June 2014

Pulse on the Road in San Diego!

Pulse on the Road is our three-hour insurance symposium that updates families with bleeding disorders on insurance reform in their state, the Affordable Care Act (ACA), and how to compare and research insurance policies. Sponsored by Baxter Healthcare, implemented by us, we bring expert speakers, like NHF policy expert Nicole Quinn-Gato, to families around the country.

This week, San Diego! We had a cozy gathering of about 40 family members at the San Diego Zoo, minus about 40 kids who went off with “Birdman” for the kids program. Seated in the Treetop Conference room, families listed to our speakers all morning, while Spanish translation was provided by specialists to our Latino families.

Elizabeth Stoltz, Senior Manager, Healthcare Economics & Reimbursement at Baxter Heathcare, presented an overview of the Affordable Care Act, including the many benefits and things to watch out for. It’s 2014, and about 8 million Americans have enrolled in the Marketplace—the on-line place to shop and compare insurance plans. Remember, it’s mandatory now that every American have health insurance or face a penalty.

Elizabeth Stoltz of Baxter Healthcare

I next spoke about the importance of choosing your own plan. While there is a lot of technically policy stuff to cover in these symposia, I aways stress to the audience we teach this to save you money, out of pocket expenses. And to emphasize this, I ask a question after each speaker, relevant to what they just presented on. Whoever gets the right answer gets $10! This wakes up our audience, creates a friendly competitive spirit, and is just plain fun!

Our audience got so into this game that even Robert, the man who set up the translator booths—and has nothing to do with hemophilia—answered one of our questions correctly first! Of course, he didn’t get the $10 as he is not related to hemophilia!

Laurie helps Taylor with the Marketplace
Mateo reads a My First Factor Book!

And lastly, Nicole gave a one hour live demonstration of “coveredca.com,” California’s marketplace. Wow, what a challenge! The exercise showed how complicated the California Marketplace is; it was a tough exercise, but I was proud at how families stuck with it, got the answers they needed, and ask us all for assistance in locating certain pages on the site. We had provided laptops for each table and they were all used well!

Lunch was served afterwards, where we got a chance to socialize with the families. This was then followed by goodbyes and a visit to the largest zoo in the world, compliments of Pulse on the Road!

Next stop? Houston in August!

Be a Hero!

To continue with our updates of programs and services from Pharma, here’s a new and exciting one!

Jeffrey Leiken Master Trainer at Evolution Mentoring

Remember when you were evolving from a teenager to a young adult – trying to assert your independence, work through all the social politics of high school, and figure out the direction of your life? Then consider the challenges of living with hemophilia on top of that – it can add a whole other layer of complexity. That’s why Novo Nordisk is offering HeroPath™ to help youths between ages 15 and 20 better navigate this transitional life stage.

HeroPath will be led by Jeffrey Leiken, Master Trainer at Evolution Mentoring and creator of HeroPath – you may remember his passionate speech about life transitions and personal growth at National Hemophilia Foundation’s 2013 Annual Meeting. HeroPath  will focus on helping participants grow their skills for success in areas including relationships, education, and careers.

Here’s the exciting part: Up to 25 selected US participants with hemophilia A or B* between ages 15-20 will have the opportunity to work directly with Leiken, his team of expert coaches, and their peers at a kickoff weekend in Chicago from August 22-24, as well as continue to receive personalized monthly coaching for the following year.

Visit http://www.changingpossibilities-us.com/HeroPath to complete your official HeroPath™ application, including several multiple-choice questions and an up to 150-word explanation of why you are a great fit for the program. Prefer not to write? Provide a 60-second video explanation instead – just upload your video to YouTube™ and paste the URL into the application form!

All entries must be received by 11:59 p.m. EDT on Thursday, July 17, 2014. Leiken will select up to 25 participants based on responses to the multiple-choice questions and the level of creativity and original thought conveyed through the essay or video entry.

What parents need to know: HeroPath™ isn’t just a weekend retreat – it’s a yearlong program designed to help offer teens or young adults the tools to identify:
·        What direction to move in professionally based on interests, strengths, talents, and natural proclivities
·        What kind of people to associate with personally and professionally (Leiken and his team will help participants give voice to their own values)
·        What kind of person they want to be

HeroPath™ is also structured to help develop their ability to manage their state, stay focused, and perform under pressure to help make better decisions.

Questions? See Complete Official Rules at www.changingpossibilities-us.com/HeroPath for details.

*Participants cannot be on a federal health care program.

This sevice announcement was sponsored by Novo Nordisk.

