One Giant Leap Backward for Treatment: Novitas, WTH?*

(see update in red below!)

who knows me, knows I don’t swear. Really. Went through childbirth three times,
twice without anesthesia, and not a foul word passed my clenched teeth. (See translation
for “WTH” below.) But when I read a recent decision by a payer, I was tempted
to let loose. 

is a Medicare administrative contractor for two jurisdictions: 1) AR, CO, NM, OK, TX, LA, MS and 2) PA,
DC, MD, DE, NJ, and parts of northern VA. That’s a lot of people impacted by their decisions. Remember I’ve been
warning you and warning you, since 2005 to be exact, that payers would be exerting more infleunce over our treatment? You do remember the “Coming Storm”
article, yes?
it’s happening. And the community has been vigilantly safeguarding our right to
access to therapies and treatment. First payers got in on the specialty
pharmacy gig. Cigna, Aetna… they all started forming their own specialty
pharmacies and could legally force you to switch to use theirs. That was a game
treatment changes. So many HTC people told me this wouldn’t, couldn’t, mustn’t
happen. How could a payer, an insurance company, have a say in what a physician
Wake up. Novitas has mandated in
those jurisdictions above that if patients use NovoSeven RT® (to treat their inhibitor)
they must infuse under the direct supervision of a physician. Like, in his office! And NovoSeven usually requires every
two hour doses to resolve a bleed.

This decision is called “local
coverage determination (LCD)” and it is troubling: 
Novitas writes “Factor VIIa
… is indicated for the treatment of bleeding episodes in hemophilia A or B
patients or with acquired hemophilia. NovoSeven, as noted in the Prescribing
Information for the product, should be administered to patients only under the
supervision of a physician experienced in the treatment of hemophilia. Novitas
notes this product would not be appropriate to be used in a self-administration
situation and the supervision should be ‘direct’ as in ‘incident to’ in an
office under Part B Medicare.”

Uh oh. As noted in the Prescribing Information? True. The Novo Nordisk
PI that comes with the product does say “Administer NovoSeven RT to
patients only under the supervision of a physician experienced in the treatment
of bleeding disorders.” Yikes. It’s not what we meant to say! But Novitas is
taking us literally, so yeah, they do have a case there.  
But, according to the Social Security
Act, Medicare allows for hemophilia patients deemed competent to administer at
home. Without medical supervisions. Like, the way we have been doing it for
years. And it doesn’t say everyone except
NovoSeven users. So Novitas, yeah, you are restricting treatment against what the federal government allows.

And NHF’s MASAC recommendation #227
specifically says patients should always keep doses on hand for emergencies
(like, at home).

