form system for terminal patients
seeking access to investigational
treatments.
Other Options to
Obtain Access
In addition to FDA Expanded Access
Programs, patients who meet
certain criteria may have the option
to import the needed drug
directly from another country;
this is known as personal importation
of medicines. This would involve
the patient, the caregiver, or
the physician finding a source of
the drug, such as the manufacturer
or wholesaler outside of the
United States that is willing to
sell the drug to the patient. The
FDA does not have stated regulations
for personal importation of
these medicines; rather, it provides
guidance on how this can be
done for non-FDA approved
drugs. (Importing FDAunapproved
drugs is illegal. However,
under certain circumstances,
the FDA will use its regulatory
discretion to allow the drug to
enter the United States for treatment
of a patient.)
Cheryl Sacks, a rare disease patient
with Fabry disease, turned to
the FDA when her health started
42—iPain Living Magazine
declining severely. Although she
was taking Genzyme’s Fabrazyme
® at the time, her doctor felt
that her health was not improving.
Therefore, they sought out Shire
Pharmaceutical’s competing drug
Replagal®, which was approved
in Europe but not in the United
States.
“The FDA was critical to my success
in getting Replagal®,” says
Sacks. “My contact there helped
to facilitate the process of shepherding
my medicine through to
me.”
There is no direct approval required
from the FDA, but working
closely with its representatives
to obtain specific instruction
on bringing the medicine into the
U.S. is critical so that there are no
delays when the medicine arrives
at the border (as these shipments
will need to be cleared by customs
officials).
Additionally, "there is no regulatory
infrastructure spelling out
just how patients and physicians
should go about accessing investigational
drugs or how drug companies
should respond." Harriet
Hall, MD expressed concerns that
patients may not completely comprehend
the risks involved in taking
medications available under
the right-to-try law, nor understand
the low probability of success,
especially patients who were
not healthy enough to qualify to
participate in clinical trials. She
states these patients may have
other medical conditions that
could make them more vulnerable
to complications from experimental
treatments.
Ultimately the decision to take
unapproved medicines should be
made in close consultation with
your physician, and it is important
to remember that there is always a
chance that it may not work or it
may have serious side effects.
To get in touch with an FDA representative
and make sure no delays
occur when your medicine
arrives, please reach out to the
FDA Office of Health and Constituent
Affairs
(www.fda.gov/AboutFDA/
CentersOffices/OC/
OfficeofExternalAffairs/
ucm343095.htm?source=govdelivery).
This article was adapted and updated
from the original article; Access to Un-
Approved Medicine: is this An option for
Me? (For USA) by Global Genes.
(Connued from page 41)