As research institutions await a ruling by the U.S. Supreme Court that could determine whether university ownership of patents from taxpayer-financed research is in the public interest, a group of patients with a rare, life-threatening ailment wants to turn up the heat on Mount Sinai School of Medicine and the Genzyme Corporation over one such patent. In March, the patients filed a federal class-action lawsuit accusing the school and the biotechnology company of negligence and of violating the federal Bayh-Dole Act by rationing and withholding a drug that could ease the patients’ symptoms or extend their lives.
The drug, Fabrazyme, is based on a patented invention by a Mount Sinai professor, Robert J. Desnick, whose work was financed with a grant from the National Institutes of Health. The patent for the drug, which treats Fabry disease, has been licensed exclusively to Genzyme since the early 1990s.
Now several of those same patients are calling on the NIH to rehear their petition to “march in"—as provided for under the Bayh-Dole Act—and break Mount Sinai’s exclusive license so that another company can make the drug.
Since June 2009, Genzyme has been unable to produce enough of the enzyme-replacement drug to meet patients’ needs in the United States, because of contamination problems at a manufacturing plant near its Cambridge, Mass., headquarters. In response, the company has sold the drug to existing customers at half the dosage approved by the Food and Drug Administration, and denied it to newly diagnosed patients entirely.
The NIH denied the original “march in” petition in December, mostly on the grounds that a march-in wouldn’t solve the problem of the immediate shortage, because it could take three years before another manufacturer could manage the logistics and FDA approvals to get the drug into the market.
C. Allen Black, the lawyer for the patients, said his clients were repetitioning because Genzyme recently reported that it would be further delayed in producing adequate supplies of the drug. The new petition also asks that NIH issue regulations to more clearly define the circumstances under which it would ever grant march-in rights. (It never has done so).
The petition asks as well that Francis Collins, director of the NIH, recuse himself from the case because, it says, his financial interests in at least 19 Bayh-Dole inventions create at least the appearance of a conflict of interest.
Mr. Black has said it is time for NIH officials to step in. “What happens when you license it to somebody who completely screws up?” he asked.
In a written statement, the NIH said it was “concerned” about the further delays in the production of Fabrazyme but had not decided what action it would take on the petition. The agency also said Dr. Collins had no conflict of interest, because he had already terminated his financial interests in patents he holds that could be subject to march-in action.
Unsafe for Patients?
The federal lawsuit, filed in March in Pittsburgh, is the first to test whether, under Bayh-Dole, patients can recover damages if they can prove that a party receiving federal research funds had withheld the resulting invention from the public, according to Mr. Black, the lawyer.
The suit alleges that Mount Sinai explicitly or implicitly consented to a rationing plan that is unsafe for patients and has led to increased incidences of renal, cardiac, and neurological injury, to impairment of hearing and vision, and to the premature deaths of many patients with Fabry disease.
Mr. Black said estimates indicate that 1,500 to 2,000 people in the United States have the disease, which causes severe disorders of the gastrointestinal system.
Mount Sinai’s actions show the “inherent tension” in expecting universities to act in the public interest when doing so might conflict with their own financial interests, he said, noting that Mount Sinai had received millions of dollars in royalties from its license with Genzyme. “It would be nice if the Supreme Court took that into account and said, ‘Look what happened here,’” he said.
In a written statement, Mount Sinai said that the lawsuit against it “has no merit” and that the school did not believe that any new circumstances would cause the NIH to reconsider its previous ruling on the petition. It also said: “As a health care provider, Mount Sinai supports the government’s efforts to assure that as many patients as possible have access to safe and effective medication during the ongoing shortage of Fabrazyme.”
A Genzyme spokeswoman said the company would not comment on the lawsuit or the petition.
In 2010, sales of Fabrazyme totaled $188.2-million, or 5 percent of Genzyme’s total revenue. In 2009, before the manufacturing problems, sales were $429.7-million, or 11 percent of the company’s revenue.
This month Genzyme was acquired for $20-billion by Sanofi-Aventis, a Paris-based pharmaceutical company.
