Hospital Pharmacy - July/August 2018 - 234

783992
research-article2018

HPXXXX10.1177/0018578718783992Hospital PharmacyGabay

Rx Legal

RxLegal: A Rapid Review of Right-To-Try

Hospital Pharmacy
2018, Vol. 53(4) 234-235
© The Author(s) 2018
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https://doi.org/10.1177/0018578718783992
DOI: 10.1177/0018578718783992
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Michael Gabay1

Abstract
Right-to-try legislation is intended to allow patients with life-threatening illnesses access to investigational medical treatments
without formal Food and Drug Administration (FDA) involvement. Currently, right-to-try laws have been enacted in 40
states. Despite the increased passage of right-to-try legislation at the state level, individuals have detailed arguments both
for and against these laws. Proponents state that right-to-try removes regulatory burdens and improves timely access to
potentially lifesaving medications for terminally ill patients, reduces inequalities regarding access, and improves patientprovider communication and decision making. Opponents argue that right-to-try does not really guarantee access, reinforces
preexisting health care inequalities, prioritizes rapid access over safety and the interests of the individual over the public, and
produces concerns regarding informed consent. Despite these issues, right-to-try has recently gained traction on the federal
level with both Congressional chambers passing separate bills.
Keywords
legal aspects, ethics, investigational drugs
Since 2014, there has been a surge in interest on both the
state and federal levels in "right-to-try" legislation. This legislation is intended to allow patients with life-threatening
illnesses access to investigational medical treatments without formal Food and Drug Administration (FDA) involvement.1 Currently, right-to-try laws have been enacted in 40
states.2 The main requirements of these state laws are fairly
similar as they are based on a model law drafted by the
Goldwater Institute (a libertarian public policy think tank).3,4
These requirements generally include that the patient has a
terminal diagnosis with no other treatment options available; the investigational agent is recommended by the
patient's health care provider; the treatment has successfully
completed phase 1 safety evaluations and is at least in early
phase 2 testing; and the patient or his or her guardian has
given informed consent.3
Even without passage of right-to-try legislation, patients
are able to access investigational treatments through enrollment in an ongoing clinical trial or via the FDA expanded
access program.1,5,6 However, some patients do not meet criteria for inclusion in a clinical trial or may reside too far
away from a trial site to realistically participate. In addition,
terminally ill patients may not want to risk enrolling in a
clinical trial where there is a 50% chance of assignment to a
control group instead of the desired investigational therapy.3
Accessing an investigational therapy through an expanded
access program, which attempts to balance rapid investigational agent access without interfering with the conduct of
ongoing clinical trials, requires approval by both the FDA

and an independent institutional review board (IRB).
Historically, this program has been time-consuming and
cumbersome for patients and providers; however, more
recently, the FDA has made strides in reducing the complexity of forms and the time to approval, particularly with regard
to emergency requests.
With the intensifying interest in right-to-try legislation,
individuals have detailed arguments both for and against
their enactment.3,7 Proponents of right-to-try state that these
laws remove regulatory burdens and improve timely access
to potentially lifesaving medications for terminally ill
patients, reduce inequalities regarding access, and improve
patient-provider communication and decision making as
more potential therapeutic options are available for terminal
patients. Opponents argue that right-to-try does not really
guarantee access, but only confers a "right to ask" the pharmaceutical manufacturer. The manufacturer may still decide
to deny access and many are inclined to due so for various
reasons such as potential negative publicity if a patient develops adverse effects or dies as a result of the investigational
agent. In addition, right-to-try may reinforce preexisting
inequalities as many laws result in patients losing health care
benefits (eg, patients who use an experimental treatment may
1

The University of Illinois at Chicago, USA

Corresponding Author:
Michael Gabay, College of Pharmacy, The University of Illinois at Chicago,
833 South Wood Street, Chicago, IL 60612, USA.
Email: mgabay@uic.edu
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