20 JUNE 2014 VOL 344 ISSUE 6190 1329 SCIENCE sciencemag.

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By Kelly Servick
S
everal state legislatures, prodded by
desperately ill patients and a liber-
tarian think tank, are defying federal
regulators to make experimental
drugs available outside of clinical
trials. The new laws, called Right to
Try, allow terminally ill patients to request
unapproved drugs after early-stage test-
ing. Last month, Right to Try went on the
books in Colorado and Louisiana; a similar
law awaits the governors signature in Mis-
souri, and Arizona will put the issue to a
vote in November.
The movement taps into long-standing
frustration over what some view as the
sluggish pace of clinical trials. Although
the laws are framed as a boon for dying
patients, critics raise a long list of caveats.
Companies, they say, will often be reluc-
tant to offer a drug outside of its clinical
trialsand the untested therapies will rarely
work. Only about one in 10 drugs in phase
I clinical trials ever demonstrate efficacy to
win Food and Drug Administration (FDA)
approval. It sounds like, Were throwing
you a life preserver, and youll have your shot
at being cured, says Arthur Caplan, a medi-
cal ethicist at New York Universitys Langone
Medical Center in New York City. I cant tell
you how rare that scenario is.
Rare it may be, supporters say, but not
impossible. Any patients alive today with
ALS will most likely be dead by the time
were finished with our trials, says I. Rich-
ard Garr, president of Neuralstem Inc. in
Germantown, Maryland, whose stem cell
treatment for the fatal neurodegenerative
disease is in phase II clinical trials. His
company plans to take advantage of Colo-
rados Right to Try law to make its unap-
proved treatment available to ALS patients,
well ahead of any FDA decision.
Right to Try laws are being spearheaded
by the Goldwater Institute, a libertarian
group based in Phoenix. The laws require
that patients be terminally ill, have con-
sidered other available treatment options,
and be unable to participate in a clinical
trial. The institute describes the laws as a
complement to an existing expanded ac-
cess program at FDA, which allows patients
with serious or life-threatening diseases to
try experimental drugs if theyve exhausted
their other options. But with expanded ac-
cess, an institutional review board must
also approve each request, and the patients
doctor must report follow-up data to FDA.
According to FDAs submission receipts
report for fiscal year 2013, the agency
granted all but three of nearly 1000 ex-
panded access requests it received. When
patients cant access experimental treat-
ments, the holdup is often the drug de-
veloper, not FDA, Caplan says. Companies
cannot be forced to supply an experimental
treatment outside a clinical trial, and often
they dont wish to do so. Manufacturing
more of a drug can strain a companys re-
sources, says Emil Kakkis, CEO of the bio-
tech company Ultragenyx Pharmaceutical
Inc. in Novato, California, and president
of the EveryLife Foundation for Rare Dis-
eases, which promotes innovation for rare
disease treatments.
Companies also worry that a bad out-
come from FDAs expanded access will
torpedo a therapy, even though the agency
maintains that this has never happened.
The fervor over Right to Try has some
patient advocates worried that existing
options for dying patients will be over-
looked. A one-person expanded access
request through FDA is an uphill battle,
but the system can accommodate larger
groups, says Jess Rabourn, director of the
ALS Emergency Treatment Fund in San
Jose, California, which helps biotech com-
panies apply for and fund expanded access
programs.
Legally, Right to Try raises major ques-
tions. Federal law limits moving drugs or
their components across state lines with-
out first applying to FDA, notes Meredith
Manning, a lawyer specializing in the FDA
approval process at Hogan Lovells in Wash-
ington, D.C. If FDA were to assert that
the federal law takes precedence, it would
have a good argument, she believes. So
far, FDA has not taken a public position on
Right to Try, and agency officials declined
to comment on the laws for this story.
I dont think that patient populations
and foundations and advocates should be
duped into thinking that this is something
that its not, Rabourn says. Its not some-
thing that changes the federal law, and
thats the law of the land that governs drug
development. Its what any shareholder-
owned drug company has to abide by.
Garr says that if FDA forbids the com-
pany from offering its unapproved treat-
ment, an injection of neural stem cells
into a patients spinal cord, he ll follow the
rules. Still, Garr, who accepted an invita-
tion to join the Goldwater Institutes Right
to Try National Advisory Council, says he
believes in offering the treatment to pa-
tients who want it. The inclusion criteria
will be almost identical to those of the on-
going trial: Patients must be in the early
stages of ALS. So far, no other company has
said it will follow Neuralstems lead.
Meanwhile, Garr says, were already
getting tremendous numbers of people
writing, saying theyre ready to move to
Colorado tomorrow.
Colorado Governor
John Hickenlooper
signed the first Right
to Try law in May.
Right to Try laws bypass FDA
for last-ditch treatments
But companies may be reluctant to supply unapproved drugs
PATIENT ADVOCACY
Published by AAAS

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