Genzyme Charged With Double Standard By Patients

Genzyme Charged With Double Standard By Patients

May 13th, 2011 // 1:54 pm @ marquee

In the latest flap over Genzyme, its ongoing manufacturing problems and subsequent product shortages, a group of Fabry patients are charging the biotech has employed a double standard when it comes to providing its Fabrazyme med to US patients and those in Europe.

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For the past two years, Genzyme has had difficulty supplying two drugs for rare diseases due to a series of manufacturing gaffes that led the biotech to signe a consent decree and pay a $175 million fine. Despite repeated attempts to resume full production, Genzyme has been unable to deliver and forced patients to accept rationing that amounts to lower dosages or no treatment. The ongoing delay has allegedly contributed to at least three deaths and worsening health among some patients.

In response, several US patients filed a lawsuit against Genzyme and petitioned the National Institutes of Health to override use patents held by Genzyme (see this). By filing a so-called march-in petition, they hope to secure rights that will attract a partner interested in grabbing a chunk of a $400 million market and provide a backstop in the event Genzyme remains unable to restore supplies this year as promised.

Now, though, this same group of US Fabry patients charge Genzyme has offered full dosages to patients in Europe. They cite a pair of letters sent last year to doctors in Europe that, due to serious adverse event reports among patients on low dosages, full treatment should be reinitated (see this and this). And they are angered, since the biotech allegedly has not tested whether reduced dosages are safe or effective. In fact, in November 2010, the European Medicines Agency determined the lowered dose of Fabrazyme resulted in patients having more strokes, heart attacks, renal disease and other symptoms (read here).

“In other words, during the worldwide shortage, Genzyme advised European physicians to either restore the original dose of Fabrazyme to European patients or switch to them to Replagal (a drug made by Shire Pharmaceuticals),” writes Allen Black, a lawyer who represents the US patients, in a letter to the NIH. “US physicians have not been advised to return US patients to the original dose, despite having observed the same clinical deterioration in US patients.”

He goes on say at least half of Fabry patients in Europe were on a low dose, which he suggests indicates at least some Europeans are receiving the original dose. By contrast, all US patients are experiencing rationing. And Black charges Genzyme took this step in to maintain market share in Europe. “It is unconscionable that Genzyme is restoring full doses to patients in the European market, which already has access to an alternative medication, while simultaneously denying access to the recommended dose for Americans during the shortage,” he writes.

A Genzyme spokeswoman offered this explanation: “In Europe, any guidance regarding the dosing and treatment of Fabry patients is made by the EMA. Genzyme followed this guidance through the distribution of letters to healthcare professionals in Europe. It is important to note that there is an approved alternative treatment available in Europe that is not approved in the US, and the majority of Fabry patients in Europe have been placed on this alternative therapy,” which is Replagal.

“Therefore, physicians are managing the limited supply of Fabrazyme in Europe differently than in other markets like the US. In the US, Genzyme worked closely with the Fabrazyme Stakeholders Working Group (consisting of Fabry medical experts, patient organization leaders and Genzyme representatives) to determine how to best use the available product in the U.S. and our communications to the US Fabry community reflected those discussions. We keep the FDA informed of our guidance.”

In response, Black writes this: “That is not true. US doctors would manage the situation just the same way if Genzyme would let them. Physicians in the US only prescribe the full dose, but Genzyme will not honor the doctor’s request, so doctors in the US must give the lower dose. Doctors here are as much of a hostage as the patients. It is absolutely shameful that Genzyme is now trying to blame US physicians for Genzyme’s allocation policy and the injury it has caused to Americans…

“The fact that Genzyme informed they FDA that they had adopted a double standard does not mitigate except to implicate the FDA in abandoning its responsibilities to Americans…What is worse, the Bayh-Dole act specifically prohibits a patentee from denying drug to US citizens where the citizens paid for the development…The bottom line is that Genzyme kept Europeans healthy and safe while continuing the deadly experiment on sick and dying Americans. All paid for with US tax dollars.”

He adds that American Fabry Stakeholders working group all receive some form of compensation from Genzyme.