News from Pharma

So much has been happening in the hemophilia community lately, I decided to post some news releases about new products and new services from three different pharma manufacturers. Last week we shared the news about Biogen Idec’s Eolctate approval; below are more interesting items, straight from the horses’ mouths.

From CSL Behring
New–CSL Behring’s world-class biotechnology manufacturing facility in Broadmeadows, Australia. The facility is for late-stage production of hemophilia therapies and is one of the largest and most advanced of its kind in the world. Leading-edge science is at the core of the $250 million expansion, which is expected to drive long-term growth in CSL Behring’s promising bleeding disorders portfolio.
In particular, the facility will produce novel recombinant therapies on a large scale for international clinical trials. Our recombinant technology uses a unique recombinant-albumin-fusion platform, which we believe could offer distinct clinical advantages and improve patient experiences.  Suffice it say, CSL Behring’s steadily-rising research and development spending has played a key role in developing these and other new technologies. For example, in 2011/2012 we spent $368 million on R&D (about 8% of total sales). And over the last 5 to 10 years, we have advanced an enviable pipeline with exciting product candidates that may offer enormous treatment benefits. 

From Baxter Healthcare

Mobile video gamers looking for the next app for their smartphone or tablet now have an option developed specifically for the hemophilia community. Baxter Healthcare Corporation has created a free multi-game app, called Eco-Trek, primarily for people between the ages of 15 and 32. Yet based on the reactions of pre-release product testers, Baxter expects that Eco-Trek will appeal to people of all ages, from adolescents waiting to be seen at hemophilia treatment centers, to older adults that enjoy competing in video games. Eco-Trek, which is available to everyone regardless of their treatment, is the first socially connected video game designed for the hemophilia community. With hemophilia-specific content and Facebook score-sharing built into the game, Eco-Trek is a first-of-its-kind mobile app. A national leaderboard will create competition among players in Eco-Trek’s three online adventures, each of which have 10 levels:
·     Bushwhacked! – Find your way through a forest maze without losing supplies from your backpack
·     Alpine Summit – Compete in an uphill race against the clock, dodging obstacles along the trail
·     Roughin’ It – Set up camp and complete specific tasks while pesky critters try to get in your way

“We are really excited about Eco-Trek because it’s simple, interactive, and offers important educational information about hemophilia A and ADVATE [Antihemophilic Factor (Recombinant)], while you’re competing in fun activities,” said Jeff Schaffnit, senior director of US Hemophilia Marketing. “For teens and young adults with hemophilia, this type of format can make it a lot more fun to gain knowledge on hemophilia.”

Eco-Trek is a free app available for download from the Apple App Store and Google Play. For more information, contact your Baxter representative.

From Pfizer

Pfizer understands that health care costs can be a challenge for those with hemophilia and their families, and strives to provide access and patient-centered support. Therapy is just one part of the equation, which is why we offer financial support for eligible patients as well as educational programs for patients with hemophilia who infuse with Pfizer Hemophilia Recombinant Factor products.

We’re pleased to share with you three program updates and hope that you will help us share this news with the hemophilia community. As of April 1:

•    Pfizer increased the maximum cap on its Pfizer Factor Savings Card
Program. Eligible patients can now save up to $5,000 annually on copay, deductible and coinsurance costs. Terms and conditions apply. Visit PfizerFactorSavingsCard.com to learn more and fill out a brief registration form. If you have any questions about the use of this Pfizer Factor Savings Card, please call 1-888-240-9040 or send questions to: Pfizer Factor Savings Program, 6501 Weston Parkway, Suite 370, Cary, NC 27513.

•    To continue to meet the changing needs of today’s patients, Pfizer is consolidating its various prescription assistance programs, formerly known as Pfizer Helpful Answers, into a new comprehensive program called Pfizer RxPathways. Pfizer RxPathways is a family of prescription assistance programs that provides eligible patients with financial support and reimbursement support services. Access the program today by visiting PfizerRxPath.com.

•    The Soozie Courter Hemophilia Scholarship Program, a tuition assistance program
for students with either hemophilia A or hemophilia B, is now accepting applications for the 2014-2015 school year. To apply, please visit http://www.hemophiliavillage.com/resources-support/scholarship-assistance 

Another Product Debut: FVIII This Time!

We were hoping for an August approval, but it seems the FDA is making Biogen Idec’s day. Two products approved in two and a half months! Both with extended half-life. Eloctate [Antihemophilic Factor (Recombinant), Fc Fusion Protein] is the newest factor VIII product.

A new era of hemophilia treatment has begun. I read somewhere that the last truly “new” product that was introduced was Advate, in 2002. True?

