Medicines Watchdog?

The clinical trial evaluating the use of puberty-blocking drugs in children questioning their gender has been paused due to safety concerns raised by the UK medicines watchdog, the Medicines and Healthcare Products Regulatory Agency (MHRA). This decision comes as the MHRA seeks to address significant issues regarding participant safety and the trial's design prior to recruitment.

Last updated: 05 October 2023 (BST)

What’s happening now

The MHRA has paused the Pathways clinical trial, which was intended to assess the risks and benefits of puberty blockers for children aged 11 to 15 who are distressed about their gender identity. This pause follows concerns that the current age limit for participants may be too low, and the agency has indicated that it may recommend raising the minimum age to 14. The MHRA emphasised that the "safety and wellbeing" of participants is paramount, and discussions will take place with researchers from King's College London to address these issues before the trial can proceed.

Key takeaways

The Pathways trial has been paused due to safety concerns from the MHRA.
Discussions are set to take place between the MHRA and King's College London regarding participant safety.
There are calls to raise the minimum age for trial participants to 14.
Concerns have been raised about the informed consent process for participants.
The trial is designed to assess the impact of puberty blockers on physical, social, and emotional wellbeing.

Timeline: how we got here

The Pathways trial was approved by the MHRA and Health Research Authority in November 2022. The trial was designed to involve over 220 children aged 11 to 15, who are currently accessing gender services. However, concerns regarding the safety of these treatments have led to ongoing scrutiny. The MHRA's concerns were articulated in a letter released on 5 October 2023, prompting the current pause and forthcoming discussions aimed at resolving the safety issues identified.

What’s new vs what’s known

New today/this week

The primary development is the pause of the trial, as announced by the MHRA. They expressed worries about the long-term biological risks associated with puberty blockers for participants under the age of 14. The agency's recommendation to consider raising the age limit reflects a cautious approach to the trial's design and the need for thorough monitoring.

What was already established

Prior to this pause, the Pathways trial was seen as a critical step in understanding the impacts of puberty blockers on young individuals questioning their gender identity. A 2024 review by Dr Hilary Cass highlighted concerns about the safety and efficacy of these treatments, leading to a ban on their use for under-18s. This review indicated that the field of gender medicine required more robust evidence to support its practices.

Impact for the UK

Consumers and households

The pause of the trial affects families seeking answers and support regarding gender identity issues in children. It may delay access to potential treatments for those experiencing distress related to their gender identity. For parents and guardians, this situation raises questions about the adequacy of existing support services and the implications for their children's mental health and wellbeing.

Businesses and jobs

For healthcare providers and organisations involved in gender services, the pause may impact ongoing treatment plans and the operational landscape surrounding gender identity support. It could also affect employment opportunities within the sector, as the trial's outcomes may shape future healthcare policies and practices.

Policy and regulation

The MHRA's actions and forthcoming discussions with King's College London will likely influence future regulatory frameworks concerning puberty blockers in the UK. The trial's outcome could lead to stricter guidelines for clinical practices related to gender identity treatments, reflecting an increasing emphasis on participant safety and ethical considerations.

Numbers that matter

220+ children were expected to participate in the Pathways trial.
Minimum age for participants may be raised from 11 to 14.
Puberty blockers for under-18s were banned in 2024 due to safety concerns.
The MHRA has called for more detailed monitoring of bone density in trial participants.
A 2024 review indicated that gender medicine was based on "shaky foundations".

Definitions and jargon buster

Puberty blockers: Hormonal treatments that delay the onset of puberty in children, often used in gender dysphoria cases.
MHRA: Medicines and Healthcare Products Regulatory Agency, the UK agency responsible for ensuring medicines and medical devices are safe and effective.
Pathways trial: A clinical trial assessing the impact of puberty-blocking drugs on children questioning their gender identity.

How to think about the next steps

Near term (0–4 weeks)

In the immediate future, stakeholders will await the outcome of discussions between the MHRA and King's College London. This dialogue will address the safety concerns raised and may lead to adjustments in the trial's design.

Medium term (1–6 months)

If the trial resumes, it will be essential to monitor recruitment and the subsequent data collected on participant outcomes. This period may also see the establishment of new guidelines regarding informed consent and participant age requirements.

Signals to watch

Updates from the MHRA regarding the trial's status and any recommended changes.
Reactions from healthcare providers and advocacy groups concerning the trial and its implications for treatment practices.
New research findings or reviews concerning the use of puberty blockers in under-16s.

Practical guidance

Do

Stay informed about updates from the MHRA regarding the trial.
Seek professional guidance if you have concerns about gender identity or treatment options for children.
Engage with support groups that focus on gender identity issues for additional resources.

Don’t

Don’t disregard the importance of informed consent in medical treatments.
Don’t hesitate to ask questions about the safety and efficacy of treatments being considered.
Don’t overlook the potential psychological impacts of delaying treatment for children experiencing gender dysphoria.

Checklist

Review the latest guidelines from the MHRA regarding puberty blockers.
Consider the age and wellbeing of the child when discussing treatment options.
Assess the qualifications of healthcare professionals involved in gender services.
Evaluate the available support systems for families navigating gender identity issues.
Stay updated on any legal developments related to the trial and treatment practices.

Risks, caveats, and uncertainties

There are significant uncertainties surrounding the safety and efficacy of puberty blockers, particularly for young individuals. The MHRA's pause reflects concerns over potential long-term biological risks, which have yet to be fully quantified. Additionally, the ethical implications regarding informed consent for minors remain contentious, raising questions about whether children can genuinely understand the long-term consequences of such treatments. As discussions progress, stakeholders should remain cautious and attentive to emerging evidence and regulatory changes.

Bottom line

The pause of the Pathways trial underscores the need for rigorous safety assessments in clinical trials involving vulnerable populations. As the MHRA seeks to address its concerns, it is crucial for families and healthcare providers to remain informed about developments in gender identity services and treatment options in the UK.

FAQs

What are puberty-blocking drugs?

Puberty-blocking drugs are medications used to delay the onset of puberty in children experiencing gender dysphoria. They aim to provide time for individuals to explore their gender identity without the immediate physical changes associated with puberty.

Why has the Pathways trial been paused?

The Pathways trial has been paused due to safety concerns raised by the MHRA regarding the risks associated with puberty blockers for participants under 14 years of age, as well as the adequacy of the informed consent process.

What is the MHRA's role in clinical trials?

The MHRA is responsible for regulating medicines and healthcare products in the UK, ensuring that clinical trials are conducted safely and ethically while safeguarding the wellbeing of participants.