Right to Try
Understanding an Emerging Pathway for Access to Investigational Treatments
What Is Right to Try?
The Right to Try Act was signed into law in 2018 and created a pathway for certain patients with life-threatening illnesses or conditions to request access to investigational treatments that have not yet received approval from the U.S. Food and Drug Administration (FDA).
Right to Try was designed for individuals who have exhausted approved treatment options and are unable to participate in a clinical trial. Under this pathway, patients, their physicians, and treatment developers may work together to request access to certain investigational therapies without FDA review of the individual request.
It is important to note that Right to Try does not guarantee access to a treatment. Participation remains voluntary for treatment developers and manufacturers.
Who May Be Eligible?
Under the federal Right to Try Act, patients generally must meet several criteria:
Have a life-threatening disease or condition
Have exhausted available approved treatment options
Be unable to participate in a clinical trial involving the investigational treatment
Receive a recommendation from a treating physician
Provide written informed consent
Eligibility determinations are made on an individual basis and may involve additional medical, legal, and logistical considerations.
What Is an Investigational Treatment?
An investigational treatment is a drug, biologic, or other medical intervention that is still being studied and has not yet been approved by the FDA for general use.
These treatments may show promise in research settings, but they have not yet completed the full FDA approval process. As a result, important questions about safety, effectiveness, appropriate dosing, and long-term outcomes may still be under investigation.
How Is Right to Try Different from Clinical Trials?
Clinical trials are research studies designed to evaluate the safety and effectiveness of investigational treatments. Participants are enrolled according to strict research protocols and contribute data that may help determine whether a treatment should receive FDA approval.
Right to Try is different. Rather than participating in a research study, eligible patients may seek access to an investigational treatment outside of a clinical trial.
Because Right to Try is not a research pathway:
The FDA does not review individual Right to Try requests.
The treatment is not being provided as part of a research study.
Safety and effectiveness may not be fully established.
Manufacturers are not required to provide access.
Potential Benefits and Limitations
Potential Benefits
Expanded access for individuals facing serious or life-threatening conditions
Additional options when approved treatments have been exhausted
Greater flexibility for patients who cannot participate in clinical research
Important Limitations
Treatments available through Right to Try have not been approved by the FDA.
The risks and side effects may not be fully understood.
Effectiveness may not be established.
Access may be limited by manufacturer participation, cost, availability, or legal considerations.
Right to Try does not guarantee that a treatment will be provided.
Patients considering investigational treatments should discuss potential benefits and risks with qualified healthcare professionals.
Why Is Right to Try Being Discussed in Psychedelic Medicine?
As research into psychedelic-assisted therapies continues to expand, some advocates, clinicians, researchers, and policymakers have explored whether Right to Try may eventually play a role in providing access to certain investigational psychedelic treatments.
Several psychedelic compounds, including psilocybin and MDMA, are currently being studied for conditions such as depression, post-traumatic stress disorder (PTSD), end-of-life distress, and substance use disorders.
However, many psychedelic therapies remain investigational and are not broadly available through standard medical pathways. Questions continue to be explored regarding how existing laws, regulations, and access frameworks—including Right to Try—might apply to these emerging treatments.
The legal and regulatory landscape remains complex and continues to evolve. Access pathways may vary depending on federal law, state law, clinical research status, and treatment-specific regulations.
Looking Ahead
Right to Try represents one of several pathways being discussed as healthcare providers, researchers, policymakers, and patients explore how to responsibly increase access to promising investigational treatments.
As scientific research advances and regulatory frameworks continue to develop, conversations about safety, access, ethics, affordability, and patient choice will likely remain central to the future of emerging therapies.