So what’s a manufacturer, pharmacist, or patient to do?
Well, Barb Forss of Washington figured that
one out. She’s a severe FVII deficient patient who self-infuses NovoSeven RT
daily for prophylaxis, and every 3 hours for a bleed, until it’s resolved.
 She’s been using this product for over 14 years.  Is she supposed to
go to her HTC every time she needs treatment? She would have to move in with
her HTC team whenever she as a bleed. I hope they have a nice room with a view for
her there.
But Barb doesn’t want a new apartment in a
hospital. She wrote to Novitas, and said, “I went undiagnosed for 47 years. I
was hospitalized over 50 times and tethered to an infusion pole for days and
sometimes even weeks.  If they threw enough [fresh frozen plasma] at me, I
would eventually stop bleeding, then sometimes start again after being released
from hospital.  You can’t imagine how the quality of my life has improved
being able to self-infuse my NovoSeven and avoid hospitalization from bleeding
for the first time in my life.” She adds: “If this decision were to come down
in WA state when I’m on Medicare next year, that would mean I would need to
drive 220 miles roundtrip, daily, just for my prophy infusion at my HTC in
Seattle.  And I suppose just bring an overnight bag and camp out at my HTC
for the duration of any internal bleeds so that I could be monitored by my
doctor, right?”
I think Barb is close to swearing.
In case Novitas still doesn’t get it, she
adds:  “Please reconsider this determination.  Every patient I know
who uses NovoSeven can self-infuse blindfolded, it’s as easy as pie and does
not require a doctor to be there.”  
Novo Nordisk wrote in as well, asking to change the PI text to  “Initiate treatment with NovoSeven RT
under the supervision of a qualified healthcare professional experienced in the
treatment of bleeding disorders.” Then the patient can take over (like we always
know the Hemophilia Alliance, NHF and HFA are all mobilizing to get this
time is running out. This decision took effect April 9. Barb tells me there is
a 45-day response window. We need to let Novitas know this is not a good
decision, and must be rolled back.
Noviats does allow you to challenge the decision,
but only individually: “If changes or revisions to the policy are to be
considered, you must submit a LCD Reconsideration request which can be found on
our website at:  This process is a mechanism for interested parties to
request a revision to an LCD.  Requests must be submitted in writing, and
must identify the language that the requestor wants added to or deleted from an
LCD. Requests must include a justification supported by new evidence, which may
materially affect the LCD’s content or basis.”
Blah. Lots of words and work. But we will do
it, until our community rolls it back. Hey, if we can get ABC to eat their
words about hemophilia being a result of inbreeding, and have them redo a sitcom,
surely we can get Novitas to change this horrible decision.
Ironically, in the signature of the email of
the Novitas CMD Research Analyst, Medical Affairs, who is responding to the
community, is this thoughtful little quotation:
 “It is not what you do for your
children, but what you have taught them to do for themselves, that will make
them successful.” –Ann Landers

That means
everything except self-infusion with Novo Seven RT, I guess.
For more
info and to get involved, contact NHF at 212.328.3700,
Contact the Novitas rep at (717) 526-3785.
the heck?

 Great Book I Just Read
Dead Wake: The Last Crossing of the Lusitania
Erik Larson [Kindle]
Larson is one of America’s best historical storytellers and his latest
book, on the 100th anniversary of the sinking of “Lucy” on May 7, 1915,
is fantastic. He deftly weaves together WWI history, politics, engineering of
these empresses of the sea, and personal stories of individuals on board. A
tragic tale, Larsen shows how plenty of intel was available that could have prevented
the sinking of Cunard’s masterpiece by a single torpedo from U-20 and the
deaths of over 1,700 people. Still, no one would believe that Germany could
fire upon a passenger ship. Contrary to popular belief, the sinking did not
bring the US into the war, not right away. Another 2 years would pass. Five/five

UPDATE 5.12.15 from NHF: “NHF is aware of the Medicare contractor, Novitas’ new local coverage determination on clotting factor coverage and its policy that patients infusing NovoSeven may not self-infuse in the home.  NHF staff, joined by HTC Medical Directors from LA and PA have spoken with Novitas’ Medical Director about our concerns with this policy and have provided Novitas with requested literature on this subject.  In addition, MASAC, at its last meeting passed a resolution recommending that self-infusion of inhibitor treatments is the current standard of care.  This information will also be sent to Novitas.

We suggest that patients on Medicare living in states under Novitas’ jurisdiction, who are on NovoSeven talk to their HTC about the impact of this policy on their care.  Together with the HTCs, patients can determine whether there is a need to contact Novitas. “

Mix It Up: New Device for NovoSeven RT Users

Here’s a press release about a new reconstitution device from Novo Nordisk. It’s very cool that the idea originated with the Novo Nordisk’s Consumer Council, which I used to facilitate (and miss! great times):

Novo Nordisk is pleased to announce the introduction of a new reconstitution device for NovoSeven® RT (Coagulation Factor VIIa [Recombinant]). Beginning on May 1, 2013, NovoSeven® RT with MixPro™ will replace the current NovoSeven® RT and will be available to congenital hemophilia A or B patients with inhibitors, acquired hemophilia, and congenital factor VII deficiency.

