For the second time in less than a year, a group of Fabry disease patients are petitioning the National Institutes of Health to override use patents held by Genzyme, the beleaguered biotech which has experienced ongoing problems producing its Fabrazyme medication. Although their first effort failed, the patients hope to find a way to produce the drug, which is the only such treatment currently available for a rare, inherited disorder that causes kidney and heart problems (back story here and here).

They also want NIH director Francis Collins to recuse himself from reviewing their petition because they maintain he has a financial interest in at least nineteen federally-funded inventions, which creates “at least the appearance of a conflict of interest,” according to a statement. As part of their salvo about such potential conflicts, they note that former NIH director Elias Zerhouni is now the director of R&D for Sanofi-Aventis, which is acquiring Genzyme in a $20 billion transaction.

By filing a so-called march-in petition, the patients hope to secure rights that will attract a partner interested in grabbing a chunk of a $400 million market and provide a backstop in the event Genzyme remains unable to restore supplies this year as promised. Even though the NIH rejected their earlier bid, their concerns rose two weeks ago, when Genzyme disclosed that a lot of its Fabrazyme med was rejected for failing to meet quality standards, which will further limit supplies to patients who are already suffering from rationing (see this).

In a statement, the patients say progress towards resupplying the US market is not proceeding as originally indicated by Genzyme, which signed a consent decree and paid a $175 million fine for its manufacturing gaffes. The most recent delay, they note, means restoring Fabrazyme supplies will be pushed back from early 2011 to late 2011, adding that “without a second source of Fabrazyme there is no safety net to protect against future supply disruptions…(and) the NIH did not address the issue of whether march-in would protect Fabry patients from future supply disruptions.”

These are some of the same patients who recently petitioned the FDA to insist that overseas stock of the med is first made available to US citizens and, last month, filed a lawsuit seeking class-action status against Genzyme and Mt. Sinai Medical Center, which licensed Fabrazyme to Genzyme and went along with the rationing plan (back story here and here). The suit cites that rationing includes making supplies available at reduced dosages, which the patients argue have not been tested safety or effectiveness.

Last December, the NIH denied their first request by explaining such a move would not address the problem in the short run, due to the amount of time needed to conduct clinical trials and receive FDA approval. The NIH also cited the fact that, since no manufacturer has previously requested a license, an open license will not be granted based on a patient request (Shire Pharmaceuticals recently withdrew its application with the FDA for its Replagal med while more clinical data is obtained).

In seeking the march-in petition, the patients want the NIH to offer regulations regarding about when and how such petitions would be available where tax-payer inventions are withheld from the public. Specifically, they ask the agency to codify events that would trigger a march-in; create a system for notifiying when shortages are imminent; create a compensation program for patients harmed by shortages, and require licensees to find alternative manufacturing sources of life-saving meds that were discovered under provisions of the Bayh-Dole Act in order to mitigate impact of supply problems (read the petition here).

“Banning patient access to a publicly-funded invention violates the Bayh-Dole act. It is illegal..While the NIH may not have much confidence in what Congress proposed as a remedy, it has an obligation to at least try it,” their lawyer, Allen Black, writes us in a statement. “Fabry patients are dying…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.”

“It would be unconscionable to refuse march-in at this point. Genzyme has caused three recent drug shortages…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.”