Back to—and from—the books

Some of you might have been wondering about my uncharacteristic silence since the 2^nd edition of my book was born. No—it’s not from post-partum depression. From June until Thanksgiving I have either been working caring for patients or been immersed in the ritual hazing known as preparing for the “Boards.” I am now coming back to my usual self and a more normal life. I have just taken the ABIM’s Infectious Diseases computer-graded subspecialty examination, yet won’t know the results for months. While recovering from this miserable rite of passage, I thought I would get back to commenting on recent notable events.

Two of my favorite pharma scandals to follow have been intermittently in the news. First is the ongoing saga of Pfizer’s Trovan trials, now of Wikileaks fame. Guess apologies are due for my previous guarded defense of Pfizer. Sorry…

Genzyme is often in my thoughts. Perhaps their story reminds me of karma. They were one of the darlings of biotechs. First, they had great respect, having developed life-saving drugs for orphan diseases, Gaucher’s, Fabry’s, and Pompe’s disease, each caused by in-born errors in metabolism.

Then the company’s avarice caught up with them, and they exploited their position as the sole provider of treatment, as I’ve written about here, both in the US and in Latin America, where courts have upheld the constitutional right of access to medication, regardless of the cost.

Last spring, Genzyme’s products were found to have viral contaminants and even bits of trash, prompting a shutdown of its plant and rationing of the life-saving drugs. This has led to the FDA’s issuing a costly consent decree and fast-tracking a rival’s drug. The rationing has gone on for 18 months (for Fabrazyme) and no end is in sight.

Genzyme has also been the target of a takeover bid from Sanofi Aventis in recent months.

So, patients petitioned the NIH and DHHS to override Genzyme’s patent exclusivity, given that the NIH funded the research at Mt. Sinai, which then licensed Fabrazyme only to Genzyme. Last week, NIH turned down this appeal for licensing rights.

Given that patients have no other source of vitally needed medicines, that the drug development was funded by the public, and that Genzyme has not been a good steward of its patent monopoly, do you think pro-business laws like the Bayh-Dole act should be reconsidered? Should NIH now reject patent exclusivity in favor of patient access? What do you think?

Genzyme's Business Model-You've Got to Read This!

An excellent, thought provoking article by Stephen Heuser, "One girl's hope, a nation's dilemma," appeared in the Boston Globe on June 14. Mr. Heuser details the appalling, rapacious approach Genzyme takes to marketing orphan drugs. A guest response, detailing some of the lesser known implications of Genzyme's predatory practices, follows:

"The Boston Globe article about Genzyme’s business tactics is quite alarming.
Nearly every country in Latin America has incorporat ed a legal guarantee to the "right to the highest attainable standard of health" (a.k.a. right to health) in their constitutions. This judicial language has been enforced in a number of countries.

The right to essential medicines is defined by WHO as “those that satisfy the priority health care needs of the population," with the caveat that they “are intended to be available within the context of functioning health systems at all times in adequate amounts, in the appropriate dosage forms, with assured quality, and at a price the individual and the community can afford."

Recent court cases, particularly in Brazil (initially surrounding access to ARVs) have increasing granted the right to access to essential medicines in Latin America, insisting that the government provide the drugs, no matter the cost. In the past this has led to successful cost-lowering mechanisms which both made the drugs available and didn't run the health budget completely dry. For instance, Brazil put significant pressure on international pharmaceutical companies to lower the prices on anti-retrovirals by developing the pharmaceutical capacity to produce their own generic forms as well as fighting heavily in the World Trade Organization. They also focused attention on prevention efforts, as a way to curb the number of people who would eventually need the drugs.

However, the same leverage does not necessarily apply to a small country like Costa Rica. The pharmaceutical capacity is probably not there, and it is too small a population for market interest in many circumstances.

But this particualar case raises a interesting and less discussed issue--- are drugs for rare diseases considered essential medicines, and does the government have the responsibility to provide them to any affected citizens, no matter the cost? In some ways the problem is ultimately about the ability of the pharmaceutical country to charge any amount, unrelated to a reasonable assessment of the costs of production and a marginal profit.

The U.S. has dealt with the issue of rare diseases by creating special incentives for companies to develop drugs for these diseases, on the assumption that there will be no market for profits. This has been moderately successful.

But Genzyme, is breaking the rules (at least in a moral sense). They are using very dirty tactics and enormous resources to engage governments in a questionable manner. While the right to health is open to interpretation, I find it hard to justify such enormous expenditures for so few individuals. That being said, it is not inconceivable for the drug to be viewed as essential.

The problem really lies in the fact that the company is charging such an enormous price, with a huge profit margin, and very little threat of competition in the form of generics, and they stay below the public health activists radar to a great degree.

While pharmaceutical companies have the right to make a profit, just like any other corporation, they should not be allowed to intentionally drain the already strapped resources of the Ministries of Health of developing countries."

Heather Stone
Founder, President of G.A.A.P.E.:
Global Action Against Poverty Everywhere!
Smith College