The hemophilia industry is big, with much at stake. The introduction of two new products, with distinct and so far unique,  differences, may cause a shake up. And more products, with longer half-lives included, are coming.

More products give us more choice.  But you have to know what it is you are choosing between.  I have to say I don’t think hemophilia parents and patients are as well informed as they should be. I just conducted a recruitment of patients and caregivers this past week, looking for those who use “plasma-derived” products ONLY. I couldn’t believe the number of people who called to participate who use recombinant. They asked: what does plasma-derived mean? Isn’t the product I am using plasma-derived because it contains Albumin? Or worse, arguing with me that a recombinant product is plasma-derived.

We’ve written so many articles describing the difference between the two categories. All factor is either plasma-derived (originates from human blood) or recombinant (from human genes in the lab). The new product is a recombinant product manipulated to last longer in the blood—basically through a longer half life.

One savvy mom I’ve known for year, who has a son with the World’s Most Stubborn Inhibitor, wrote this to me: “It’s important for Biogen Idec to make clear that this product has not been tested in Previously Untreated Patients (PUPs), and the risk of inhibitors is highest in this group.”

Biogen Idec will be opening up a clinical trial for PUPs and I am encouraging anyone who wishes to use the Alprolix (long-lasting recombinant FIX) in a previously untreated patient, to wait and enroll in that trial, so Biogen can gather the appropriate data to understand how safe the product will be in PUPs.

Please always speak with your HTC hematologist to learn which product is best for you or your loved one. (And I don’t usually use underlines in my writing! Yeah, it’s that important)

To learn more about Eloctate, visit:


Eyes Wide Open: The Boy from Burma

I was in Australia May 8-15, attending the World Federation of Hemophilia Congress. I had the pleasure of meeting a young man named Aung, from the Asian country Myanmar, formerly known as Burma. Aung had written to me months ago requesting sponsorship to the event, plane tickets that cost about $2,000. I am asked frequently for assistance and need to bank my money on people and programs I think will give a return–not to me personally, but to the cause of hemophilia. Myanmar is not a member organization of the WFH and therefore not eligible for funding to the Congresses. Kind of a Catch 22–the countries that need to be there can’t be there unless they have a hemophilia organization. These countries can’t get help forming a society, because they don’t have a society!
But one of my specialties is trying to get organizations formed in countries where none exist in the hopes that one day they will be accredited by WFH. It’s a great partnership which sometimes works! In this case, something about Aung struck me. A young man in a rather isolated country, who hasn’t walked in 7 years, in a wheelchair, willing to for the first time fly to another country, all for hemophilia and his people. That’s courage. He impressed me so much we helped fund him; please read his thoughtful and beautiful essay below and prepare to be impressed. And to realize how lucky we are in developed countries. We live in two worlds—the haves and have-nots, and the have-nots are only asking for the know-how to learn to stand on their own—literally and figuratively.
Aung with Fred Wensing of Australia

My mother’s worries perhaps foresawthe difficulties ahead. It’s not an easy task for me to deal with the airline
staff who told I am not allowed to travel on the flight because I have no
companion to look after me – because I am disabled. However I had a hard time
with my anger management in my teenage years especially on discriminations
against haemophilia and disability, I showed great demeanor in engaging the
conversation with the staff of the airline. They finally said they would make a
call the captain if he allowed me to be on the plane. It was a huge
disappointment I have ever had on the last minutes before the flight on the
airport. But this time I managed as my best to stay cool and wait for the
response, which was positive.

Just as I have come out of the
airport main building, I found Fred Wensing and his wife, Maria. It’d be funny to say
but I had never seen his photo before and had no idea how to find him. Luckily,
he found me instead. The wheelchair at least made it easy for him to spot me in
the crowd.

He and his wife took me to the hotel where I was staying during the Congress days. This is my first trip outside of my country and I have to say everything I perceived in Melbourne is total opposite of what my country has in a positive way. There’s no disabled-friendly sidewalk in Yangon, let alone the other parts of the country. There’s proper treatment for people with haemophilia except blood products which, according to my experience of catching malaria, are really risky. The main difference between Australia and Myanmar is economic conditions, no doubt. But the attitudes of the people here, especially the attitudes on the disability are what I would call really civilized. Whenever I go in Melbourne, I was never welcomed by the quizzical look saying “Why this guy in wheelchair is here?”  Attitudes are not about material power or economic prosperity of a country. It’s about
education and nature, I believe. Even if so, there will be a lot of decades
before I will be seen as a person, not as a person in wheelchair.

