By: Scott A. Hagen
Originally published in Utah Physician Magazine’s February/March 2025 Issue
One of the most difficult moments in life comes when a patient and the patient’s loved ones recognize that the patient is suffering from a terminal disease and all potential remedies have been exhausted. At that point, faced with an end-of-life prognosis, the patient often wants to try anything that might bring relief. Physicians also are reluctant to simply advise a patient to prepare for the end of life rather than look for a cure.
Sometimes, there are potential remedies still in the developmental or experimental process; such remedies might be beneficial, but have not yet met all of the requirements to receive approval from the Food and Drug Administration. Such potential remedies are “experimental” and would not be approved for use in ordinary circumstances. Right to Try laws and the FDA’s Expanded Access Program provide a limited avenue for use of such experimental remedies.
The Utah Right to Try Act, enacted in May 2015, sets out certain circumstances under which physicians licensed under Utah law may prescribe an “investigational drug” or “investigational device” for a patient without being subject to liability for medical malpractice.
First, the patient must have been diagnosed with a terminal (end-of-life) illness that a physician determines will pose a greater risk to the patient than the risk from treatment with a particular investigational drug or device.
Second, the “investigational drug” or “investigational device” must meet the definition of “investigational new drug” or “investigational device” in federal law and must have successfully completed Phase I testing under FDA supervision.
Third, the patient, physician, and manufacturer must agree to an “informed consent document” that meets certain statutory criteria designed to ensure the patient enters treatment with full awareness of the risks.
If these criteria are met, the patient can receive this experimental treatment without waiting for formal FDA approval or supervision, and the physician is protected from liability in the event of an adverse outcome. Indeed, “right to try” laws were inspired at the state level by libertarian institutions as a way to obtain last-chance experimental medical treatment without “big government” involvement.
The federal right to try law, enacted in 2017 and effective in 2018, bars the FDA from using clinical outcomes in right-to-try treatments to delay or adversely affect FDA review or approvals except in narrow circumstances. In addition, the federal law requires manufacturers to report the use of investigational drugs and devices pursuant to right to try laws and any adverse outcomes and protects physicians and manufacturers from liability except in cases of reckless or willful misconduct.
Ironically, there has apparently been very little use of this right to try process. As far as I can tell from the FDA website, the right to try procedure, where investigational drugs or devices are used to treat a patient with a terminal illness who has tried all other options, was only invoked 16 times from 2018 to 2023. See https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/right-try-annual-reporting-summary (last accessed on February 2, 2025).
This is likely because of the FDA’s “expanded access” program, where patients are permitted to use investigational drugs or devices in essentially the same circumstances as addressed in right to try laws, but with formal FDA approval of the use for that one patient. Under FDA expanded access, physicians can apply for FDA approval to use an investigational drug or device in treating a particular patient using an emergency procedure that typically results in FDA approval in a very short period of time, even as short as a day or two. For this reason, in the vast majority of circumstances, the investigational drug or device has been provided with FDA approval under the expanded access program. In other words, experience has shown there is no need to use the right to try procedure, even when providing an investigational treatment not yet fully FDA-approved.
Most recently, some states are expanding on the original right-to-try legislation in two ways. First, rather than limit use to circumstances where the patient has a terminal illness and has exhausted all other treatment options, new laws allow investigational treatment when the patient has a severely debilitating illness and has merely considered all other options. Second, investigational treatments include individualized genomic and genetic treatments that need not have completed an FDA Phase I clinical trial. It appears that this right to try expansion has not yet been proposed in Utah.
State and federal right-to-try laws, along with the FDA’s expanded access program, provide terminally ill patients with a pathway to the use of experimental treatments when all else has failed. Physicians should be aware of these options for use when confronted with these difficult life and death situations.
Scott A. Hagen
moc.nqr@negahs
801-323-3328
Scott A. Hagen is the Chair of the Firm’s Healthcare Law Section and former Chair of the Employment and Labor Section. His practice includes labor relations, employment litigation, employee benefits (ERISA) litigation, and representation of clients before administrative agencies. Mr. Hagen has represented management in collective bargaining, labor arbitrations, unfair labor practice charges, representation elections, collective bargaining, state court injunctions regarding improper picketing, and hybrid lawsuits alleging breach of a collective bargaining agreement and breach of the fair duty of representation.