Federal "Right to Try" Law Faces Renewed Scrutiny Over Access for Children

A recent social media post by "The Rabbit Hole" has reignited public discussion regarding the federal "Right to Try" (RTT) law, specifically questioning its application for children seeking potentially life-saving experimental treatments. The tweet, which garnered attention on September 25, 2025, directly appealed to House Speaker Mike Johnson and the House of Representatives to "consider" allowing children access to such therapies when "the alternative is much worse."

The federal "Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017" allows terminally ill patients to access investigational drugs that have completed Phase 1 clinical trials but are not yet fully approved by the Food and Drug Administration (FDA). This legislation, enacted in 2018, was partly spurred by high-profile cases like that of Josh Hardy, a child with a rare form of kidney cancer who sought an experimental drug in 2014. The FDA's role under this law is limited to receiving and posting information, not approving individual requests.

Despite the law, drug manufacturers are not obligated to provide investigational drugs, and the FDA does not review or approve individual Right to Try requests. This framework has led to ongoing debate regarding patient access and the responsibilities of pharmaceutical companies. In a related development, the "Right to Try Clarification Act" (H.R.1825) was introduced in March 2023, aiming to extend the federal RTT law to include Schedule I controlled substances that have completed Phase 1 trials. This bill remains under consideration by House committees.

Advocates for expanded Right to Try access argue it provides a crucial last resort for patients with no other options, emphasizing patient autonomy. However, some patient groups and pharmaceutical industry representatives have historically expressed concerns. They highlight potential risks of unapproved therapies and the need for robust clinical trials to ensure safety and efficacy. The current appeal to Speaker Johnson underscores persistent calls for legislative review and potential amendments to ensure children, in particular, are adequately served by these compassionate access pathways.