Although the product will remain the same, NovoSeven® RT with MixPro™ will now include a syringe prefilled with diluent and a vial adaptor to reduce the number of steps needed to prepare NovoSeven® RT for infusion. This is expected to assist NovoSeven® RT patients and their caregivers. Previously, the NovoSeven® RT package included two vials, one containing the active drug substance as a lyophilized powder and the other containing diluent, requiring two additional steps for reconstitution.

This enhancement was developed in response to suggestions from the Novo Nordisk Consumer Council, which comprises bleeding disorder community members. NovoSeven® RT with MixPro™ received approval from the U.S. Food and Drug Administration in October 2012.

It is important to note that although the MixPro™ prefilled diluent syringe uses a standard luer-lock connector, not every needle-free IV system is compatible with glass prefilled syringes like MixPro™. If you are a health care professional with questions, please contact Novo Nordisk Medical Information at 1-877-NOVO777 for assistance and compatibility information. All others should contact a health care professional with questions.

For more info visit

From Diabetes to Hemophilia

Novo Nordisk is best known throughout the world as the leading manufacturers of insulin to treat diabetes; in the hemophilia community, it is best known for NovoSeven®RT, an inhibitor bypassing agent, a miracle drug to many. (Photo: with Lars Sørensen and Stephanie Seremetis)

I visited Novo Nordisk’s headquarters in Copenhagen, Denmark on Friday, to speak with CEO Lars Rebien Sørensen, arguably the most famous businessman in Denmark. The country is very small, about the size of my home state Massachusetts, and Novo Nordisk is its biggest company. Denmark is very pretty, a haven and heaven for cyclists, as everyone seems to bicycle everywhere. It doesn’t take long to get to Novo Nordisk from my hotel; indeed, it wouldn’t take long to get anywhere in this diminutive and charming country.

I am struck by how understated Novo Nordisk is. The buildings, designs, interiors are all minimalist, low key, humble even. Entering the executive suites, I am surprised to see that no one has offices per se; just desks, no walls, no glass, only hip-high borders to segregate one work area from another. Quite the opposite of the US and most other places, where executives usually prefer to have corner offices with glass, big cherry furniture, and doors for privacy. Here, everything is open, transparent.

Lars Sørensen came to the US in March (see to meet for the first time the key opinion leaders in the hemophilia community, and to also meet some patients. This meeting now was to follow up on points we touched upon, including products in the pipeline and the company’s policies for international humanitarian donations of product.

I first had lunch with Lise Kingo, executive vice president, Corporate Relations, and Charlotte Ersbøll, corporate vice president, who graciously filled me in on their programs. Next, a meeting with Charlotte and Trine Thomsen; Trine is heading the HERO project, a massive multicounty research that will attempt to quantify and publish responses about the psychosocial aspects of life with hemophilia. (Watch our blog for more news about this)

Last, a meeting with Lars and Dr. Stephanie Seremetis, formerly with a New York HTC. I had heard of Stephanie for so many years and each of us couldn’t quite believe we had never yet met! Lars is an energetic and welcoming executive, and we spoke for an hour about:

1. New products. We spelled out some of Novo Nordisk’s new products in research in the February issue of PEN: long-acting FVIIa; recombinant FVIII (to take on Advate and now Xyntha); a long-acting rFIX product; and a FXIII (13) product. We spoke about causes of inhibitors, patients’ personal experiences and what physicians are requesting (nonimmunogenic factor VIII—in other words, factor that does not cause inhibitors, although it’s not clear yet if how and why factor causes inhibitors)

2. Hemophilia in developing countries. One of my passions is to increase product donations to developing countries, and Novo Nordisk as of yet is not a big donor—although they donate a lot of product in the US, its main market. One problem is production itself: a specialty product for such a rare complication of such a rare disorder means not a whole lot of product sits on the shelf anywhere in the world. In other words, there isn’t much to donate. At Project SHARE we are over the moon when a rare donation of NovoSeven reaches us from some patient, HTC or homecare company, and usually it’s only a few boxes. It will be a while before donations become a routine thing and for now we will be dependent on third parties who donate NovoSeven to us. Novo Nordisk supports Project SHARE’s operating expenses, as do quite a few other pharmaceutical companies and others, and for this we are glad, but we look forward to the day when we and others can also expect product donations.

