Indeed, Genzyme's overseas track record—about 50 percent of revenues come from outside the United States—is above average.
But, that international success has occurred in countries with intact healthcare systems. Thus far, Algeria is one of very
few countries where Genzyme has been successful in developing the kind of reciprocal relationship it is after, where a government
that did not previously support reimbursement for therapy starts paying. (Genzyme also worked with health officials in Romania,
population 22 million, to come up with a plan for including Cerezyme in its annual healthcare budget.)
China A prime target today is China, where 100 Gaucher patients currently receive Cerezyme for free. Enthusiastic about the prospects
there, Genzyme recently opened an office in Shanghai, and plans are underway to open in Beijing.
"China is 1.3 billion people," says Smith. "There are about 13 cities that are projected in the next five years to have a
middle class that can access Western-style medicines. So you're talking about 100 million people that could—and will, based
on existing growth and the infrastructure of China—be potentially needing our products."
But consider: In Algeria, a country of only 30 million people, the number of Gaucher patients wanting Cerezyme increased nine-fold
once communications efforts within the region improved. It's reasonable to expect the number of patients receiving free Cerezyme
in China to increase at a similar rate (or higher, given China's inherent advantages over Algeria due to size). That leaves
900 people, at minimum, needing the drug. In accordance with its "everyone-who-needs-it-gets-it" policy, Genzyme will have
to give away $180 million worth of Cerezyme per year, until the Chinese government decides to reimburse for the drug—a milestone
that Termeer admits will take at least a decade to accomplish. That's $1.8 billion over 10 years for just one drug, in one
That's a huge investment (by comparison, Genzyme gave away only $46 million worth of all drugs combined in 2004), but Termeer
says it's okay. "Anything that you engage in from the beginning will take ten, fifteen years to amount to anything." China
is "starting from scratch," he says. "We need to be on the ground in a multi-functional sense—not as a cheap resource, but
as a participant in building the market."
In the United States, the job of getting reimbursement for Cerezyme has been relatively straightforward. Most patients are
covered in full by their insurance policies. Patients who need help dealing with their coverage can turn to Genzyme Treatment
Support (GTS). The group is made up of case management specialists, who help patients review their current insurance policies
and, if necessary, work with them to obtain additional coverage to maintain long-term therapy. GTS also supports patients
who need Fabrazyme and Aldurazyme.
Rhonda Buyers, executive director of the National Gaucher Foundation, the disease's foremost support group, says the feedback
she's received from patients indicates GTS is an effective system. "Once [patients] have been identified," she says, getting
access to treatment "can be a very fast process—certainly if their insurance company is already paying for other Gaucher patients."
For US patients who don't have insurance, Genzyme offers the Charitable Access Program through which Cerezyme, Fabrazyme,
and Aldurazyme are provided for free. There is no cap on the number of people who can be in the program at a time (as of January
1, there were 37), but it is temporary. Patients and their families are expected to continue exploring alternative funding
options while Genzyme pays for their treatment.
So far, payers have been good about paying for Genzyme's super-expensive products. This is partly because there are so few
patients who need the drugs, and therefore, the collective impact on individual insurance companies is minimal. It's also
because the drugs work.