Sometimes called "compassionate use,"
Expanded Access is a way for patients to get access
to an investigational medical product.
Investigational medical products are drugs,
biologics or medical devices that are still
being tested to see whether they are safe and
effective.
This means that FDA has not concluded that these
products are safe for their intended use.
In addition, products may or may not be effective in
the treatment of the disease or condition.
Using an investigational medical product may cause
serious side effects that are not expected.
Cooper's been diagnosed with neurofibromatosis,
type 1, which is a genetic condition that causes
tumors to grow on nerves throughout the body.
And there's also other manifestations of NF, such
as tibial bowing and learning issues.
So Cooper's primary issue is the tumors in his body
Because there were no current approved
treatments, Cooper's geneticists let us know
that there was a possibility of access to
experimental medication through the FDA's Expanded
Access Program.
I have a tumor on my neck.
And it's growing and growing and growing and
growing and growing.
And the more it grows, if it grew anymore, I would
have less air.
Patients are encouraged to participate in clinical
trials to help learn whether investigational
medical products are safe and effective.
However, sometimes it's not possible for a patient
to be a part of a clinical trial.
For example, when there are no available current
clinical trials; when a patient may not fit the
selection criteria for the clinical trial; or when a
patient lives too far from a clinical trial location.
Patients or caregivers who have questions about
participating in a clinical trial should
first contact the person who is running the
clinical trial, also known as the clinical
investigator.
FDA's Expanded Access Program might be an option
for a patient to try an investigational medical
product.
A patient may fit the criteria for FDA's
Expanded Access program if the patient has a serious
or immediately life-threatening disease
or condition; if there is no satisfactory or
comparable alternative therapy to diagnose,
monitor, or treat the disease or condition; if
participating in a clinical trial is not
possible; if the possible benefit to the patient is
reasonable compared to the possible risk of
treatment; and if providing the patient with
the investigational medical product under
Expanded Access will not slow down or hold up the
clinical trial process for the product.
I would suggest that you do your research, try to
learn more than you think you should need to know,
be an advocate.
Really find both medical professionals that you can
partner with that are willing to go through the
process to gain access to the medication you need
for yourself, for your child.
And also connect with other patients maybe
who've been through the process.
I think something that parents have to know,
especially parents of children who have a rare
disease or a rare illness, is that a lot of times you
feel like you know more than the medical
providers.
And then sometimes that's the case.
But I think it's important to remember, especially
if you've done your research, to remember that
nobody knows your child better than you do.
And so the medical providers are the experts
on the condition, but they don't really know your
child in the same way.
And so it's really good to partner with them so that
those two bodies of knowledge can come
together.
Our doctors have to work really hard to do that, so
try to be helpful to them and try to give them
extra kindness.
There are 5 steps in the process for requesting an
investigational medical product under Expanded
Access.
First, you should talk to a licensed physician
before deciding if an investigational medical
product is right for you.
A licensed physician can review your medical
history and help you consider all possible
risks and benefits.
Second, ask your licensed physician to contact the
medical product company to request the
investigational medical product through Expanded
Access.
The medical product company decides whether
they are able to provide the investigational
medical product for treatment under Expanded
Access.
A medical product company is not
their product under Expanded Access and there
may be reasons a company is unable to supply it -
for example, a limited supply needed for clinical
trials.
Medical product companies are required by law to
publicly post their general Expanded Access
policies once they reach a certain stage of
development.
Third, an Institutional Review Board, also called
an IRB, reviews the Expanded Access treatment
plan and informed consent document.
The IRB makes sure that the patient will be able
to understand the investigational medical
product for the planned treatment as well as
possible risks and benefits.
An IRB is an independent third-party group who
protects the rights and welfare of the person
involved in scientific research.
Fourth, have your licensed physician visit "FDA's
Expanded Access: Information for
Physicians" webpage.
The physician will need to complete an Expanded
Access request form on your behalf and submit it
to FDA.
FDA reviews and determines if the Expanded Access
request may proceed.
Historically, FDA has allowed the majority of
Expanded Access requests to proceed.
And fifth, once your Expanded Access request is
determined safe to proceed by the licensed physician,
the medical product company, the IRB, and the
FDA, you can begin treatment.
Read the informed consent carefully, ask your
licensed physician questions you may have
about the treatment, and make sure that you
understand the potential risks associated with the
investigational medical product.
You may stop your participation in the
Expanded Access program at any time, even after
signing the informed consent.
The costs of your treatment with the
investigational medical product might not be
covered by third-party payers, such as private
insurance or Medicare.
You may be expected to pay out of pocket for some or
all of your treatment with the investigational
medical product.
It's important to note that even if you meet the
criteria for Expanded Access, the licensed
physician, the medical product company, the IRB,
and the FDA must all agree that treatment with the
investigational medical product under the Expanded
Access program is appropriate.
FDA is committed to increasing awareness about
the Expanded Access process and the procedures
for getting access to investigational medical
products.
If you have any questions about FDA's Expanded
Access Program, please contact FDA's Patient
Affairs Staff at