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Patients afflicted by Fabry disease April 5 filed a request for rehearing for a petition asking the National Institutes of Health to “march in” under the Bayh-Dole Act to make the drug Fabrazyme available to them at full dosage.
Fabry disease is a rare but life-threatening disorder with an estimated prevalence in the general population of one in 117,000 people, with as many as 1,500 or more patients in the United States. The patient-petitioners contend that the patent for Fabrazyme, enzyme replacement therapy with agalsidase beta, falls under the authority of the Bayh-Dole Act because the product was developed by Mount Sinai Medical Center as a result of public funding by NIH. The Bayh-Dole Act of 1980 gave universities, nonprofits, and small businesses the option to obtain intellectual property rights for inventions that were developed as a result of federal funding.
The Bayh-Dole Act allows any “responsible applicant” to request march-in rights under 35 U.S.C. §203, which empowers the federal agency funding the research to “march in” and issue patent licenses on its own when the holder of a patent that resulted from the research is not “reasonably” satisfying U.S. health or safety needs.
Genzyme decreased production of Fabrazyme in mid-2009 as a result of a viral infection in its Allston, Mass., plant. The Food and Drug Administration initiated action against Genzyme that resulted in a consent decree that required the drug maker to correct the plant quality violations and to disgorge $175 million in unlawful profits from the sale of products made at Allston.
Because of the disruption in production, Fabrazyme was rationed, and patients were instructed to take less than a full dose. Newly diagnosed patients have not been able to receive the drug at all.
The patients' petition to NIH was originally filed in August 2010. The petition was denied Dec. 3, 2010. NIH said it had decided not to exercise its march-in authority because “any licensing plan that might result from such a proceeding would not, in our judgment, remedy the current problems associated with the access to Fabrazyme.” It noted that FDA approval of another manufacturer's product would simply take too much time and that Genzyme expected to restore full access to patients in the first half of 2011.
The petition for rehearing was prompted by Genzyme's latest manufacturing errors and further delay of access to the drug, as reported on March 25. At that time, Genzyme changed the projection for full access to Fabrazyme from the first half of 2011 to late 2011. “The previous decision for march-in rested on 'facts' that are now known to be untrue,” the petition said, referring to Genzyme's statements about when the shortage would end.
The petitioners also requested that NIH come up with regulations regarding when and how march-in would be available when taxpayer-funded inventions are withheld from the public.
“The NIH decision was predicated on an illogical belief that there is a conflict of law between the Bayh-Dole act and the [Federal] Food, Drug and Cosmetic Act (FDCA), both of which are designed to save lives, not sacrifice them,” the petition said. “By pitting the two statutes against each other, the NIH has asserted that it is impossible to both 'protect the public against nonuse or unreasonable use of inventions' mandated by the Bayh-Dole Act and ensure a safe drug supply under the FDCA.”
In addition, the Fabry patients asked that NIH Director Francis Collins be recused from the decision on the rehearing because he has a financial interest in at least 19 Bayh-Dole inventions. They also warned NIH that former director Elias A. Zerhouni has become the director of research and development for Sanofi-aventis, which recently acquired Genzyme.
The petitioners further noted that the original NIH decision declining to exercise its march-in authority merely said that march-in was “unlikely” to have an effect on the short-term supply and did not address the issue of whether march-in would protect Fabry patients from future supply disruptions.
The petition was filed by attorney C. Allen Black Jr. of Allison Park, Pa., who, after NIH denied the original petition, also filed a request with FDA to allocate the full Fabrazyme dose to U.S. patients before shipping supplies of the drug abroad, and initiated litigation against Genzyme for negligence and for a private cause of action under the Bayh-Dole Act.
Black said in a statement, “Banning patient access to a publicly-funded invention violates the Bayh-Dole act. It is illegal. The only express remedy in the statute is march-in. While the NIH may not have much confidence in what Congress proposed as a remedy, it has an obligation to at least try it.”
“Fabry patients are dying, so any decision regarding march-in needs to be based on the best interest of these patients,” Black continued. “As it stands, refusing the petition reduces the chance that march-in will help patients from 'unlikely,' as stated by the NIH, to absolutely zero.”
Black concluded, “After nine consecutive false promises to patients, investors, and, now, the NIH, there is no reason to believe that Genzyme will ever be able to properly supply drug to Fabry patients. After what these Fabry patients have had to endure, they deserve a reliable drug supply, which the NIH has the power to grant.”
Black told BNA in an e-mail, “If march-in is rejected again, our only resort is an appeal.”
The petition for a rehearing can be found at http://op.bna.com/hl.nsf/r?Open=jaqo-8fmj8k.
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