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