October 2011

Looking Into the Future in Chicago

While a winter storm gathered back on the east coast, I was in Chicago for a meeting with the Bayer Multidisciplinary Board Meeting. This is a group of thought leaders from the hemophilia community who meet once a year to discuss current trends in treatment, products and insurance, and predictions of what the community will look like and need in five to ten years.

I was unable to attend last year’s meeting at this time, so it was very nice to be back with the group, all of whom I knew. The team includes top hematologists, insurance experts, some adult patients, and homecare, distributor and nonprofit representatives. Of course, the hot topic is insurance! How are payers affecting the decisions being made now on treatment choices and product pipeline? For example, it’s already happening that payers are dictating brand choices in some places, said one participant. The impending merger of Express Scripts and Medco, two of the three largest PBMs, will radically upset the hemophilia apple cart and change the hemophilia landscape forever. Exactly how that will change was open to much speculation.

The most extreme prediction involved the fate of HTCs. One prediction is that in ten years there will only be half the current number of HTCs operating. There will be fewer hematologists. “We are victim of our own success,” expressed one doctor. Hemophilia has become so manageable that patients are less dependent on the HTCs. With cuts in federal funding, expect to see many close their doors forever. I saw this myself in New Mexico, where the HTC is struggling.

The bleak forecasts were accompanied by good news: treatments are better than ever, allowing a more normal life. Newer products, greater choice and who knows, maybe even cheaper products. Stay tuned–in 2012 I will provide a forecast by our community experts on what you can expect in the next ten years. It’s going to be a roller coaster ride, folks.

Interesting Book I Just Read
Sing You Home
Jodi Picoult

I normally don’t read fiction unless it’s a classic. This book was given to me as a gift. It tackles several hot buttons: gay rights, surrogate pregnancies, Christian fundamentalism. A music therapist—Zoe—finds herself childless at age 40, after a stillbirth. After her husband Max leaves her, Zoe eventually falls in love—with female counselor Vanessa. Eventually they wed in Massachusetts, my home state, and the first to make same-sex weddings legal.

Max, a recovering alcoholic, begins to attend a fundamentalist Christian church at the behest of his brother. The pastor is anti-gay. Max becomes a born again Christian.

The plot really thickens when Zoe asks Max to release one of their stored embryos to her so that Vanessa can carry their child, for the two women to raise; he counters by suing her for custody of them. So the book covers controversial issues as gay rights, evangelical Christians, in vitro fertilization, surrogate parenthood, and divorce. All this happens within six months! The story is from the points of view of three characters in alternating chapters. It’s a thought-provoking book on change, tolerance and what constitutes a family, when the biological parents have such different beliefs and life-styles.

Life is more complicated than presented here, however. Max became a born-again just in time as a set up to the ethical question of who should raise the kids. Christian fundamentalists seem very stereotyped. There are a lot of issues competing for the readers’ attention, and the ending is too neatly wrapped up, too quickly. But if you like to read, want a book to stimulate some thoughts about many current topics, this is one you can get through quickly. As an editor, I tend to be pretty harsh on how a book is written, but sometimes it’s nice just to read a different kind of book. Two stars.

Drought in New Mexico

I just returned tonight from a weekend visit to New Mexico, a state rich in history and desert beauty. I adore the Indian motifs everywhere and the startling bronze statue of an Indian warrior leaping out to greet visitors, an eagle soaring from off his hand. The tranquil beauty belies the trouble facing this western state.

I was invited to speak at the Annual meeting of Sangre de Oro (Gold Blood), the hemophilia foundation that serves New Mexicans with bleeding disorders. I was very honored to attend, and yet so saddened to hear of the struggles facing the members.

The Ted R. Montoya Hemophilia Program and Treatment center, established in 1980 by the New Mexico State legislature to identify and care for New Mexico residents with bleeding disorders, is facing severe budget cuts. Add to that a lack of staff as their long-time hematologist recently left. This is also a huge state, and many families live great distances. So the challenges they face are huge, particularly in this era of healthcare reform, in which we don’t know how this will all play out.

