Gaucher Community News - National Gaucher Foundation

Bringing Hope to People with Gaucher isease
Submitted by Genzyme
When it comes to helping patients with rare genetic
diseases, discovering and developing effective treatment
is just the beginning. Once a treatment is fully developed
and the necessary clinical trials have been completed,
regulatory agencies such as the Food and Drug
Administration (FDA) must review all of the information
about the safety and efficacy of the drug and approve it
for use. Then, depending on the health care system in the
country, an agreement to pay for the cost of the medicine
must be reached, usually through either a governmentsponsored
healthcare plan or private insurers. The more
uncommon a disease is, the higher the treatment cost for
a single patient usually is, which means that patients with
rare diseases may not be able to afford treatment on their
own.
In countries with an advanced healthcare infrastructure,
a means is usually found to pay for most drugs that have
been shown to be safe and effective. However in socalled
“emerging markets” – nations whose economy or
healthcare infrastructure are still developing – it may be
that no one can afford to pay for the cost of treatment or it
is difficult to get the necessary insurance.
Genzyme introduced the first treatment approved for
Gaucher disease in 1991. From the beginning, Genzyme
leaders knew that just developing ground-breaking
therapies would not be enough to treat the people in the
world affected by Gaucher disease. And that was exactly
what our goal was – to help all people with Gaucher have
access to safe and effective treatment.
In the early days, treatments were available only in the
United States and some European countries. Eventually
we opened offices in more than 40 countries around the
world. In each country, we work closely with regulators,
health administrators, insurers, clinical experts and patient
organizations to gain approval and pay for the cost of
therapy.
From the early days, we knew there would be Gaucher
patients who could not afford treatment, and we looked
for ways to make sure these patients could be treated.
Eventually it became clear that we needed a large-scale,
global program to improve patients’ access. In answer
to this need we developed the Genzyme Humanitarian
Program. This program provides patients with Gaucher,
Fabry, Pompe, and MPS I diseases with Genzyme products
free of charge. Genzyme distributes the product through a
large international infrastructure.
The Humanitarian Program is guided by an expert
medical committee who makes patient treatment
decisions, monitors and reviews patient progress, and
advises local treating physicians regarding patient care
as well as a board that recommends and approves those
requests.
In designing the Humanitarian Program, Genzyme
knew that we needed to create something that we
could support for the long term. We knew we could not
provide free drug to every patient and still keep our
business going. Therefore we have worked carefully
to set limits and priorities for the Program that allow
us to help patients over the long term. To do this, the
Genzyme Humanitarian Program does more than
provide free drug to patients. It works with physicians,
patient advocates, and government organizations to
increase understanding of rare genetic diseases and
to build sustainable healthcare systems in developing
countries. Currently more than 700 patients in countries
across the globe benefit from the program.
Genzyme’s commitment to our Humanitarian Program
has always been unwavering. During the supply shortage
one of the first decisions that we made was to disregard
charitable status when allocating drug. This means that
patients who receive treatment through our charitable
access programs were allocated drug in the same way as
other patients.
We still have a long way to go in our mission to make
sure that all people with Gaucher have access to a safe
and effective treatment. Our supply problems interrupted
access to treatment for patients around the world. And
there are still people with Gaucher disease whose illness
goes undiagnosed, who don’t even know that treatment
is possible.
We are working hard to overcome these challenges.
We have new manufacturing facilities coming online
that will greatly expand our capacity to make protein
therapies. We work with the Gaucher Registry to
expand awareness of Gaucher disease so that more
physicians can recognize the symptoms of Gaucher.
And we continue to expand our operations into more
countries, working in each one to develop a healthcare
infrastructure that will allow full and sustainable access
to treatment for all.
For more information, consult your physician. You can
also contact Genzyme at 1-800-745-4447 (option 2).
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