The concept of a “Right to Try” has been spreading through the U.S. over the last four years, beginning with the first passing of such laws in Colorado in 2014. Most recently, President Trump has echoed support for a federal Right to Try law in his State of the Union address. The core concept of a “Right to Try” is that terminally ill patients should be able to access experimental therapies that have successfully completed a Phase I clinical trial but have not completed FDA Phase II and III clinical trials and received FDA approval to enter the market.
As of April 16, 2018, 39 states have passed Right to Try laws, including:
Alabama, Arizona, Arkansas, California, Colorado, Connecticut, Florida, Georgia, Idaho, Iowa, Illinois, Indiana, Kentucky, Louisiana, Maine, Maryland, Michigan, Minnesota, Mississippi, Missouri, Montana, Nevada, New Hampshire, North Carolina, North Dakota, Ohio, Oklahoma, Oregon, Pennsylvania, South Carolina, South Dakota, Tennessee, Texas, Utah, Virginia, Washington, West Virginia, Wisconsin, and Wyoming.
On March 22, 2018, the U.S. House of Representatives passed H.R. 878, the “Right to Try Act of 2017.” This federal legislation would enable these state laws, protecting participants, including patients as well as physicians and treatment manufacturers, from FDA and other federal enforcement actions.
Specifically, the federal Act provides two principal protections:
- The producer, manufacturer, distributor, prescriber, dispenser, possessor or user of an experimental treatment will not be held liable for a treatment used in compliance with the relevant state law.
- The production, manufacture, distribution, prescribing, dispensing, possession, or use of a treatment under the law cannot be used by a federal agency, like the FDA, when reviewing the treatment for approval.
The Act has two main sections: the requirements that a state law must have for the protections to apply, and the protections the Act provides to the entities and individuals involved in providing the treatment.
The proposed federal law has two threshold requirements for a state, physician, manufacturer and patient to take advantage of its safe haven protection from any federal enforcement action: 1) the state statute must authorize the treatment; and 2) the patient must be diagnosed with a terminal illness.
The first criteria, recognition of the treatment is fairly consistent across these state laws. Generally, state Right to Try laws require the treatment must have first completed (successfully) Phase I clinical trials. The reason for this threshold is clear: A Phase I clinical trial determines effective dosing of the particular product being tested while monitoring for safety at a maximum allowable dosing. Phase I trials typically involve a smaller number of patients than later testing, and are done in both healthy and sick patient populations depending on the intended use of the test product.
Typically a drug product that is being tested under an Investigational New Drug application will, assuming acceptable safety data was obtained from a Phase I clinical trial, then move to a Phase II to study the study drug’s efficacy and collect further safety data. State Right to Try laws generally do not require Phase II clinical trials to qualify a drug product for use under the terms of the state statute.
The second criteria in the Act is that the patient be diagnosed with a terminal illness. While the proposed federal law does not define “terminal illness,” the definitions used in various state laws are relatively consistent. For example:
- Illinois defines a terminal illness as “a disease that, without life-sustaining measures, can reasonably be expected to result in death in 24 months or less.”
- Florida defines a “terminal condition” as “a progressive disease or medical or surgical condition that causes significant functional impairment, is not considered by a treating physician to be reversible even with the administration of available treatment options currently approved by the United States Food and Drug Administration, and, without the administration of life-sustaining procedures, will result in death within 1 year after diagnosis if the condition runs its normal course.”
- Oregon defines “terminal illness” as “an illness or a medical or surgical condition that in a physician’s reasonable medical judgment will result in the patient’s death within six months.”
The effect of the protections of the new federal Act is to allow terminally ill patients to use these treatments without putting the physician or the drug manufacturer (or the manufacturer’s regulatory approval applications) at risk. By protecting patients, physicians and the drug sponsor from this risk, the hope is that patients who otherwise have no course of treatment or hope of survival will have available to them new courses of treatment to combat their terminal illness. Although the data collected from these terminally ill patients will be more idiosyncratic and not available for use to support a New Drug Application (as it will not replace the controlled clinical testing that the regulations require), it may be persuasive data which could be used to support the clinical trial outcomes. This data could over time lead to new uses and more research on these terminal illnesses.
If you have any questions about a state’s Right to Try laws, their impact on your company’s practices or the FDA treatment approval processes, please feel free to contact a member of our team. We have attorneys well-versed in the FDA approval process for various medical treatments, including working with hospitals on clinical trials and managing risks.