[Ip-health] NIH March-In Petition Press Release
Biotech. Info. Inst.
biotech at biopharma.com
Tue Dec 7 12:01:35 PST 2010
Much as NIH "March-in" involves the government acting, why do petitioners seemingly want a company to step in? Presuming that this is a public health emergency, why not petition NIH (and/or FDA) to develop and manufacture the product, i.e., immediately hire a contract manufacturer? Depending on one's view and with it unrealistic to expect the private sector to develop and manufacture a me-too product with no viable market, why aren't patient, anti-patent and other activists petitioning for the government to step in and immediately issue development and manufacturing contracts under a crash program for U.S. patients? Isn't this what would be proposed, if say the current sole manufacturer had to similarly stop producing say on-patent essential HIV drug and severe shortages developed?
Why not have the feds do much the same as presumably would be done if this involved a major biodefense, pandemic influenza or other public health hazard readily preventable or treatable by a pharmaceutical product? Presuming "March-in" was granted with Fabrazyme, I would think this government vs. private sector route would be the most realistic and quickest (and probably only) way of actually implementing an alternative manufacturing source.
Otherwise, does the "March-in" petition also demand Genzyme relinquish ownership/control and provide such things as a copy of its full BLA and all supplement filings (i.e., relinquish data exclusivity), reference standards, allow inspections and provide other key information and materials that would realistically be needed to quickly develop and manufacture the enzyme at commercial scale? As illustrated with glucosidase alpha, even Genzyme could not make the same product at larger scale in its own, but different, facilities, with the company having to take the time and effort to get full approval for what FDA considered a different product produced at larger scale, Lumizyme. With there apparently being much truly proprietary information (trade secrets; never disclosed even in patents) involved with agalsidase beta and Fabrazyme manufacture, I cannot imagine any company developing a new replacement product within say 3-4 years from start to approval, and even then, proving safety, efficacy and biosimilarity (if it is to be approved as a biosimilar version) on this timeline would be a major challenge. Or, are "March-in" proponents in favor of FDA lowering its standards in this and other cases of a public health emergency, e.g., granting approval without the need for biosimilar comparative-type trials (which I recall Dr. Love has cited as being unethical) or traditional Phase III-type safety/efficacy trials (with even more delay involved)?
Ronald A. Rader
Author & Publisher of BIOPHARMA: Biopharmaceutical Products in the U.S. and European Markets (www.biopharma.com)
Biotechnology Information Institute
1700 Rockville Pike, Suite 400
Rockville, MD 20852
E-mail: biotech at biopharma.com
Web sites: www.biopharma.com; www.bioinfo.com;
On Dec 7, 2010, at 11:19 AM, Allen Black wrote:
> FabrazymeR - DHHS denies patient's march-in request to end Genzyme's
> rationing of treatment for Fabry Disease citing that FDA rules block
> manufactures from supplying the drug in a timely manner.
> Washington, DC - December 7, 2010, -- On August 2, 2010, Joseph M. Carik of
> North Las Vegas, Nevada, Anita Hochendoner and Anita Bova both of
> Pittsburgh, Pennsylvania, petitioned Department of Health and Human Services
> Secretary Kathleen Sebelius to exercise "March-In" powers under the federal
> Bayh-Dole Act, and issue an open license to use patents needed to
> manufacture FabrazymeR to restore the drug supply to Fabry patients.
> Today, the delegated review agency, the National Institutes of Health,
> denied the petition stating that providing a march-in license would not
> increase the drug supply because FDA rules prevent manufacturers from timely
> producing enough drug to make up the shortfall. Specifically, any
> manufacturer would need to spend at least two years seeking FDA approval to
> manufacture FabrazymeR even under a march-in license. Thus, the NIH has
> found that the Bayh-Dole remedy of march-in is likely to be futile and
> ineffective where the invention is regulated by the FDA.
> Despite the immediate denial of the petition by Fabry victims, the NIH plans
> to monitor the situation and keep the matter open, encouraging manufacturers
> to contact Mt. Sinai for grant of a license. Further, as part of the denial
> of march-in, Mt. Sinai has agreed with the NIH not to seek injunctions
> against any infringers during the shortage. The agreement directly affects
> Mt. Sinai's infringement lawsuit against Shire pharmaceuticals in Germany
> and Sweden. Although the NIH has decided to take no action, it acknowledged
> that the situation grave and expressed concern for the patients.
