The Right-to-Try (RTT) law, which has been in effect since May 2018, may have a misleading name, given that it doesn’t require drug manufacturers to grant access to experimental treatments that have passed phase I trials to any patient who seeks it. Rather, the law grants terminally ill patients the “right to ask” the drug company directly, but the request can be denied for several reasons (e.g., limited supply, expense).

Compassionate Use

The U.S. Food and Drug Administration’s (FDA’s) Expanded Access program, also known as compassionate use, already allows terminally ill patients to access investigational treatments outside of clinical trials and in fact approved 99.7% of applications in 2017. The program was streamlined in 2016 to accelerate the typical response time to a few days, sometimes only a few hours in the case of emergencies. If a treatment is approved, FDA provides guidance on dosing, managing side effects, and administration. This is crucial for providers and nurses who would not have clinical experience with the investigational drug. Insurance companies are also required to cover the cost of drugs obtained via the compassionate use program and any associated costs of care.

If the expanded access program exists and seems to be working well, why create a new law? Some supporters say that RTT removes the red tape with applying for compassionate use and places the patient more in control of their care. RTT is touted to be easier and less time consuming, and it allows terminally ill patients quicker access to these treatments because FDA oversight has been removed. Senator Ron Johnson (R-WI), a major proponent of the bill, argued the decision of whether a dying patient receives investigational agent shouldn’t be left up to the government, but to the patient themselves. However, under RTT, the gatekeeper ultimately becomes the drug company, and this raises concern for many.

Facts About Right to Try

This is the first federal RTT law; 39 states already have legislation in place.
Treatments must be under an active investigational new drug application and have completed phase I trials.
Drug manufacturers are not required to provide the requested drug.
Insurance companies are not required to cover any costs.
FDA will not provide guidance related to administration, side effects, or dosing; providers can seek input from the manufacturer, but they are not required to provide any guidance.
Physicians are not required to assist patients in seeking access to investigational drugs via RTT; however, the application must include a physician certifying the patient has “exhausted approved treatment options and is unable to participate in a clinical trial involving the investigational drug.”
Providers and drug makers have limited liability related to the use of investigational treatments.
FDA cannot use clinical outcomes from investigational treatments in its regular review process.
It limits the cost a patient can be charged for the investigational treatment; however, companies are not required to disclose what is provided to individual patient nor the costs.

Note. Based on information from ASCO (2018), Dooley Young (2018), and Kearns & Bateman-House (2017).

But Is It Safe?

Many experts point out that RTT bypasses the built-in patient safeguards under expanded access (i.e., institutional review boards to ensure the patient understands the risks and benefits, guidance on administration, dosing, and side effects). In addition, it does not provide for oversight of how, where, or who administers a medication, and it removes some of the checks and balances put in place to ensure safety. Some believe this makes it riskier for patients and drug manufacturers. In addition, the short turnaround time and approval rate of applications for compassionate use begs the question of whether RTT will really improve access or if the bill is trying to fix a problem that doesn’t exist.

Seventy-seven organizations have voiced opposition to the law, including ASCO, National Comprehensive Cancer Network, and American Cancer Society Cancer Action Network. Some drug manufacturers are citing concerns about the potential impact to ongoing clinical trials such as reduced supply of drugs and affected data collection, ultimately slowing the process of bringing new, safe, effective medications to more patients. FDA’s associate commissioner for public health strategy, Peter Lurie, MD, MPH, highlighted concerns, including safety issues and the ability to maintain integrity of the clinical trial process.

What This Means for APRNs

Because RTT has been signed into federal law and is garnering media attention, patients may ask their providers about RTT. More terminally ill patients may choose to seek investigational agents, feeling as though they’ve been granted new access to promising drugs. However, APRNs must convey that the drugs have only been tested in phase I trials and may not be effective or could cause more harm.

APRNs should be prepared with knowledge of the law, its requirements for those wanting to apply for investigational treatment, and the potential risks. As with any treatment or procedure, keeping patients and families well informed of the risks and benefits of pursuing an investigational drug is critical. Understanding the difference between RTT and the FDA compassionate use program is also important. Some patients may certainly be candidates for RTT; however, a thorough conversation about their options is prudent. ASCO has developed several resources for providers to use in those discussions.

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