I must say the families who attended were delightful–especially the young boys who eagerly tried to answer my questions to the audience: How many people are there with hemophilia in the US? “1 million!” exclaimed one young teen. “100,000!” said another. (Correct answer: 17,000).

What impressed me most was the dedication of the staff, including executive director Loretta Cordova and president Johanna Chappelle (who I have known forever!). Great ladies, who with their team, are dedicated to advocacy to get funding and taking care of their members until things settle again. My deepest admiration to them all!


Good Book I Just Read
Doing Good Better: How to Be an Effective Board Member of a Nonprofit Organization
Edgar Stoesz and Chester Raber

This 1997 book has not lost any of its timely information on how to run a nonprofit effectively. It covers in a no-nonsense way how boards should form, act and what to expect as your nonprofit grows. The special emphasis on conflict resolution (conflict is good and necessary for growth) is excellent. There are also lots of templates for executive director/CEO performance reviews, and checklists for filing paperwork and board self-assessment. A quick read and very valuable! Three stars.

Recombinant VWD Product on the Horizon?

I just received word that Baxter has started Phase III of its recombinant VWD product. The investigational product is BAX 111, the first recombinant von Willebrand product in clinical development.

Currently, we have Humate-P (manufactured by CSL Behring) and wilate (manufactured by Octapharma), which are both plasma-derived. Wilate was the first product indicated for the treatment of bleeds in VWD patients. Humate-P has been the standard for years in the US. But don’t forget Alphanate (from Grifols), originally for treating hemophilia A bleeds, and now indicated for treatment of VWD patients (FDA-approved for surgical and/or invasive procedures in certain patients with VWD). Not FDA-indicated but sometimes effective is Koate-DVI (Grifols, distributed by Kedrion in US), because it has VWD in it, along with FVIII.

No one can say that America doesn’t have choice!

It’s always exciting when there are new products on the horizon. Please remember too that all US FDA-approved drugs are considered both safe and effective for treatment of bleeding for VWD patients.

The Baxter study will assess a minimum of 36 patients in trial sites in the United States, Canada, Europe, Australia, Japan and India. Information about the trial including enrollment is available at www.clinicaltrials.gov or by calling 1-805-372-3322.

I am quite sure, that when and if the product hits the market, Baxter will think of a catchy name for BAX 111!

For more info, call Marie Kennedy, (805) 372-3543– I know her and she is a very trusted source and nice person!

Interesting Book I Just Read
The Nine Rooms of Happiness
by Lucy Danziger and Catherine Birndorf

This perky, feel-good book uses a clever metaphor for getting women to think about their life, stress level and aspirations: think of your life as a house, and each segment of your emotional life is a room. Basement (memories, childhood), Family room (family), living room (friends, relationships), bathroom (self-esteem, health) etc. In which room are you? Where do you have the most problems? Is your bathroom too close to your family room? In a sense, the authors are asking us to compartmentalize, ironically a task usually associated with men. The authors use real life vignettes from women of varying backgrounds and situations to reveal some universal anxieties women share, and then applying the “house” metaphor to show how they can get unstuck from their unhappy situation, and move into a different room, and therefore happiness.

Sound simplistic? It is. The book is okay for those new to the self-help genre and studying relationship and introspection; I think they will read it and come away feeling understood, optimistic and less alone with their unhappiness. But to someone who is well versed in more serious relationship books (Harriet Learner has excellent ones), this is psycho-lite. The book is based on many presuppositions: woman must have female friends, you must keep old friends in order to be happy (even if they drive you batty?), women are prone to anxieties, women are unhappy. I found the book putting much blame on women for their condition, which I am sure the authors didn’t intend! For example, why should a woman be made to feel in “denial” about getting older, or narcissistic because she tires of her long-time friends who are aging, speaking about grave plots, and do nothing to engage in life, while she is full of spirit and wants to take on life and adventures? Why is that a “problem,” as the authors clearly state? They insist she needs to keep these stick-in-the-mud friends while cultivating new friends. What is she doesn’t have time? What if some people really are just jerks and not just a result of a relationship problem stemming from “regression,” or “transference” or “denial”? The authors seem to have a psycho answer for everything, and not a street-smart, pragmatic way of viewing relationships.

