If you have a rare disease
or are a caregiver or
patient advocate for
someone who does, you can
play a vital role in
advancing potential
treatments.
How?
By participating in
Natural History Studies
and Clinical Trials.
Let me explain.
Natural History Studies
are often a first step to
help researchers
understand different
characteristics
of rare diseases.
In these studies,
researchers follow
participants who are
at risk for or have a
specific disease or
condition to determine how
specific factors, such
as family history, age,
workplace and
environmental exposure
could be related to the
disease process, how the
disease changes over time
and possibly worsens, and
how it affects a patient's
overall health and quality
of life.
When you participate in
a natural history study,
you're giving a gift to
yourself, but also a gift
to everyone else that is
struggling or fighting or
advocating for the disease
that you have, because
you're contributing to the
science that makes future
treatments and future
cures possible.
Participation in a natural
history study is really a
win- win-win proposition,
but especially a win for
the patient.
This is the way the
patient goes to our best
clinical sites, receives
our best clinical care
that's available,
receives all the
information that they
can use on the latest in
research.
And this is the way the
patient feels, "Finally,
I'm no longer helpless.
I'm no longer hopeless.
I have confidence that I
can help, that I can make
a difference in
curing this disease".
The information obtained
by patients through
Natural History Studies
can give researchers more
details about the disease,
trigger new ideas on how
to best treat patients,
and ultimately be used to
design clinical trials
that test new treatment
options and can bring
medical products to
market.
Clinical Trials study how
safe and helpful tests and
treatments are.
They help researchers
answer specific scientific
questions related to
diagnosing, preventing or
treating diseases
and conditions.
 FDA uses the information
gained from clinical
trials to better
understand the benefits
and risks of the therapies
to decide if a therapy
should be approved and
made available for
companies to market them.
Without well-designed and
well-run clinical trials,
the true effects of a
therapy could go unknown
and may expose people to
more harm than benefit.
Patients deserve to
have access to not only
accepted treatments, but
the possibility of hope
from new treatments.
Clinical trials give them
the possibility of trying
medications that are not
available to all patients
yet.
It sometimes gives them
hope for the future, for
maybe a longer life, or
the hope of actually
giving information that
can care for their
families later
in the future.
Before joining a clinical
trial, it's important to
learn as much as possible
about the study.
Patients should discuss
the clinical study with
their doctor to determine
if it's a good option for
them and then share their
questions and concerns
with their doctor and the
researchers conducting the
clinical trial.
FDA realizes the
importance of both natural
history studies and
clinical trials to speed
development of new, safe
and effective medical
products for rare
diseases and conditions.
That's why FDA's Office
of Orphan Products
Development, Orphan
Products Grants Program
continues to support
innovative research by
funding both natural
history studies and
clinical trials
in rare diseases.
If you're thinking about
participating in a natural
history study or clinical
trial, your doctor is a
good resource to help you
find studies that might be
right for you .