The exciting Congress days were ahead. I was not able to sleep until 3 am on my first day of staying at Travelodge. My head was flooded by the seemingly unstoppable thoughts of what I would do and who I would meet in the Congress. It was 1:30 pm when I woke up. I
ran, of course in the motorized wheelchair, to a food shop nearby and had my brunch. The shop sold Indian food and I found for the first time that Myanmar is a cousin of India when it comes to cooking dishes. Their curries greeted my taste buds as old buddies do to each other.
It was the first day of the Congress and I planned to go to the Congress only for the opening ceremony. Fred appeared at the lobby at 5 pm. With Aboriginal music and dance, the Congress was wonderful. I met some people who have haemophilia including an elderly person, who has pseudo tumor from haemophilia, a teenager, whose legs becomes paralyzed after being injected painkillers through his spine, a severe haemophilac from Tasamania who has inhibitors and joints with bleeds. I met with some other people but the image of these three people left on my mind.
Back in my country, I always had this feeling of being alone before. I found no one who is put into wheelchair by haemophilia and related causes.
Second Day at the Congress
Second day was probably the biggest day of my life and the opening of my adventures. I went to the Congress alone in the middle of the walking crowd who were going to all the directions. I can enjoy the view of the Yarra River along the way to Melbourne Convention Center while enjoying the freedom given by the wheelchair-friendly environment. I went to the session, Management of Hemophilia with Little or No Factor. I had a conversation with one of the speakers, Dr. Daniel Hart from the U.K, on my health problems with blood products. According to him, some form of antibody might have developed in my body due to the prolonged contact with FFP or cryoprecipitate whenever I have bleeding. These antibodies make the treatment with blood products less effective and my immune system is prone to fever and cold when I have blood products as a result. It makes sense of the problems I am facing
when I inject blood products.
Appointment with a haemophilia specialist
I had an appointment with the doctor after the session. Although I am seeing haematologists often in my country, they don’t seem to be able to give me a solution to cure the bleeding episodes of my knees, which turned me into a wheelchair user when I was 14. My intention to see the doctor at the treatment room of the Congress is to consult with him about my knees. The doctor told me it would take approximately $100,000 to have knee replacement operation to be able to walk. It might be a ray of hope although I do not have any financial strength for such expensive
The doctor also recommended me to see a physiotherapist in the treatment room. I have never thought physiotherapy played a role in giving treatment to people with haemophilia before. The physiotherapist advised me to have some basic exercises in the water. The very important thing, she said, is to do the exercise slowly and to the limit my joints will not bleed. It will be a little bit difficult for me since I have to practice on my own. But I will have to try some of easy exercise. My muscles have suffered atrophy over the period of 7 years I haven’t been able to walk. Hopefully, regular exercise and diet will make the muscles and joints of my legs fit for some movement.
Wheelchair Talk
I saw the man from Tasmania again
and we headed to a restaurant for our lunch together. He is 33 years now and he is in wheelchair because of his damaged joints. Inhibitors in his body made it difficult for him to undergo a joint replacement therapy. On the way to the restaurant and at the restaurant, we were engaged in the conversation ranging from relationship and romance to our favorite music. He said he has been going out with a female friend recently but he doesn’t want it to evolve into a relationship. “I don’t want her to babysit me, you know.” I could
give him nothing but an emphatic nod. I know what it feels like to be in wheelchair and to have romance at the same time. Anyone who has been in
wheelchair for seven years will know it too.
We continued to talk about our lives and at one point he asked me if my government gives me a disability pension. It is a new word to me because our country does not have any social welfare service offering to its citizens. Luckily, I have earned money for my personal expenses from freelance translation and writing this year. I will have to thank to my parents for keeping me at the university until I graduated. I departed
him for the next sessions of the Congress after I finished my meal.
I would not forget the moments of struggles with the manual wheelchair on the way back to the Melbourne Convention Center which
was a couple of blocks away from the bar. I was going back to the Center alone and the tense feeling is running along my arms while I was pushing the wheelchair myself. I blamed myself for not noticing the steep ground towards the Convention Center. On the way up, I felt as if my arms were going to explode from the cries of muscles. But I felt the need to prove myself that I am independent and I am capable of going to where I want. Of the things that I have built, self-respect is a real ingredient of my life. I possibly cannot survive all the physical pain from haemophilia and the chronic depression without the sense of self-worth to my own existence and capability shaped by this existence.
But it was not all my effort that took me to the destination back. I was stuck at some places along the way and I would not get
back to my destination successfully without the support of the nice people among the pedestrians.
Experience Sharing
There was one interesting session waiting for me
at the Congress. There were many speakers sharing their experiences such as a
doctor, a community leader and a person with haemophilia under the main topic,
In Our Backyards: Eliminating the Gap in Care Between Developed and Developing
Worlds. As someone who comes from a country with 2 percent of the budget into
healthcare sector, I have concerns for the people with haemophilia in Myanmar.
We have to pay for all the expenses of the treatment and it is extremely
difficult even for the middle-class people like my parents. There will be much
more people in rural area who are not diagnosed with haemophilia and who passed
away because of haemophilia. Personally, I heard the sad stories of the people
with haemophilia who passed away for the inadequate treatment. After I managed
to land the mic calmly in my hands, I raised the question to the speaker on the
difficulty we faced in forming the organization from the start with no data for
haemophilia and related bleeding disorders as well as the hospital with expensive blood products and no factor products. I caught curious eyes from the audiences while I was talking about Myanmar on the mic. In the rest of that day, people came and asked me about the conditions in Myanmar. I cannot bring better treatment, better living conditions and better lives to the people with haemophilia in Myanmar myself alone. But I can spread all the information to the global community and in this way, I might be able to find somebody who is willing to work together with us to find a solution for approximately thousands of people with haemophilia in Myanmar.
Learning things with open eyes, Meeting
people with open heart
I love learning, one of the things that give you a reason to live in the world. The very first word of English I learned obviously is “Haemophilia”. When I had access to the Internet four years ago, I typed the same word in Google search box. I still want to learn more about the bleeding disorder with which I will be living until my last breath. Some of the sessions of the third day are interesting to me although I may not be able to apply the knowledge from them to me or to my friends in Yangon since they target mostly to the people in the developed world. But I acquired medical knowledge such as what happened when blood is in joints, and how to manage haemophilia with no factor products. There is hopeful
news such as the half life of the new concentrate products become longer, which means the treatment will be cheaper with less amount of concentrate used. Maybe prophylaxis treatment will be available to us in the coming decades.

