Access to Unapproved Medicine:
The regulations also define the
different pathways that are available,
including:
Single Patients: A single-patient
pathway is the process whereby a
doctor can ask a drug manufacturer
if they would be willing to supply
their drug for a patient in need
even if it is not available in the
United States or is still being
studied in clinical trials. If the
drug company agrees,
they will provide
the doctor with
any needed information
required
by the FDA.
The doctor must request approval
from the FDA to treat
the patient and FDA has 30
days to respond to this request.
If FDA approves, the
doctor still must periodically
request approval from FDA
for re-supply of the drug.
There are also provisions for
urgent requests that are made
over the telephone with the FDA,
and approval can be granted same
day or within 24 hours. The drug
manufacturers approval is required
prior to submitting all requests
to the FDA, even emergency
requests.
Groups of Patients: The FDA established
specific criteria in which
a drug company can identify a
group of patients that it wishes to
allow access to its drug. An example
could be when a drug is not
being developed because the disease
is so rare, impeding a company
s ability to recruit enough patients
for clinical trials. In the
group scenario, the drug company
establishes written criteria (called
treatment protocol) that clearly
defines the exact population that
they will allow access to the medicine.
A doctor can then enroll
appropriate patients in the program
and receive access to the
medicine until the drug becomes
commercially available or for extended
periods (potentially years)
if the drug company is not planning
to commercialize.