One interesting insight is Novo Nordisk’s take on product donations. As you can imagine, donating tons of products (just like doling out money to charities without expectation of anything in return) into a poor country can lead to dependency, which only perpetuates a bad situation; this is a constant threat to donations of anything in the humanitarian field. Lars explained that right now, the Novo Nordisk diabetes model has been extremely successful throughout the developing world, and perhaps this is the model to be followed in the future with hemophilia, when emerging markets like India and China start to purchase products. In diabetes, Novo Nordisk’s biggest single financial commitment is its annual donation to the World Diabetes Foundation. At the company’s Annual General Meeting in 2002, the shareholders made an agreement obliging the company over a 10-year period to make an annual donation of 0.25% of the net insulin sales for the preceding financial year (not to exceed approximately $12 million or 15% of taxable income).

In diabetes, the company’s donation policy is tied to product sales: higher sales mean a higher donation to the World Diabetes Foundation, and you can see where this would make good business sense. Also, clinics get established in this model; I saw some of these while traveling in Africa this year. Novo Nordisk is big in Africa, and does a lot to help those with diabetes.

In hemophilia, Novo Nordisk donates about $3 million to the Novo Nordisk Haemophiia Foundation (NNHF). NNHF supports sustainable local and regional projects as well as the advancement of relations between healthcare professionals, patient associations and authorities within the hemophilia community.

In hemophilia, a model like the diabetes model will take a long time to develop. But there are signs: patients in developing countries are integrating and speaking up like never before, thanks to information’s and resource exchange on the internet, and entities like the World Federation of Hemophilia, which brings patients, physicians and nonprofits together to learn and network, and to institute programs that train medical staff. Even at Project SHARE we sometimes, often, dangle product donations like a carrot to get patients organized and to take action. It works, no doubt. But people are still dying, weekly, and we will always need to donate life-saving products with no strings attached sometimes.

Hemophilia is a rare disorder, an expensive one, and we are a small community. Changing the current situation in poor countries will take a long time, and I plan to stick around for as long as humanly possible to ensure that change happens. Just so happens that Novo Nordisk’s slogan is “Changing Possibilities in Haemophilia®” and that needs to be believed no where more importantly than in hemophilia, by the patients, governments, humanitarians and very companies that provide the product. (Photo: Hartnell of Jamaica, who died of inhibitor complications in August 2009 at age 20)

Thanks to Lars and his team for welcoming me, for partnering with us, for sharing so much of their time and ideas. Let’s count on changing possibilities in the near future and make some dreams come true for those patients in countries where no treatment is available now.

Great Book I Just Read
Lord Jim
By Joseph Conrad
This classic was the basis for the movie “Apocalypse Now.” A young sailor, Jim, runs from his past by taking job after mundane job in far ports in the 1800s. He had served as first mate on the Patna, a ship carrying 800 Muslim pilgrims, which hit something in the dark waters one night during his watch and threatened to sink. Jim and the rest of the crew abandoned ship, thinking there is no hope of saving the passengers. The ship does not sink, and when Jim and the crew are rescued, their reputations are ruined. This story within a story is narrated by Marlow, an old sailor, who is fascinated by Jim, and spends the rest of his life trying to help the young man find redemption. Eventually Jim is offered a job to run a trading post in the far reaches of Southeast Asia, so remote few white people have ever visited. Eventually, his leadership and good deeds earns him the title “Lord Jim,” and he is beloved by the people. Soon his honor and character are tested as never before, earning him a last chance at redemption. The writing is amazing, the structure complex, superb, but difficult: some paragraphs are three pages long, and the story within a story can be confusing at times (think of the movie “Inception”!), but a great read; not to be missed. Four stars.

