Eligibility for Right to Try - Personalized Stem Cells

The Right to Try Act was passed into federal law in 2018. It provides a new channel for eligible patients to request access to investigational drugs outside of any FDA clinical trials. Often, clinical trials are not available, too restrictive, or too far a distance for the patient. Patients are eligible if they have a life-threatening disease or severely debilitating illness. Life-threatening means diseases or conditions where the likelihood of death is high unless the course of the disease is interrupted. Severely debilitating means diseases or conditions that cause major irreversible morbidity (illness)¹. Eligible patients must have also considered all other treatment options currently approved by the United States Food and Drug Administration, exhausted all these approved treatment options, and are unable to participate in a clinical trial involving the drug or biologic of interest².

Eligible patients need to work with a physician to help them gain access to an unapproved drug outside of the clinical trial of interest. The patient’s physician will provide a treatment plan and an informed consent for the use of the investigational drug, so that patients understand fully the investigational drug treatment’s risks and benefits. With the plan and consent in hand, physician and patient can reach out to the manufacturer or sponsor who is developing this new drug to learn if they are willing to make the investigational drug available³.

For an investigational drug to be eligible, it has to have completed at least one phase I clinical trial (basic safety) and have never been approved for commercial sale by the FDA for any medical use. It must also be under active development or production and not discontinued by the manufacturer or placed on a clinical hold by the FDA⁴.

A major goal of the Right to Try Act was to provide patients suffering with a life-threatening disease or severely debilitating illness new access to treatments after they have exhausted approved treatments without success and are unable to gain access via a clinical trial that may hold promise for treating their disease or condition⁵. FDA approval is not required for participation via the Right to Try Act.

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