Aung with Laurie Kelley
I had a fantastic meeting with the board members of the Haemophilia Foundation ACT on the third day of the Congress. Although we live in different lands and cultures, we share a common identity – haemophilia. It was never difficult to talk with the people throughout the Congress. Board members of HFACT are interested in the conditions of my country and me. I received great advices from them in running a haemophilia organization. Their organization was formed 30 years ago and they have been making progress with the care and treatment of haemophilia during
these years. But luckily, as Fred put it, we will not have to wait for 30 years
because science and technology are waiting for us.

I had a meeting with the lady from the US, Laurie Kelley, who sponsored my air tickets.  Project SHARE, organized by Laurie and her team, has a positive impact on the haemophilia communities in developing countries. I had talked with her on the possibility of her visit to Myanmar. She said she might visit Yangon during her trip to South East Asia in September. I hope to be able to work with Project SHARE and Save One Life
organized by Laurie for the sake of people with haemophilia in Myanmar in the future.

Aung with Alain Weill, president of WFH

I also had a conversation with the president of WFH, Alain Weill, at the social event, Taste of Australia. He is a good listener. He promised me that he would make sure the people in Myanmar can have the treatment of factor products donated by WFH after the membership application of Myanmar Haemophilia Patient Association is accepted by WFH. My country, Myanmar, has been out of the map of the global haemophilia community
until now. I am probably the first person with haemophilia from Myanmar that has been to a WFH congress. It is very important for all the people with haemophilia in my country to have a representative in the Congress.

I also found the people from the neighboring countries of Myanmar during the Congress days from Malaysia, Indonesia and Cambodia. We also showed interest in forming a haemophilia society for ASEAN. We would be able to lobby the ASEAN governments to improve
the care and treatment of haemophilia as one voice. I will keep in touch and work with these people to form an ASEAN Haemophilia Association in a few years.
Goodbye, Melbourne
The Farewell Dinner also gave me a perfect condition to make friends with the leaders from haemophilia communities throughout the world. I talked with a president of the Vietnam Haemophilia Association –another contact from ASEAN, young people from Australia and New
Zealand, and the doctors and nurses specializing in haemophilia.
Before I came back, I took a tour around the city, Melbourne, in wheelchair. The streets were teeming with people of different sorts. The shops were full of souvenirs. There were street
musicians entertaining the pedestrians. Even the city was dancing along the tunes of the street musicians.
Having haemophilia and living in an isolated country, I always have the feeling of ‘only me’. This is the first time in my life I have seen so many people who have haemophilia at one single
place. Their happiness and motivation diffused to me. Along with other gifts
from Fred, Maria and their son, Damian, Melbourne gave me another gift – courage.
A courage to live with haemophilia and be a community leader for the people with haemophilia in Myanmar is probably the most important gift I have received from the Congress in Melbourne.
Kaung Myat
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