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FEATURE
The Realities of “Right to Try”
coordinator a full workweek to get everything in
place to apply for a drug through the FDA’s program.
“Most of that time is not spent interacting with the
FDA,” he explained, adding that “the FDA is quick to
make a decision. Most of the time is spent in writing
a protocol and an informed-consent document.”
Like any patient participating in a clinical trial,
patients requesting access to drugs through the
FDA’s program must provide informed consent
consistent with federal requirements. While the
text of the Right to Try Act of 2017 states that eli-
gible patients must provide the “treating physician
written informed consent regarding the eligible
investigational drug,” it offers little guidance as to
the content of this consent form. 1
“In the typical FDA procedures for informed
consent, physicians are required to describe the
potential risks and benefits, alternatives to the
investigational drug, and more,” Dr. Bateman-
House said. “All that is removed with ‘Right to Try’
[legislation]. There are no standards about what
these documents need to say.”
For therapies administered through the ”Right
to Try” pathway, it also is unclear who will explain
to patients that they may be responsible for the
costs of the drug, its administration, and any costs
incurred as a result of the investigational treatment.
According to Dr. Sekeres, many insurance
companies will typically pay for the “usual care”
associated with clinical-trial treatment, but it is
unclear whether insurance companies will pay for
treatment accessed under “Right to Try” legislation.
“The decision to try a non–FDA-approved drug
lies between a physician and patient,” a representa-
tive from UnitedHealthcare told ASH Clinical News
in a statement. “We provide access to thousands of
medications through our pharmacy programs and
support participation in qualified clinical trials to
help individuals get the proven treatments needed
to support their care.”
Untangling State and Federal Laws
Another aspect of implementation that Cleveland
Clinic and other hospitals may struggle with is nav-
igating differences between “Right to Try” legisla-
tion written at the state level and that written at the
federal level, Dr. Sekeres said. For example, “Right
to Try” legislation in Ohio (which became the 33rd
state to adopt its own law, H.B. 290, in 2017) is
32 pages and includes more specific requirements
on informed consent than the comparatively slim
four-page federal document. 10
“My understanding is that, in the past, we did
not have to follow state legislation, but now that the
federal law is in place, hospitals have to determine
which guidelines to follow,” Dr. Sekeres said.
Holly Fernandez Lynch, JD, MBE, the John
Russell Dickson, MD Presidential Assistant Profes-
sor of Medical Ethics and Health Policy at Perel-
man, agreed that the issue is quite complicated.
In the U.S. system of federalism, all the power
not granted to the federal government is reserved
to the states, she explained, but in areas where the
federal government has authority, it preempts state
law.
“In general, if the federal government has total
authority over an area, states are precluded from
regulating it – this is called ‘field preemption,’” Ms.
Lynch said. “Sometimes, though, federal regula-
tion is treated as the ‘floor’ rather than the ‘ceiling,’
meaning that states are free to regulate in the same
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ASH Clinical News
space, as long as what they require does not conflict
with federal law.”
If state lawmakers enact a law that conflicts
with federal statutes, “it would be preempted as a
matter of ‘conflict preemption.’ In this case, states
can impose more stringent requirements than
federal law, but not less.”
“I don’t know why
any company that
is committed to
gathering evidence
to support a claim
of efficacy ... would
go the ‘Right to Try’
route.”
—STEVEN JOFFE, MD, MPH
In the case of “Right to Try” laws initially
passed by states in attempting to circumvent the
FDA, state law would have been preempted by
federal law. If any patient received an unapproved
therapy under a state “Right to Try” law, state laws
were in direct conflict with FDA requirements, Ms.
Lynch explained.
The passage of the Right to Try Act of 2017
complicates matters further. Ms. Lynch laid out
two scenarios: In one, the federal and state laws
might not conflict, and there would be no issue.
In the other, the federal “Right to Try” law could
create “field preemption,” when Congress, without
expressly declaring that state laws are preempted,
legislates as to “occupy” the entire field of an issue.
In the first scenario, state “Right to Try” laws
could be allowed to stand if they are more re-
strictive than what the federal law requires (for
example, stricter eligibility criteria or more detailed
informed-consent requirements) or if they cover
different areas not touched by the federal law (for
example, how accessing an investigational drug
affects patient’s eligibility for insurance coverage or
hospice).
“Ultimately, it is unclear how to view the state
‘Right to Try’ laws,” Ms. Lynch said, “and the ques-
tion of preemption will likely need to be resolved
by the courts.”
Until then, she offered the following advice for
physicians unclear about the federal and state laws:
If they want to provide access through the “Right to
Try” pathway, compliance with federal law is most
important.
“Better yet is not to provide access through the
‘Right to Try’ pathway at all because it is dangerous
for patients,” she added. “Expanded access should
be the preferred approach.”
The Buck Stops Here
Regardless of the approach a patient and physician
choose, decisions about accessing investigational
drugs rest with the pharmaceutical companies.
“I don’t know why any company that is com-
mitted to gathering evidence to support a claim of
efficacy, that knows it can use the expanded-access
pathway, and that works collaboratively with the
FDA would go the ‘Right to Try’ route,” Dr. Joffe
said. “I do not see any positive benefit.”
Companies that choose to grant access to their
experimental drugs through the “Right