[Ip-health] NIH March-In Petition Press Release

Allen Black drallenblack at verizon.net
Tue Dec 7 12:17:54 PST 2010

One of the major policy goals stated in the statute is that in return for
granting patent rights on public inventions, a remedy (march-in) would exist
for patents that are misused especially where the public is harmed.  The
ruling makes clear that no remedy to protect the public actually exists for
FDA regulated inventions .  Thus at least for FDA regulated inventions, the
Bayh-Dole act is precluded by the Food, Drug, and Cosmetic Act.  Since
Bayh-Dole is a nullity for publically funded, but FDA-regulated inventions,
the courts will likely find that the patents are also void because the
public health cannot be assured under the statute.  While such a result will
definitely have a dampening effect on private investment, innovation policy
should never come at the cost of death and suffering of American citizens
who paid for the research in the first place.  
Allen Black
Counsel for petitioners.  

From: Biotech. Info. Inst. [mailto:biotech at biopharma.com] 
Sent: Tuesday, December 07, 2010 3:02 PM
To: allenblack at sunpillar.com; ip-health at lists.keionline.org
Cc: Biotech. Info. Inst.
Subject: Re: [Ip-health] NIH March-In Petition Press Release

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)?  

Thank you.

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
Phone:  301-424-0255
E-mail:  biotech at biopharma.com
Web sites:  www.biopharma.com; www.bioinfo.com; 
    www.biopharmacopeia.com; www.biosimilars.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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