TOPICS
Filing
an Application for Compassionate Use |
Commentary on Expanded Access
Also
see
Canadian Access
Program | FDA
background on Expanded Access
Compassionate Use of Investigational
Therapies
for trial- ineligible patients
 Expanded
access trials for Lymphoma
NEWS
Oct 2009: FDA
Final rule for Expanded
Access (PDF - 603KB) AND
Charging for Investigational Drugs (PDF - 394KB)
Jun 2010: ASCO: Oncologists, FDA Team Up to Ease Access to
Novel Drugs
http://www.medpagetoday.com/MeetingCoverage/ASCO/20482
OVERVIEW
-
The requested treatment protocol must be
considered reasonable by your oncologist and you must be ineligible
or unable to participate in a clinical trial that is testing the
protocol.
The protocol must be deemed reasonable for your treatment
circumstance, relative to available treatment options.
* Potential for efficacy and sufficient data on safety.
* Preliminary evidence (clinical or plausible pharmacological of
activity against the disease)
* Probable risk of the treatment does not exceed that from disease.
If you are making the proposal, first do your homework, then
write to your doctor and make the case, concisely.
NOTE: Safety issues are not always published and available to the
patient or the doctor, leading to unrealistic expectations.
Efficacy can be overstated in early reports.)
-
Your doctor must then contact the sponsor
of the investigational agent, who must agree to
provide the drug - they may not be able or willing.
This step can be a significant obstacle. The drug sponsor has
little to gain and possible liabilities (financial, insurance,
legal) when providing investigational drugs outside the structure and
close supervision of a clinical trial.
"When
a physician would like to request an Investigational New Drug (IND)
application to use an unapproved drug or other product for a single
patient, the first step is to obtain permission from the manufacturer.
Without the consent of the manufacturer, the unapproved product will not
be available to the patient. After the manufacturer agrees to provide
the product, the recommended procedure is to submit the following
information to the appropriate review division. ... "
FDA.gov
-
Your doctor completes forms for FDA review &
consent as described in the checklist below. The FDA will probably
not stand in the way if the requested protocol is reasonable,
relative to other available treatment options.
The rate limiting
circumstance might depend on how your doctor communicates the
urgency.
-
Finally, your doctor must obtain IRB
(Institutional Review Board) approval and draw up a consent form. This
step is easier if your doctor is also a clinical investigator and is
familiar with the process.
-
Your doctor will be
responsible for monitoring, and reporting and oversight. Cost
to physician might not be compensated. Doctor
might have to assume some liability.
Compassionate
Use Checklist
|
x
|
Manufacturer has committed to
supplying the investigational drug
|
x
|
Letter of authorization from
manufacturer included or submitted separately
|
x
|
Form FDA 1571 completed and signed (Investigational New Drug
Application IND) PDF
|
x
|
Form FDA 1572 completed and signed (Statement of Investigator)
PDF
|
| |
for forms 1571 & 1572 (above)
|
x
|
CV (first two pages) including
documentation of medical licensure Informed consent form completed and signed
|
x
|
Commitment to Institutional
Review Board (IRB) approval prior to initiating administration of the investigational drug
|
x
|
Narrative: Medical history,
disease history, prior treatments, response Patient's current medical status
|
x
|
Patient or
physician's prior experience with the investigational treatment to be administered (if
any), rationale for administration of the investigational drug. Proposal for treatment of
the patient, including plan for concomitant treatments, administration of the
investigational drug, and monitoring of the patient.
|
Expanded Access (EA) and Compassionate Use (CU) -
Commentary
I feel that we have a moral responsibility
in some instances (when the patient is willing and benefit is feasible) to try investigational agents when normal channels have failed or when standard approaches
have not demonstrated benefit.
But, the patient and their loved ones should be informed if the
Compassionate Use protocol is likely to increase pain and suffering, and also how likely it is to
help.
GAPS: We need to direct the NCI to fill in the many gaps of our economic-driven system, and conduct more clinical investigations,
such as testing first line use of lower toxic immunotherapies as an
alternative to observation (expectant management).
- Karl S
|
