Pharmaceutical Commerce - September/October 2017 - 11

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shaming a drug company into providing
investigational drugs. Under current FDA
guidance, a drug manufacturer can prepare,
in a fashion, for multiple expanded access
requests by including an expanded-access
protocol in its FDA regulatory filings. (In
this case, the manufacturer itself becomes
the requester of expanded access.)
A key part of S.204 tries to clarify what
FDA can do with outcomes from a patient's
EAP. The relevant clause reads:

Navigator
Another action that FDA had taken in
2016 was to authorize the Reagan-Udall
Foundation, a nonprofit public group
set up by Congress to backstop a variety
of FDA initiatives, to design an Expanded
Access Navigator to help patients and
their physicians in preparing applications.
That site has already been "soft launched"
according to a Foundation spokesperson.

As of mid-summer, it includes a list of the
31 pharma companies who have already
written (and informed FDA) of their
expanded access selection criteria.
In abstract, right-to-tr y laws are
an assault on the very essence of FDA:
keeping scientifically unproven drugs
off the market, to protect patients and
steer healthcare dollars (no matter who
pays them) to useful purposes. Shutting

down FDA's drug approval process nearly
completely has been an idea kicked around
in libertarian and far-right circles for
years; even now, the accelerated approval
mechanisms FDA currently employs are
being watched for signs that the approval
is being short-circuited. It could be that if
Commissioner Gottlieb's goal to speed up
reviews and approvals pays off, the right-totry movement will be mollified.

"[HHS] Secretary may not use a clinical
outcome associated with the use of an
eligible investigational drug pursuant to this
section to delay or adversely affect the review
or approval of such drug ... unless-
"(A) the Secretary makes a
determination, in accordance w ith
paragraph (2), that use of such clinical
outcome is critical to determining the safety
of the eligible investigational drug; or
"(B) the sponsor requests use of such
outcomes."
All of which puts the question of
positive or negative outcomes from an
EAP application back in the hands of
FDA. Opponents of wide-open EAPs have
argued that it's bizarre not to make use
of outcome data wherever it's from in the
drug-approval process; EAP proponents
like the Goldwater Institute have argued
that it's immoral for the federal government
to withhold a treatment from a patient who
might benefit.
Jane Reese-Coulbour ne, a senior
consultant at MK&A, an industr y
consulting firm specializing in patient
and healthcare stakeholder engagement
strategies that include EAPs, notes that
expanded access has, in the past, sometimes
been very beneficial to a drug approval,
citing the instance of Iressa (gefitinib),
the Astra-Zeneca drug for lung cancer; an
unusually broad EAP involving thousands
of patients answered cer tain safet y
questions when the drug was approved
in the US in 2003. "Companies now have
an obligation to develop EAPs, although
FDA is not enforcing this aggressively," she
says. "In any case, there is an opportunity
for companies to develop these programs
thoughtfully, taking into consideration
issues like availability of the drug and
patient selection criteria."
And while there is earlier regulatory
action on how an expanded-access drug
is to be priced, economics factor into the
equation. A leader in stem cell research is
of the opinion that "There are scores of
companies in the United States who want to
sell unproven and scientifically implausible
stem cell therapies to patients, some of
whom are among the main supporters of
right-to-try legislation." Coming at the
subject from a very different direction,
at least one company, Inceptua, was
recently founded to make a business from
connecting patients with expanded access
drugs.
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Pharmaceutical Commerce - September/October 2017 - Cover1
Pharmaceutical Commerce - September/October 2017 - Cover2
Pharmaceutical Commerce - September/October 2017 - Table of Contents
Pharmaceutical Commerce - September/October 2017 - 4
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Pharmaceutical Commerce - September/October 2017 - Cover3
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