Inhibitor Summit 2008

The very successful Inhibitor Summit meeting took place this past week and weekend in Denver, Colorado. Over 80 families from all over the US attended to learn about inhibitors, current research, and treatment and insurance information. If you’ve never been with a group of inhibitor patients, you’d be amazed at their level of knowledge and expertise. I guess they have to learn fast, given the nature of the disorder, which is a severe complication of hemophilia.

I arrived a day early to help facilitate the Novo Nordisk Consumer Council, which is comprised of patients with inhibitors and parents of children with inhibitors. It’s a wonderful chance for the patient community to give direct feedback to Novo Nordisk, and to learn in detail about the company’s philosophy, products and ideas. The Council actually helps to shape some of the educational material coming from Novo Nordisk. Working from 8 am to 4 pm, with only a ten-minute break, we really accomplished a lot and learned a lot!

The Summits just keep getting better and better. Dr. Guy Young from Children’s Hospital, Los Angeles, moderated the panel speakers and also gave an excellent overview on inhibitors. He focused on differences between FEIBA and NovoSeven, the two main treatment options for inhibitor bleeds, their efficacy and safety, dosing regimens. Most exciting were the slides showing how effective it is to use these products prophylactically to prevent bleeds. Dr. Steven Pipe gave a very insightful yet understandable presentation on the future of inhibitors: what’s being worked on now around the world–very exciting things. I’m saving what I learned to publish in PEN soon.

I attended a few other sessions: Regina Butler, RN of Philadelphia, and Angela Forsythe, PT, teamed up to give a dynamite presentation on joint disease. This is especially important for those with inhibitors due to the excessive bleeding they face. Angela stressed the important role physical therapy and exercise play in rehabilitating joints. Stopping the bleeds is not the only thing we have to worry about; we need to ensure the joints are cared for to help prevent future bleeds, and future deterioration. Excellent presentation.

There were other talks on pain management, prophylaxis, and central venous access devices. Again, CVADs are very important to inhibitor patients because when they have Immune Tolerance Therapy, they are being infused daily, sometimes for months! Vein access is extremely important.

I was honored to give a presentation along with Glenn Mones, VP Advocacy for the National Hemophilia Association, and Val Bias, colleague, friend and new CEO of NHF. We spoke about the changing insurance scene, what’s causing the changes, how we can advocate to slow down change and ensure we get the insurance coverage we need for these high cost, but life-saving products. (You should know that NHF is working hard to get a bill introduced to raise life-time maximums; go to to learn more. They need your help!)

Novo Nordisk provided a grant to support the Summit, and it was great to see so many families returning from previous Summits. The event was planned and carried out by the amazing staff at CBCE. Thanks to everyone for making this event successful, useful and possible. If you have inhibitors, come to a Summit! The next one is in October in Birmingham, Alabama. If you know of someone with inhibitors, encourage them to register. The trip is free, and the knowledge gained is priceless.

(Photos: Rich Pezzillo and Sasha Cheatham; Dr. Manco-Johnson with the Wilkes family; Glenn Mones of NHF gives presentation; Kari Atkinson of Iowa and Laurie; Val D. Bias)

Great Book I Just Read: Miracle in the Andes by Nano Parrado
I had already read the book Alive by Piers Paul Read, and seen the movie, but they pale in comparison to this account, published 35 years later, in 2007, by the young man who actually walked out of the Andes, after 72 days in the most horrific conditions you can imagine. I could not put this book down. In 1972, a chartered plane crashed in the Andes, with a rugby team from Uruguay, which had been en route to Chile. The players were just boys, aged 17-21 mostly. How they survived and how they escaped is one of the greatest survival stories of all time. But this is a story of leadership, primarily, teamwork, and faith. After you read this, you might believe anything is possible. An enthusiastic four stars!

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