One of the HFA’s goals is to raise awareness about women and bleeding disorders.  Women with bleeding disorders such as von Willebrand’s Disease (vWD) often go undiagnosed, leading to serious complications especially if they undergo invasive surgical procedures, such as a hysterectomy.  Approximately 1-2% of the U.S. population has von Willebrand’s Disease.

To view Derick Stace Naughton’s presentation click here.

The NovoSeven® RT press release on the FDA approval for 8mg is attached, and may be found on the company’s website:  http://press.novonordisk-us.com/index.php?s=43&item=259

• Office of Consumer Information and Insurance Oversight

• Centers for Medicare and Medicaid Services

• Food and Drug Administration

• Internal Revenue Service

• U.S. Department of Labor

A number of interim rules that enforce the new health law have been published. Regulations are published in the Federal Register, “the official daily publication for rules, proposed rules, and notices of Federal agencies and organizations, as well as executive orders and other presidential documents”. Many of the rules are listed on the relevant agency’s website.

The public has an opportunity to comment on the regulations before the regulations become final. Comments can be submitted to regulations.gov, the online source for U.S. government regulations from nearly 300 federal agencies. Regulations.gov asserts their commitment to “improving your access to and participation in the federal regulatory process”. As such, the website allows you to search for a regulation, submit comments on a regulation, and sign up for alerts about a specific regulation.

Over the summer the HFA, the American Plasma Users Coalition (A-PLUS) and the National Organization for Rare Disorders (NORD) have written joint comments in response to proposed rules (interim regulations). The first set of comments, were submitted in response to the Interim Final Rules for Group Health Plans and Health Insurance Coverage Relating to Status as Grandfathered Health Plan under the Patient Protection and Affordable Care Act. The groups’ also submitted joint comments in response to the Interim Final Rules for Group Health Plans and Health Insurance Coverage Regarding Preexisting Condition Exclusions, Lifetime and Annual Dollar Limits on Benefits, Rescissions, and Patient Protections under the Patient Protection and Affordable Care Act.

As the interim regulations are released, the HFA will continue to participate in the rulemaking process. All comments will be posted and can be read on the HFA website under the Advocacy tab. HFA encourages individuals and organizations to participate in the regulatory process. Interim regulations have been published for the Pre-Existing Condition Insurance Plan Program (temporary high risk pools) and comments have been requested for the types of standards the health insurance exchanges should be required to meet.

For more information about regulations and the regulatory process please visit the following websites:

___________________________________________________

i Copeland, Curtis; CRS Report for Congress: The Federal Rulemaking Process: An Overview; August 28, 2008

On Saturday,  the morning agenda began with a new program called, PEN’s Pulse on the Road (POTR) led by Laurie Kelley, President of LA Kelley Communications, Inc., and sponsored by Baxter BioScience.  POTR is a series of health insurance symposia where select bleeding disorders experts discuss changes to the health system and the insurance reforms.  The expert panel consisted of Michael Bradley (Baxter), Michelle Rice (NHF), Judy Moore (Indiana HTC) and me. 

During the panel discussion, panelist had the opportunity to address the new health care law and answer questions from meeting attendees.  Concerns and question topic areas included:

• The financial impact of the new health law to the bleeding disorders community
• Immediate elimination of lifetime and annual caps
• The possibility of repealing the new law
• The regulatory process and enforcing the new law
• The impact of the new law on small businesses

POTR will head to the Washington DC area to participate in the Hemophilia Association of the Capital Area (HACA) annual educational seminar on October 23.  Congratulations to Hemophilia of Indiana on a successful meeting.  Stay tuned for additional highlights and video of POTR in Indiana.  You can read more about the meeting at Hemablog:  

 http://blog.kelleycom.com/2010/08/health-care-reform-pulse-on-road.html

Details regarding the investigational factor XIII and the ultra fastacting recombinant factor VIIa are included in the company’s quarterly report under “Biopharmaceuticals,” pg 9.  http://www.novonordisk.com/include/asp/exe_news_attachment.pdf?sAttachmentGUID=a8c5189e-50be-4393-9e10-9da901dc3c17, and excerpted below. 

