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
~ Use of Investigational New Drugs or Protocols
for trial ineligible patients ~
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 make sense in your treatment
circumstance, relative to available treatment options.
If you are making the proposal, first do your homework, then
write to your doctor and make the case, concisely.
-
Your doctor must then contact the sponsor
of the investigational agent, who must agree to
provide it.
This step is often the main obstacle. The drug sponsor has
little to gain and possible liabilities 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
Also see: Proposed Rules for Charging for Investigational Drugs and
Expanded Access to Investigational Drugs for Treatment Use - 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.
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
| PDF-Help
|
x
|
Form FDA 1572 completed and signed (Statement of Investigator) - PDF
| PDF-Help
|
| |
Instructions
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
We have a moral responsibility 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.
PROPOSAL: Create incentives for the collaboration between the industry and the NCI to set up
Expanded Access and Compassionate Use programs for new drugs judged to have a potential to help patients in end stage disease.
Appropriate Compassionate Use clinical trial protocols should be waiting to receive patients in advance of the individual emergencies that happen again and again.
INDOLENT CANCERS: Patients with indolent lymphomas often defer treatment until they become symptomatic, which alone speaks to the inadequacies of available treatments and the need for
Compassionate Use and Expanded Access programs for this group.
MISSED OPPORTUNITIES: We need to encourage frontline studies, or
Expanded Access programs to test emerging biotherapies – and combinations of biotherapies
in patients in watchful waiting status, that do not always include chemotherapies and can take advantage of the better immune status of this group.
We need to allow for Compassionate Use or
Expanded Access of investigational biotherapies in trial ineligible patients - even in non-“emergencies” when standard therapies are significantly more toxic then the investigational agent, and the standard approaches are not curative and also deemed more likely to preclude the use of subsequent therapies.
GAPS: We need to direct the NCI to fill in the many gaps of our economic-driven system, and conduct more clinical investigations testing the
first line use of low toxic immunotherapies.
For example, it is disturbing that the selected path to approval is
almost exclusively to test new drugs in patients refractory to
treatment.
REWARDS: Making Compassionate Use & Expanded
Access clinical trial protocols ready to receive patients in a timely manner is also likely to produce more meaningful data.
This will help to advance the clinical science while providing a humane service to
patients in need of emerging therapies that show promise when ineligible to
participate in clinical trials.
- Karl Schwartz
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