Long-lasting factor

The [Infusion] Shot Heard Round the World

Unless you were watching the new streaming releases of Season 6 of Breaking Bad on Netflix (for which I might excuse you) or were abducted by aliens (for which I will not) you must have heard the news….. the first extended half-life factor has been approved for commercial sale. It’s the hemophilia B drug APROLIX. The news was announced at the HFA meeting in Tampa last Saturday, and it truly is the Shot Heard Round the World (an appropriate reference to the American Revolution). This ain’t just a new product, folks; it could shake up the industry. Physicians are watching; patients are watching; Wall Street is watching. What will the “uptake” be? 

Uptake is how many patients will switch to using a new product. The hemophilia community tends to be a “wait and see” creature, perhaps due to our devastating experiences of the 1980s. When Advate was launched in 2002 (yes, drugs are launched, like ships), investors predicted a rosy 20% uptake, but initially it was more like 12%. As the risk-takers who switched early reported good results, more and more patients switched. But Aprolix is truly a different kind of drug. Not just a new generation, it is a new type of hemophilia drug: an extended half-life, meaning fewer infusions for the same results. 

And Hemophilia B has not had a buffet of drugs to try like hemophilia A. There was only one recombinant (you all know what that means, right?) FIX for years, until October 2013, when Rixubis was approved. With extended half-life factor, prepare for some major shifts in our landscape. Much may depend on the price, which no one is talking about. If it’s too high, will insurance cover it? 

And what about individual results? Will each patient respond well? Advate had major hiccups when there appeared to be an increase in inhibitor formation in users. Was this due to higher scrutiny of inhibitors with a new product? The initial scare seemed to be due to increased scrutiny. And these are among the most scrutinized things on the planet.

And lest we get too carried away, don’t forget there are other products in the pipeline by other companies, some extended half-life (the products, not the companies, though we hope they are long lasting too). Basically, the news about Aprolix marks the beginning of a new era in hemophilia. Maybe a Revolution, maybe the Renaissance. A Hemophilia Renaissance. 
Below, an article in its entirety about the news….
U.S. FDA approves Biogen’s hemophilia B drug
Alprolix
Biogen Idec Inc has won U.S.
approval for its long-acting hemophilia B treatment Alprolix, the U.S. Food and
Drug Administration said on Friday.
Hemophilia B is a rare, inherited disorder in
which a person’s blood does not clot properly, which can lead to prolonged
bleeding and bruising. It affects about 4,000 people in the United States and
25,000 worldwide.
Alprolix is a bioengineered version of the blood
coagulation factor IX, a protein needed for normal blood clotting. Biogen is
developing the drug in partnership with Swedish Orphan Biovitrum AB. It
received approval in Canada a week ago.
Hemophilia drugs in general must be infused two
to three times a week to prevent bleeding episodes. Biogen’s drug needs to be
administered once a week or once every 10 to 14 days.
The company is also developing a long-lasting
drug, Eloctate, for Hemophilia A, a more common form of the disease, that
affects about 20,000 people in the United States. Patients with hemophilia A
lack or have reduced levels of coagulation factor VIII.
Current treatments for hemophilia B generate
about $1 billion a year, according to Biogen. The market for hemophilia A
therapies is worth about $6 billion.
Eloctate would compete with Baxter International
Inc’s drug Advate. Baxter is seeking to develop its own long-acting version but
Biogen is expected to be first to the market.
Alprolix is expected to generate sales of $286
million by 2019, according to Thomson Reuters data. Eloctate is expected to
generate sales of $1.1 billion.
(Reporting by Toni Clarke in Washington; Editing
by David Gregorio)  Fri Mar 28,
2014 5:11pm EDT
For more info:
http://www.alprolix.com/

Infuse Less, Play More


I spent this weekend in Newport, Rhode Island to attend the Bayer Multidisciplinary Board meeting. This is a group of representatives from the community (from home care, NHF chapters, manager care, pharma, HTCs, consumers) who get together twice a year and brainstorm, share and offer opinions to the marketing team at Bayer HealthCare. Most manufacturers have these, and even some home care companies. These forums are a great way to learn what is happening in the community in an informal lieu, with intelligent and proactive individuals.

Now some of what we discussed is confidential, but the most exciting news is not confidential. It’s about the clinical studies for the longer lasting factor VIII product. The study is moving into phase II, with 250 patients from many countries participating. Patients are treated with a factor product each week in this study, but the study is double-blinded, which means that neither the physician nor the patient knows whether they are getting regular factor or the longer acting factor. This is the largest clinical study in hemophilia history, I believe. Results are promising: some day I believe we will have a factor product that can be infused once a week, but keep factor levels high the entire week, eliminating the need for three time a week infusions.

Bad News: Tomy McNulty, chief clinical officer of Novologix, a consulting firm, updated us on the payer side of the reimbursement crisis. He affirmed what we announced back in 2004: the system of reimbursement for hemophilia products has changed permanently; home care will continue to consolidate; hemophilia consumers will no longer have complete choice of product, physician or provider. Of the three, provider choice is of the least concern to the payer. In other words, you’ll use payer-designated home care X and like it. We’ve already seen two home care companies go out of business: who next?

Good news: Bayer unveiled for us a new website called “Living Beyond Hemophilia,” for teens and young patients with hemophilia to help them through their transition to adulthood. It’s an excellent site, with a career assessment form, thought provoking questions and answers) about how to prepare for a first job interview, how to prepare for college, and even internships that may be of interest. Having a 21-year-old still in college an struggling to live on his own, this is the kind of site he can go to again and again to get tips on being prepared for what life brings. Check it out http://www.livingbeyondhemophilia.com/

Great Book I Just Read: The Picture of Dorian Gray, by Oscar Wilde
I am ashamed to say I never read anything by Wilde other than his pithy quotations. (His last words on his death bed are rumored to be: “Either these curtains go, or I go.”) This is the only book he ever wrote, and it is masterpiece. Considered one of the last books of the Gothic horror age, it is also a scathing summation of upper crust British society, which in the 1800s is obsessed with appearances–the appearance of being wealthy, beautiful, talented. The book asks, and answers: What does a life of pure hedonism and egoism do to the soul?

Dorian Gray is by all accounts a stunningly handsome man, from a wealthy family, and yet innocent at heart. Noting his handsome face, an artist creates his portrait, a chillingly accurate representation. With constant adoration of it and of Dorian himself from the adult men, Dorian eventually wishes that he always look like his youthful appearance in the portrait, and never age. In a Faustian bargain, the wish is granted. Dorian remains eternally youthful, while the portrait ages, and not just ages, but mirrors the deterioration of his soul as Dorian embraces a life of extreme and callous hedonism. Just like every person with a dark secret, he hides his portrait from all eyes. But this secret eats away at his humanity. Without any physical or visible consequences of his wretched lifestyle, he continues to sample every vice there is, earning the condemnation of his friends and of society, who yet still envy him his eternal beauty! Eventually, his lifestyle impacts others deeply (there’s a murder, suicide, etc), and finally causes him to ponder what he has become. No matter how much he hides his wanton lifestyle and feelings, the portrait reflects greed, suffering, hatred, extreme consumption, lack of purpose, narcissism, and amorality. The portrait holds an iron grip on his soul. Wilde is an interesting writer: while the dialogue appears to ramble at times, and there is a lot of overt melodrama, Wilde is, after all, a playwright. It’s Wilde’s command of the English language that is pure joy: razor sharp, line after line; I found myself ingesting his richly nourishing ideas, strategically placed words and flowing prose. Four stars!

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