Biopharmaceuticals

A phase 2 trial evaluating the safety, pharmacokinetics and efficacy of NN1731, an ultra-fastacting recombinant factor VIIa analogue, in treatment of joint bleeds in haemophilia A or B patients with inhibitors has been completed. The trial found that NN1731 has a safe profile for all doses investigated, the ultra-fast-acting profile was confirmed, and NN1731 was efficacious in stopping joint bleeds. Further, all efficacy parameters tested trended favourably for the highest dose of NN1731 compared to NovoSeven®. The control arm confirmed the efficacy and safety profile of NovoSeven® as observed in previous clinical studies. Based on the positive phase 2 trial results, the pivotal phase 3 trial programme for NN1731 is currently being designed.

A pivotal phase 3 trial in factor XIII congenital deficiency, investigating a recombinant FXIII compound, has been finalised. Factor XIII congenital deficiency is a rare bleeding disorder with about 600 diagnosed patients worldwide. This genetic disorder affects both genders and all ethnic backgrounds, and is usually diagnosed at birth. The phase 3 trial enrolled 41 patients for a one-year treatment regimen. The trial proved that recombinant FXIII has a safe profile when administered as prophylactic, monthly replacement therapy to patients with congenital factor XIII deficiency. Compared to a historic control group of individuals who did not receive prophylactic infusions, treatment with monthly injections with recombinant FXIII significantly decreased the annual number of bleeding episodes requiring treatment. Novo Nordisk expects to file for marketing authorisation with the regulatory authorities in the US and EU in the first half of 2011.

New York City Hemophilia Chapter Names Glenn Mones Director of Communications

New York, NY – August 10, 2010 – The New York City Hemophilia Chapter (NYCHC) has appointed Glenn Mones to the position of Director of Communications, according to chapter President Shari Bender. “We’re thrilled to have Glenn as the newest member of our professional team,” Bender said. “Glenn’s extensive knowledge and experience will really help the chapter achieve its goals and mission,” Bender said.

Please Contact:   Melissa Penn   (212) 877-0280  melissa.penn@nyhemophilia.org

Monday, August 9, 2010

 Washington, DC: The Hemophilia Federation of America (HFA) is pleased to announce the addition of two new Blood Brotherhood sites, Hemophilia Foundation of Northern California (HFNC) and Hemophilia Foundation of Minnesota/Dakotas (HFMD). Other states hosting local community support groups include Tennessee, Texas, North Carolina, Arizona, New York, Illinois, Indiana, Oregon and Maryland. Click Here for the Press Release

Pfizer is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted approval for the use of a Prefilled Dual-Chamber Syringe for administration of XYNTHA^® Antihemophilic Factor (Recombinant) Plasma/Albumin-Free to hemophilia A patients. XYNTHA is an injectable recombinant factor VIII product previously approved by the FDA for both the control and prevention of bleeding episodes and surgical prophylaxis in patients with hemophilia A. XYNTHA does not contain von Willebrand factor and, therefore, is not indicated in von Willebrand’s disease.

The approval of the Prefilled Dual-Chamber Syringe is an important milestone for hemophilia patients due to its innovative, convenient reconstitution system that eliminates the transfer step. Pfizer is committed to the hemophilia community not only through the development of novel reconstitution systems focusing on convenience, such as the XYNTHA Prefilled Dual-Chamber Syringe, but also through early clinical research of other proteins, including Factor Xa and Factor VIIa.

 “Because I strive to lead an active life, I am excited by the approval of the Prefilled Dual-Chamber Syringe that eliminates the transfer step,” said Christian, a Pfizer Lifelines Ambassador and hemophilia A patient. “I look forward to being able to use a device that has one less step when I reconstitute my XYNTHA.”

 We encourage you to review the press release below, announcing the approval of the Prefilled Dual-Chamber Syringe, for full information. The release was issued to the general public over Business Wire on August 9, 2010.

