Genzyme Charged With Double Standard, Again

Genzyme Charged With Double Standard, Again

November 2nd, 2011 // 12:19 pm @

Throughout the controversial shortage of Genzyme medicines, the biotech has upset Fabry disease patients with rationing of its Fabryzyme med in the US. The official reason given has been there simply is not enough to go around, although patients in Europe were offered full dosages, fueling perception of an inexplicable double standard (back story).

Genzyme, you may recall, has had trouble supplying two drugs for rare diseases due to manufacturing gaffes over the past two years that led to a consent decree and a $175 million fine. Despite repeated attempts to resume full production, the biotech has been unable to deliver and forced US patients to accept rationing that amounts to lower dosages or no treatment (see this).

Earlier this year, a spokeswoman for Genzyme, which is now owned by Sanofi, explained that disparities between the US and Europe were due to guidance issued by the European Medicines Agency and the biotech was following the guidance. She added that an alternative drug is available in Europe, where Shire Pharmaceuticals’ Repagal is approved; the med is not yet approved in the US, although Shire is seeking fast-track approval.

The explanation further infuriated Fabry patients, given that Genzyme was contradicting a goal that was noted in an August 2009 update, when shortages were becoming critical and a plan was being developed to manage supplies. One of the stated goals at the time was to “ensure equity on a global basis so that no one country is contributing disproportionately compared to others” (see here).

More recently, an October 3 document, which attempts to offer an update on global supply allocations, reiterates that “authorities recommend that patients be treated at a full dose or switched to an alternative product” and that Genzyme “does not make treatment recommendations.” In other words, the decision to offer meds to European patients remains out of its corporate hands (read here).

Yet, Genzyme did not explain why Fabrazyme would be shipped to Europe when an alternative was still available. Moreover, the biotech has been involved in determining treatment recommendations dating to 2009, when it formed dose conservation, or rationing, guidelines with the Fabry Support Working Group, which included Genzyme employees and others with ties to the biotech (see this and follow the * at the bottom of this post for more details).

Asked about the continued discrepancy in allocations, a Genzyme spokeswoman wrote us that “availability of alternative treatment options, treatment practices and regulatory guidelines, etc. mean that that the timing and amount of Fabrazyme available to patients may vary significantly from country to country.” She added that these decisions are being made by doctors and patients.

As for EU regulatory authorities, she insisted Genzyme has tried to “accommodate” requests that patients be treated at full dose or given Replagal and maintained that about 70 percent of European Fabry patients have been switched since mid-2009. “This allowed for a smaller number of patients to be treated with a full dose of Fabrazyme,” she wrote. “In turn, this led to an overall decrease in the proportion of Fabrazyme supplied to Europe. Currently, there are also a handful of European patients who are on a reduced dose of Fabrazyme.”

But Fabry patients remain upset and an attorney who represents several of them, argues the logic is commercially motivated. “Preferentially allocating full dose Fabrazyme to patients that have another supply alternative, which is Replagal, is medically irrational and results in the most vulnerable population – US patients – being put at greatest risk of death compared to Europeans for no apparent reason other that Genzyme’s desire to prevent eroding market share in Europe,” writes Allen Black.

“The bottom line is that Genzyme’s preferential allocation system means American Fabry patients will die, while European patients remain safe having a choice of either FDA/EMA approved doses of Fabrazyme or EMA approved doses of Replagal, a choice that Americans don’t have. Americans get half doses sporadically, or, if diagnosed after June 2009, nothing at all, despite having participated in the clinical trials and funding the research leading to the invention.”

He goes on to argue that Genzyme is, effectively, promoting off-label use, even though the biotech does not recommend using less than the full dose. “Although a bold statement, it is utterly ridiculous. While the FSWG may promote non-FDA approved doses, Genzyme cannot chose between the FSWG recommendation and the doctor’s recommendation. No doctor I am aware of recommends using less than full dose.

“Instead, the most egregious form of off-label promotion is to ban patients from receiving FDA-approved doses…The Genzyme allocation system guarantees 100 percent off-label use no matter how loudly physicians, the FDA, or patients protest. If anything, this form of off-label use completely defeats all FDA regulations and protections. Quite simply, no amount of advertising has ever been as effective as Genzyme’s rationing system in ensuring a off-label use of a drug.”

* – among those who were listed as members of the FSWG were Joel Charrow, who has received research support from Genzyme ; Michael Mauer, who has received lecture fees and grants from Genzyme; Manesh Patel, who has served as a Genzyme consultant ; C. Ronald Scott served as advisor or consultant to Genzyme; Katherine Sims received research support; Maryam Banikazemi has received honoraria and is a member of the Board of Advisors of the Fabry Registry David Warnock has received grants and done consulting and William Wilcox has received speaker and consulting fees .

Source: Pharmalot

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