> A copy of the NIH opinion is available on the web at
> The petitioners find the current situation unacceptable and plan to appeal
> this decision on procedural and substantive grounds. First, no opportunity
> for public comment was provided for victims, legislators and other concerned
> citizens to discuss the issue in an open forum. Secondly, the NIH has
> imposed an improper evidentiary burden of a "guarantee of success" standard
> for march-in petitioner's requests. Third and somewhat ironically, the HHS
> approved the FDA rules that block companies from manufacturing the drug in a
> timely manner under Bayh Dole, even thought the HHS controls the FDA and the
> The current Fabrazyme shortage is one of the longest drug shortages in US
> history (18 months at this point and expected to extend for another year)
> and the NIH has found that the Bayh-Dole remedy of march-in is unavailable
> due to FDA regulations. Since no other drug is available as an alternative,
> Fabry patients now have no hope of a solution, even though US taxpayers
> funded the discovery and invention of FabrazymeR.
> FabrazymeR is the only FDA-approved enzyme replacement treatment for Fabry
> disease, a relatively rare genetic disease. Currently, patients are
> rationed to only 30% of the recommended dosage and no newly diagnosed
> patients are eligible for therapy. As a result of rationing, patients'
> symptoms have recurred and patients are increasingly likely to die of the
> disease. The patents were obtained as a result of public funding by the
> National Institutes of Health.
> Fabry disease is a rare disorder with an estimated prevalence in the general
> population of 1 in 117,000 people. Those with the disease are unable to
> metabolize fats properly leading to numerous symptoms, the most serious of
> which are renal failure and degenerative heart disease. Most patients did
> not live much beyond 50 prior to the development of enzyme replacement
> therapy such as FabrazymeR.
> Genzyme, which produces FabrazymeR under an exclusive license from Mt. Sinai
> Medical Center, has been unable to produce enough drug to treat the US Fabry
> disease market since mid-2009 due to various manufacturing errors.
> Initially, Genzyme's bioreactors were contaminated by a virus and later
> vials for injection were produced containing foreign contaminates. As a
> result, Genzyme entered into a consent decree with the FDA in which Genzyme
> agreed to a fine of $175 million dollars.
> Despite the FDA action, Fabry patients are still unable to receive the
> recommended dosage of the drug. As a result, patients have had a return of
> symptoms and are at increased risk of complications including heart disease
> and renal failure. Newly diagnosed patients are not eligible for treatment
> until the supply is restored, sometime in late 2011 according to Genzyme.
> The European Medical Association found that the health of many patients
> world-wide is declining and recommends a return to full dosing.
> Mr. Joseph M. Carik was diagnosed with Fabry disease in 2005. Mr. Carik's
> cousin Ms. Anita Hochendoner was diagnosed with the disease in 2004. Anita
> Bova, daughter of Ms. Hochendoner was the first family member diagnosed with
> the disease in 2003. As a result of rationing, all petitioners have had
> their symptoms return, including pain and burning in their extremities
> (neuropathy); decreased kidney function (proteinuria), severe
> gastrointestinal symptoms, and cardiac problems.
> Even though FabrazymeR is covered under the patent law, the "march-in"
> provision of the Bayh-Dole act allows additional licenses to publically
> funded inventions where the health and safety needs of the public are not
> met. NIH funded the discovery and development of FabrazymeR.
> The petitioners are represented pro bono in the matter by C. Allen Black,
> Ph.D. who is a licensed patent attorney. Prior to attending law school he
> was an assistant professor at the University of Pittsburgh Medical School
> where he researched infectious diseases and vaccines. He currently is in
> private practice and teaches Biotechnology law at the University of
> Pittsburgh law school.
> James Love of the non-profit organization Knowledge Ecology International
> (KEI) offered the following comments on the March-in request: "Persons who
> have Fabry's disease are at risk today because of legal barriers to the
> competitive supply of agalsidase beta, marketed by Genzyme under the
> tradename FabrazymeR at a price of roughly $700 per day, or more than
> $250,000 per year. The Obama Administration officials who have the
> responsibility of approve or reject this petition will set a standard for
> the degree to which a patent on an NIH funded invention can be held
> accountable, when there are abuses of the patent monopoly. The petitioners
> seek the freedom to obtain independent competitive suppliers for this
> medicine. Genzyme has already earned billions of dollars on Fabrazyme. The
> NIH needs to end the legal monopoly, and permit greater competition in the
> supply of agalsidase beta. This will not only enhance the supply of this
> medicine, but it will send a message that the NIH will not tolerate abuses
> of patent rights for government funded inventions."
> Contact information:
> C. Allen Black, Ph.D. Esq.: TEL +1.412.908.3268; FAX 1.412.318.4815; email:
> allen at patentlawyersite.com; www.patentlawyer site.com.
> James Love: Director of KEI: TEL: +1.202.332.2670; . FAX +1.202.332.2673;
> james.love at keionline.org; keionline.org
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