Important Safety Information for XYNTHA

• Allergic reactions are possible with XYNTHA. Signs of an allergic reaction may include hives, rash with itching, chest tightness, difficulty breathing, faintness, or fast heartbeat. XYNTHA contains trace amounts of hamster protein. You may develop an allergic reaction to these proteins. Tell your doctor if you have had an allergic reaction to hamster protein. 
• Call your doctor right away if bleeding is not controlled after using your factor VIII replacement therapy; this may be a sign of an inhibitor. Inhibitors have been observed in patients receiving factor VIII products, including XYNTHA.
• The most common adverse reaction in study 1 (safety and efficacy study) is headache (24% of subjects) and in study 2 (surgery study) is fever (41% of subjects). Other common side effects of XYNTHA include nausea, vomiting, diarrhea, or weakness.
• Please see accompanying Full Prescribing Information.(6528) W10547C001 ET01 (CLEAN PHYSICIAN) FACTOR VIII AF RECOMBINANT

(Cincinnati, Ohio)  July 20, 2010 –

Rielly has helped lead BioRx to become one of the fastest growing specialty pharmacies in the U.S., doubling sales each year since beginning operations in 2004 with only three employees. Annual sales for 2009 were $55 million.  BioRx won a 2009 and 2008 Fast 55 Award, sponsored by the Cincinnati Business Courier, which recognizes the fastest growing privately-owned companies in the Greater Cincinnati Region.   Also in 2008, BioRx was named a finalist in the Cincinnati Business Courier’s Best Place to Work in Greater Cincinnati Contest.

The Ernst & Young Entrepreneur Of The Year® awards program celebrates its 24^th anniversary this year, expanding to over 135 cities in 50 countries throughout the world.  The program honors entrepreneurs who have demonstrated exceptionality in such areas as innovation, financial performance and personal commitment to their businesses and communities.

“Entrepreneurs contribute so much to our economy and the fabric of this nation,” said Alan Greenwell, Ernst & Young LLP Entrepreneur Of The Year Program® Director for South Central Ohio and Kentucky.  “These finalists help our region create jobs, while encouraging community growth, development and innovation.  We are pleased to honor them.”

As a South Central Ohio & Kentucky award recipient, Rielly is now eligible for consideration for the Ernst & Young LLP Entrepreneur Of The Year^® 2010 national program.  Award winners in several national categories, as well as the overall national Ernst & Young Entrepreneur Of The Year^® award winner, will be announced at the annual awards gala in Palm Springs, California, on November 13, 2010, hosted by Jay Leno.  The awards are the culminating event of the Ernst & Young Strategic Growth Forum, the nation’s most prestigious gathering of high-growth, market-leading companies.

About BioRx

Based in Cincinnati Ohio, BioRx is a national pharmacy specializing in highly customized care for patients with bleeding disorders, immune deficiency disorders,  hereditary angioedema, and nutritional/digestive disorders.  As one of the nation’s fastest growing providers of specialty pharmaceuticals, the company’s clinical staff reaches patients and physicians in 50 states.

To learn more about BioRx and its products and services visit www.biorx.net or contact:

Judith Cline 513/382-5409 (cell) or 866.44.BIORX; email:  jcline@biorx.net

Sponsors

Founded and produced by Ernst & Young LLP, the Entrepreneur of the Year awards are pleased to have the Ewing Marion Kauffman Foundation and SAP America as national sponsors.  In South Central Ohio and Kentucky, sponsors include Thompson Hine LLP and Smart Business Magazine.

About Ernst & Young’s Entrepreneur Of The Year® Awards Program

Ernst & Young’s Entrepreneur Of The Year® Award is the world’s most prestigious business award for entrepreneurs. The award makes a difference through the way it encourages entrepreneurial activity among those with potential and recognizes the contribution of people who inspire others with their vision, leadership and achievement. As the first and only truly global award of its kind, the Ernst & Young Entrepreneur Of The Year® award celebrates those who are building and leading successful, growing and dynamic businesses, recognizing them through regional, national and global awards programs in more than 135 cities in 50 countries.

About Ernst & Young

Ernst & Young is a global leader in assurance, tax, transaction and advisory services. Worldwide, our 144,000 people are united by our shared values and an unwavering commitment to quality. We make a difference by helping our people, our clients and our wider communities achieve their potential.

Ernst & Young refers to the global organization of member firms of Ernst & Young Global Limited, each of which is a separate legal entity. Ernst & Young Global Limited, a UK company limited by guarantee, does not provide services to clients. For more information about our organization, please visit www.ey.com.

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Posted July 21, 2010

Leeroy Carter is a rare longtime survivor of hemophilia, a bleeding disorder that often shortens the life of those who have it. But the Highland Park resident has far surpassed his peers, becoming the oldest hemophilic in Michigan and the second oldest in the country — by two months. Click here for a link to the Free Press article.