Despite the National Institutes of Health (NIH) denial of three previous requests, three persons petition the Department of Health and Human Services to exercise its “march-in rights” under the Bayh-Dole Act. The petitioners, Joseph M. Carik, Anita Hochendoner, and Anita Bova, are representatives of the entire class of patients with Fabry disease. Fabry is a rare, genetic disease that affects the body’s ability to break down fats causing kidney and heart problems that can lead to death. The petitioners claim that they are suffering from a return of symptoms of Fabry disease due to Genzyme’s rationing plan, which cut their allowance to less than a third of the recommended dose. Genzyme issued its rationing plan following manufacturing problems in its Allston plant.

The Bayh-Dole Act granted universities the ability to control the rights of their inventions that resulted from federal funding, but also retained the ability to abrogate those rights under certain circumstances (“march-in”). Under 35 U.S.C. §203, a Federal agency, here the NIH, has the right to require the exclusive licensee of an invention, created under its funding agreement, grant a license to a “responsible applicant,” so long as the action “is necessary to alleviate health or safety needs which are not reasonably satisfied by the . . . licensee[.]”

Petitioners contend that NIH funding awarded to Dr. Robert J. Desnick of Mount Sinai School of Medicine lead to the discovery that enzyme replacement therapy with agalsidase beta (Fabrazyme®) can effectively treat Fabry patients. Under the Act, Mount Sinai exclusively licensed its subsequent patents, Nos. 5,356,804 “Cloning and expression of biologically active human alpha-galactosidase A,” and 5,580,757 “Cloning and expression of biologically active alpha-galactosidase A as a fusion protein,” to Genzyme. Petioners argue that Genzyme’s current inability to manufacture the requisite amount has adversely affected the health and safety of those patients with Fabry disease. Accordingly, the petition seeks an open license from NIH in order to allow responsible entities to manufacture, import, export, or sell Fabrazyme®.

As genetic testing rapidly develops and becomes more widespread, the already heavily burdened FDA is left clamoring to establish its role in regulating new advancements. Only two days after Pathway Genetic’s May 11th announcement regarding its deal with Walgreens to begin selling direct-to-consumer (DTC) genetic testing kits, the FDA responded by publicly launching an investigation into possible federal regulation. Although agreeing to comply with the FDA by postponing the release of its product, Pathway stood firm in its pronouncement that its testing kits complied fully with all federal and state regulations. The point of contention for this type of testing kit, which is produced and marketed by the same laboratory, focuses on whether or not the product meets the definition of a “device” under Section 201(h)(2) of the Federal Food, Drug, and Cosmetic Act (FFDCA). Specifically, whether the product can be classified as an:

instrument, apparatus, implement, machine, contrivance, implant, in vitro reagent, or other similar or related article, including any component, part, or accessory, which is…intended for use in the diagnosis of disease or other conditions, or in the cure, mitigation, treatment, or prevention of disease, in man or other animals.

On a policy level, the battle lines are drawn between those who claim the results of these genetic tests are used by consumers to make life-altering medical decisions following genetic “diagnoses.” Lack of scientific certainty about whether genetic indicators represent an accurate assessments of a person’s health and the lack of consumer understanding regarding this point worry physicians and lawmakers alike. In response, industry leaders, such as 23andMe, Navigenics, and deCODE Genetics, claim that understanding the body’s genetic makeup is simply a learning tool and does not replace the physician as the ultimate aid in making healthcare decisions.

The FDA released numerous letters to these and other companies on June 11th delineating the status of DTC testing kits as devices. But these actions narrowly affected the industry as a whole until the FDA’s June 16th announcement that it would hold a public meeting to discuss the regulation of all laboratory-developed tests (LDTs) not just those produced by a select few companies. This meeting, to be held July 19-20, will impact the entire LDT industry including hundred of laboratories currently producing DTC tests and the thousands of products already on the market. As a result of this meeting and its effects, the FDA’s laissez faire stance to DTC testing regulation over the past few years may soon come to a screeching halt.

Today opens the Preparing for a Pharmeceutical Response to Pandemic Influenza symposium at Seton Hall Law School.  We have an amazing set of speakers, and we’re looking forward to an excellent event.