Right To Try Act

• The Right To Try Act allows patients with life-threatening conditions to seek experimental treatments.
• It applies when a patient has exhausted approved options and cannot enroll in a clinical trial.
• The investigational drug must have completed a Phase 1 clinical trial and not be approved or cleared by the FDA.
• Patients, their physicians, and drug manufacturers must agree to the treatment.
• The Act aims to accelerate access but does not guarantee treatment availability or efficacy.

What is the Right To Try Act?

The Right To Try Act is a U.S. federal law enacted in 2018 that permits eligible patients with life-threatening diseases to access certain unapproved medical products. These products, which include drugs, biological products, and medical devices, must have successfully completed a Phase 1 clinical trial and remain under investigation by the Food and Drug Administration (FDA). The law aims to provide an alternative pathway for patients who have exhausted all approved treatment options and are not able to enroll in a clinical trial. This legislation empowers patients and their physicians to seek access directly from drug manufacturers, bypassing certain FDA review processes that typically govern expanded access programs. The Right To Try Act explained is designed to accelerate access to potentially life-saving treatments for those with the most urgent medical needs.

Navigating the Right To Try Act Process

The process for the Right To Try Act involves a specific sequence of steps that patients, physicians, and manufacturers must follow. Unlike traditional expanded access pathways, the Right To Try Act primarily facilitates direct communication between patients and drug developers, with less direct FDA oversight in the approval process for individual patient requests.

Here’s a general overview of the process:

• Physician Recommendation: A licensed physician must diagnose the patient with a life-threatening disease and certify that the patient has exhausted all approved treatment options and is unable to participate in a clinical trial involving the investigational product.
• Manufacturer Agreement: The patient or physician must contact the drug manufacturer directly to request access to the investigational product. The manufacturer is not obligated to provide the drug but may choose to do so.
• Informed Consent: The patient must provide written informed consent, acknowledging the risks and potential benefits of using an unapproved product.
• FDA Notification (Post-Use): While the FDA does not approve individual Right To Try requests, manufacturers are required to submit an annual summary of their Right To Try uses to the FDA. This notification is for data collection and safety monitoring rather than pre-approval.

It’s important to note that the manufacturer must have completed a Phase 1 clinical trial for the drug, and the drug must be under active investigation or development.

Patient Eligibility for Right To Try

Specific criteria must be met for Right To Try Act patient eligibility. These conditions ensure that the law is applied to patients who genuinely have no other viable treatment options and are facing severe health challenges.

To be eligible, a patient must meet all of the following requirements:

• Life-Threatening Disease: The patient must have been diagnosed with a life-threatening disease or condition. This generally refers to a disease or condition where the likelihood of death is high unless the course of the disease is interrupted, or a disease or condition with an immediately life-threatening component.
• Exhausted Approved Treatments: The patient must have exhausted all available FDA-approved treatment options for their disease or condition.
• Inability to Participate in Clinical Trials: The patient must be unable to participate in a clinical trial for the investigational drug. This could be due to various factors, such as not meeting the inclusion/exclusion criteria, geographical limitations, or the unavailability of a suitable trial.
• Phase 1 Completion: The investigational drug must have successfully completed a Phase 1 clinical trial. This indicates that the drug has demonstrated an acceptable safety profile in humans.
• No FDA Approval/Clearance: The investigational drug must not be approved or cleared by the FDA for any use.

The decision to pursue treatment under the Right To Try Act should always be made in close consultation with a qualified physician, considering all potential risks and benefits.