A cookie cutter response to each vignette wears thin, as do the clichés, which run rampant (oops, there I just did it) in the book. “To have a relationship, you first have to relate” –really? The model is a clever idea, using a house, and no doubt some will feel helped by this book. It’s gotten pretty mixed reviews. Maybe good for novices, but when you finish this, move on to some serious relationship books, as this one is pop-psychology, cutesy and sends mixed messages. If you challenge the presuppositions, half the book is sunk. Two stars.

A Bittersweet Celebration

I had the great privilege to attend a private event that was to have been a fundraiser for friend and US Senate candidate Bob Massie, of Journey fame. For you hemophilia young-ones reading this, Journey was I think the very first book on hemophilia ever written. Certainly the first written by parents of a child with hemophilia. The book details life for one family in the 1950s and 60s, who later became famous as parents Robert and Suzanne authored and published the classic Nicholas and Alexandria.

But back to the event. The entertainment for the evening was the humble, humorous and infinitely talented Noel Paul Stookey of the incredible group Peter, Paul and Mary, perhaps best known for their song “Puff the Magic Dragon.” I arrived early to the stunning home of Mark and Becky Levin in Boston–the traffic was light and the weather warm. What a great night to hang out in Boston!

Noel was so gracious and unpretentious. He and Bob have been friends for 30 years, since Bob was young. The group became friends of the Massies, and I recall from the book Journey, how Noel would sing to Bob when Bob was trying to survive terrible bleeding episodes. And here they were together again!

We were all very sad to hear that night that Bob would the very next day announce his withdrawal from the US Senate race. He gave it a good fight, and spent countless hours traveling the state, meeting everyday people, and rallying people to hear his thoughts about our country and his plans to represent Massachusetts. As someone who suffered, truly suffered, with hemophilia, then HIV and hepatitis, he really could speak out on issues of chronic healthcare. Bob is incredibly intelligent, down-to-earth, educated and passionate. He has already had an amazingly accomplished life, but still wishes to do more in public service. What a hero he is to us all!

He read his withdrawal speech, which included: “I know how hard people are struggling today. In a hundred speeches I have defended the right of every citizen to a good home, a good school, a good doctor, and a good job. I have also said that despite the difficulties we are facing today, we must not forget our opportunities and responsibilities for tomorrow.” And towards the end, “We all have a role to play in this. This is an extraordinary state filled with extraordinary people and we are capable of extraordinary achievements. I plan to play my part moving forward.”

We sincerely hope this means he will return to public life, for if anyone could represent the needs of those with hemophilia and chronic disorders, it’s Bob Massie.

After his speech, Bob and Noel played a song together; Noel on guitar and Bob on banjo. Guests offered their tributes to Bob and congratulated him on his efforts and praised him for his dedication. All in all, a wonderful evening, filled with gratitude to Bob, pride in our state.

To read Bob Massie’s Speech, please visit www.BobMassie.org

Great Book I Just Read
A Hole in Our Gospel
Richard Stearns

Stearns, the president of World Vision, offers an inspiring call to action to help the world’s poor. He attempts to answer the question, “What does God expect of us?” He does not let fellow Christians off the hook just because they go to church, know the Bible, attend Bible study classes and help their neighbors. He wants them to take part in a social revolution to save the world, specifically, to help those in need in Africa. Stearns shares his startling story about life as a high-rolling, wealthy CEO of Lenox, the provider of luxury china products. When tapped to be the next CEO of World Vision, Sterns had to make a difficult choice: uproot his five children and wife to Seattle, and take an enormous cut in pay and lose all his corporate perks and benefits. After much soul searching, he accepted. He shares what he has experienced in his job, along with his theology of Christianity in action.

It’s a compelling read, and certainly makes you look at your own life, your possession (how much do we truly need in this life anyway? What would we really miss if we gave more to help the poor?) and your beliefs about the poor. It’s not flawless, and some of the reading gets quite heavy-handed. A book review on The Gospel Coalition website says, “The Hole in Our Gospel is also theologically flawed and economically misguided… but its overall charge to care for the poor and put our faith into action is a good and necessary challenge.”

