DISCOVERING AND
TREATING RARE
DISEASES
Genzyme’s sense of responsibility is grounded in our
commitment to patients – and in particular to those patients
who have few other advocates and are living in isolation.
Our company was founded to address the needs of
patients with rare diseases who previously had no
available treatment options, and we remain committed to
advancing new therapies for unmet medical needs.
Genzyme is committed to the ongoing understanding of the
diseases. Our patient focus is about much more than
developing and delivering products. We also take
responsibility for helping patients obtain access to our
unique therapies, regardless of their location, financial
circumstances or other obstacles. We establish free drug
programs, help build sustainable healthcare systems,
participate in humanitarian initiatives, partner with
advocacy groups on disease awareness and patient
support, and provide a broad range of support services, all
on behalf of our number one priority: patients.
Genzyme’s access to healthcare program is multi-faceted,
focusing on working with national health services,
government agencies and private insurers to establish or
improve patient access and coverage for our products.
The current rare diseases commercially covered by
Genzyme include Thyroid Cancer, Hypercholesterolemia,
Gaucher disease, Fabry disease, Pompe disease and
MPS-1 disease.
Genzyme’s worldwide operations serve patients in more
than 100 countries, with a global infrastructure that
includes research and development, manufacturing,
regulatory affairs, medical affairs, and more. This network
of expertise, plus our on-the-ground relationships with local
stakeholders, enables us to bring products to patients in
every corner of the globe.
Genzyme works closely with governments to facilitate
approval of our treatments. In countries with established
healthcare systems, we work with public and private
insurers to secure coverage and reimbursement. In
Discovering and treating rare diseases Factsheet
published in May 2014
developing countries and emerging markets, we lend our
experience to physicians and local authorities to help build
sustainable healthcare networks that can deliver and pay
for critical treatment. Around the world, Genzyme’s staff
members
collaborate
with
healthcare
providers,
humanitarian organizations, advocacy groups and other
knowledgeable local partners to overcome barriers to
treatment.
Where coverage is not available and in partnership with
humanitarian groups, we strive to:
-
I.
facilitate free treatment access ;
ensure sustainable treatment environment
help patients find alternative funding ; and
help countries establish sustainable healthcare
systems.
HUMANITARIAN
PARTNERSHIPS
In developing countries, government and private health
coverage may be nonexistent, or barely able to address
basic medical care. For patients who require specialized
treatment, care can be financially and logistically beyond
their means.
Living with a rare disease is often
overwhelming for families and having to advocate for your
own care is an additional burden. Genzyme has long
provided several of our therapies for free to people in these
situations, often by partnering with humanitarian
organizations.
We began in 1999 by establishing the Gaucher Initiative, a
humanitarian partnership with Project HOPE to provide
®
Cerezyme (imiglucerase for injection), our first product, to
Gaucher disease patients in China and Egypt. Over time,
as we developed new therapies for other diseases, we
evolved similar programs to distribute those treatments.
We rely on our NGO organizational partners – which
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include Project HOPE, the National Cancer Coalition,
Partners in Health, ANERA, Direct Relief and others – for
their local expertise and relationships, that help us
navigate the challenges of ensuring product reaches the
patients.
II. HELPING COUNTRIES
ESTABLISH
SUSTAINABLE
HEALTHCARE SYSTEMS
Modern, well-equipped hospitals and clinics are crucial to
ensuring that patients have access to Genzyme’s
treatments, no matter where in the world they live. We
dedicate a significant portion of our charitable contributions
to building medical infrastructure, supplying resources, and
training staff in regions of need – particularly in the
aftermath of disasters:
•
•
•
•
Following the 2004 tsunami that destroyed areas
of Southeast Asia, we partnered with the
humanitarian group Project HOPE and donated $1
million over three years for several projects. These
included helping to rebuild and equip a hospital in
Bande Aceh, Indonesia; training pediatric critical
care nurses in Thailand with a curriculum they can
use for ongoing training of peers; and establishing
sustainable healthcare infrastructure in other
tsunami-affected areas.
During the 2009 H1N1 flu pandemic, Genzyme
donated much-needed basic healthcare supplies,
such as face masks, syringes, and gloves, to
replenish Mexico’s depleted hospitals.
We committed $450,000 to relief efforts after
Haiti’s devastating 2010 earthquake. Donated over
three years to the nonprofit Partners in Health
(PIH), the money will provide laboratory and
diagnostic equipment for the new, state-of-the-art
Mirebalais Hospital as well as educate the
country’s next generation of medical professionals.
After a major earthquake hit Japan in 2011, we
responded to numerous calls for help from the
Japanese community, donating medicines that
were in short supply due to crippled Japanese
manufacturing facilities and providing an
emergency supply of medicine that could be used
to aid the treatment of power plant workers who
were exposed to dangerous levels of radiation.
Our employees contributed $51,000 in cash
donations to aid recovery efforts, inclusive of
[matched by?] a $14,000 matching grant from
Genzyme.
Discovering and treating rare diseases Factsheet
published in May 2014
III. BRIDGING
THE GAP TO
ACCESS
Even in countries with established health systems, patients
can still face delayed or limited coverage, reimbursement
difficulties, or other circumstances that prevent their
access to treatment. For such cases, Genzyme has
established several programs to provide our therapies free
of charge, while also working with governments and other
local entities to help identify sustainable, long-term
financial resources for treatment.
•
•
Our first Charitable Access Program was started in
1991 to provide patients in the United States with
free Cerezyme; the program now distributes all of
Genzyme’s enzyme replacement therapies.
In 2003, we expanded beyond the U.S. by
establishing the International Charitable Access
Program. Over the years, other targeted charitable
access programs have focused on specific
regions, such as Eastern Europe, India, and
China.
IV. EDUCATION AND
OUTREACH
Genzyme’s business was founded on treating diseases so
rare that they are often unfamiliar even to many healthcare
professionals. We are therefore acutely aware of how
important education and outreach are to identifying and
effectively treating patients. Through a variety of programs,
initiatives, and partnerships, we have long worked to raise
awareness of the diseases we treat, educate both patients
and the medical community, and advance research and
quality of care. For example:
•
•
•
•
as one of the world’s leading authorities on the
group of rare genetic diseases called lysosomal
storage disorders (LSDs), Genzyme develops
educational materials for both patients and
professionals to increase understanding of these
diseases ;
by establishing LSD registries – large, often
multinational databases to which physicians
contribute clinical data on patients – we help pool
knowledge on rare diseases that would be
otherwise difficult to study ;
we sponsor independent investigator research and
provide grants for continuing medical education
and research fellowships in our areas of focus ;
our Medical Information team is staffed across all
regions to answer questions about our treatments ;
and
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•
we with local patient organizations around the
world to support and advocate for the needs of
their patient communities.
Discovering and treating rare diseases Factsheet
published in May 2014
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