And despite taking a pay cut, Stearns still takes home a healthy $400,000 annual salary; World Vision is also not without criticisms of how the $800 million organization is run in the field. Still, you can’t doubt the author’s sincerity and depth of his calling. I don’t agree with his hero-worship of Bono; and the pages and pages and pages of glowing testimonials in the beginning actually cause the antithesis of purpose—I was very suspicious of any book that must call so much attention to itself. Why not allow the reader just to read and form their own opinion first without biasing them? But I read, and was profoundly moved as I had just returned from Africa, have seen what he has seen, and believe what he believes about social justice and about how we can each do more to help those in poverty. There’s a lot of theological interpretation you can argue, as he quotes and interprets the Bible throughout, but his social message is loud and clear, and he is a man who is walking the talk. He visits the poor; he strives to solve their problems; he does it with love; he makes results. Kudos to him! Three stars.

Biogen Idec Hemophilia’s First Advocacy Advisory Summit

Every now and then I am reminded how wonderful it is to be a part of the hemophilia community, if you can say such a thing knowing it means your loved one has hemophilia! I spent the day on Friday with a group of dedicated colleagues, some I have known for 18 years, so many I admire. Our hemophilia community is filled with such people, all trying to improve the lives of those with the condition.

So I was honored to be selected to attend Biogen Idec Hemophilia’s first Advocacy Advisory Summit on Friday, in Research Triangle Park, North Carolina. Arriving to much-welcomed balmy weather, I caught up with a cast of great people and friends, including executive directors Lynne Capretto (Ohio), Sue Cowell (N. Carolina), Kevin Sorge (New England), Kelly Waters (Virginia), Merlin Wedepohl (N. Cal), Kristian Prill (Washington), Greg Mermilliod (S. Cal), Vicki Jackson (Alabama), Sean Hubbert (Central Cal), Ken Trader, Joe Kleiber and Michelle Rice of NHF, and Susan Swindle of HFA. And special guests Dolly Shinhat-Ross of WFH and Margo Price of ISTH.

We were invited to “meet” Biogen Idec, learn about their clinical trials, and tour their plant. Jessica Swann, who many in our community know from her days as executive director of Hemophilia Association of San Diego, and then her days at Avigen and Bayer, is now Associate Director, Global Product and Market Development at Biogen Idec Hemophilia. She joins Dr. Glenn Pierce, Senior Vice President and Chief Medical Officer, who was twice president of NHF, formerly of Avigen and Bayer and who also formerly had hemophilia!

You may not recognize the name Biogen Idec: they do not have a commercial hemophilia product currently, but have two in the pipeline, in phase III clinical studies. You may have heard though about “extended half-life factor.” This is one company that is working on this.

When factor is infused into the blood stream to stop a bleed, it has a “half life.” Just as food is broken down and used, and you need to eat again, factor is used to stop bleeding and eventually broken down (degraded) in the blood stream and absorbed back into the body. The half life is how long it takes for 50% (half) of the factor to be used up. The idea behind extended half-life factor is to “catch” the factor when it is absorbed and return it back into the bloodstream before it gets degraded so it can still be used. Its half life then theoretically goes from 12-18 hours (currently normal half lives for factor) to up to 2-3 days or more. This may allow the protective effect of factor VIII and factor IX to last much longer than they currently last.

As a group, we tried to imagine what that would mean for patients: greater quality of life, fewer infusions, less stress and worry, greater protection. Make no mistake: extended half-life factor could be the next great thing in hemophilia.

Biogen is no stranger to me, but it is to our community because they did not continue its recombinant factor VIII program from 1982. It’s smart for any company to get familiar with the community before its product is launched. This company has a leg up as many of the people on its research, medical, clinical and marketing teams are from the community itself.

Still, Jess posed a question to our group of 35: How would you describe Biogen Idec? About 50% chose “Innovative,” while 19% chose “New and unknown player.” Yes, Biogen Idec is researching this new technology and new hemophilia products, but — who are you?

Biogen Idec is very familiar in the world of multiple sclerosis (MS). It has only three commercial products: Rituxan, Avonex (interferon beta), and Tysabri (natalizumab), which collectively had 2010 revenues of $4.7 billion. So it’s not a small company.But they have a big pipeline, with nine programs in Phase III (late stage) clinical studies, including extended half-life factor VIII and IX. Data just presented at ISTH in Japan this past summer looks promising.

After listening in the morning to presentations by Biogen Idec executives about the company, its history, products and mission, we listened to quite compelling testimonials from two MS patients, and a local executive director of an MS society. Some of us were stunned to be on the “other side” of the testimonial telling: usually we are the ones giving the heart-wrenching testimonials. It was stirring and emotional to listen to an articulate mother tell her story of MS, and how the Biogen Idec drug helps her to live a normal life. She also related the patient assistance programs that help each person affected, and how she has counted on this help for 15 years! She most definitely put a face to the company in terms of practical and compassionate care.

After lunch we toured the manufacturing plant. This is always fascinating to me. None of us are scientists, and yet we have some idea of what goes on in making factor. But we get reminded when we tour plants of the years and years of research that goes into preparing for manufacturing, the quality control that must be in place, and above all the cleanliness of the environment. I think this struck us all the most. As Joe Kleiber of NHF commented, the “cleanliness and complexities” of large scale production of factor is astounding.

Steve, our tour guide, stressed how in making such a product, the process needs clean water and clean air. Quality control is paramount: they measure the concentration of microbes in the air; they reduce human contact with the manufacturing process as much as possible (humans carry bacteria, and we were to purify our hands many times on the tour); all reactors have to be sterilized each time a new batch is made. The process needs pure water, to make steam, to clean the pipes and parts. He showed us software that displayed continually how the current manufacturing process was measuring up, using 50 different measures all combined into one measure, compared to historical data. Looking at a line chart on a screen, you could actually see the current production and how it compared over past weeks to a historical line. In this way, you can make adjustments needed to get production back on track. This was fascinating to us!

When we returned, we had many questions and the Biogen Idec team had questions for us. One piece of advice that was sincere and deep was not to overpromise the hemophilia community. New products are fantastic; we hunger for advanced products to help our children live more normal lives. But we have been burned in the past, when hopes were raised that a cure was around the corner.

The message was clear, for all companies: now that patents have expired and the race is on to bring new products to market, don’t build up hopes too high. Fortunately, we have great products now and our loved ones live good lives, especially compared to most other patients globally. We can wait and we want a realistic perspective. Still, there was excitement that a break-through product perhaps is not too far away.

In closing, Jess deftly asked us how we would now describe Biogen Idec? Not too surprisingly, almost everyone changed their original answer and selected, “Committed to the hemophilia community.”

I’d say that was a very successful first hemophilia Advocacy Advisory Summit.

For more info: www.biogenidechemophilia.com

Thanks to Biogen Idec for the tour and time, and thanks to all my colleagues who made for such a great meeting and day.

Good Book I Just Read
The Life You Can Save
Peter Singer

This compassionate and thought-provoking book on humanitarianism is nonetheless by a philosophy professor who once wrote that there is an argument to be made that aborting a fetus known to have hemophilia is not ethically wrong, in his book (which I have not read) ‪Writings on an Ethical Life‬. I am not going there now, but will say this book was good. Singer’s very basic premise is that most of us would not hesitate to help a child drowning in a pool, even if it were inconvenient (missing a bus, ruining our clothes), but we blindly turn from the 27,000 children who die every day from preventable causes in developing countries. He sets his case well, wondering why we do not easily give up buying things we don’t need in order to help others to live. He explores our wariness with charitable organizations, and challenges our rationalizations for not giving. This is a good book, not without its flaws in the argument, but worth reading and a very quick read. Recommended for those who are compassionate, who want to influence others to be more compassionate and giving, and for those in healthcare globally. Three stars.

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