Mr. President, Meet Hemophilia

You know all the intense town meetings with President Obama that have focused on health care reform? One was held in Denver yesterday, and our own Nathan Wilkes introduced President Obama to the attendees and to hemophilia. The New York Times writes:

“At a town-hall-style meeting in a high school gymnasium here on Saturday, Mr. Obama was introduced by Nathan Wilkes, whose family nearly lost their health coverage after costs to care for his 6-year-old son, Thomas, who has severe hemophilia, approached the $1 million lifetime policy cap….In introducing the president, Mr. Wilkes fought back tears as he described the birth of his son in 2003, and the first question the doctor asked: “Do you have good insurance?” Mr. Wilkes told of how he ‘searched frantically’ for a new policy when his son neared the $1 million cap, and how a social worker suggested that he and his wife divorce, so their son might qualify for Medicaid. Eventually they found coverage, with a $6 million cap.”

Nathan and Sonji Wilkes are colleagues and friends: Sonji is a columnist for our newsletter PEN. They are both very active advocates for health insurance reform, and Nathan ran for a political office last year. Their son Thomas has an inhibitor, and Sonji is one of our peer reviewers for our new inhibitor book coming out last this year.

This was the town meeting in which Obama sited his grandmother’s death, and in which the university student Zach Lahn asked how private companies could possibly compete with the government on health care insurance. The NYT writes that Obama replied, “‘The notion that somehow just by having a public option you have the entire private marketplace destroyed, is just not borne out by the facts,’ Mr. Obama said, adding that ‘UPS and FedEx are doing a lot better than the Post Office.'”

Congratulations to Nathan for his select role in introducing the president, and thank you for representing the entire community at such a crucial time.

Great Book I Just Read: The Brief Wondrous Life of Oscar Wao by Junot Diaz
This New York Times best seller and Pulitzer Prize winner traces the story of Oscar, a fantasy, Dungeons and Dragons and sci-fi fan who wants to be a famous author. But we meet his entire Domincian family, three generations of the DeLeons–Oscar’s mother Beli, his sister Lola, his grandmother La Inca, and his friend Yunior– and in exploring their troubled, complex relationship, we also explore the culture and history of the Dominican Republic. Diaz writes explosively, with a hip edginess, directed right at the reader. He weaves into the story the violent history of the DR, its language, its beliefs, its people, its culture, the “Fukú” –the curse– and why Dominican men behave as they do toward their women. And Oscar is so un-Dominican: overweight, can’t dance, no social skills. He longs for a girlfriend, and comes squarely up against a culture of machoism. As someone who has been involved with the DR for 12 years now, I learned a lot more about the island and its people than I already thought I knew. It’s hard to characterize a culture but Diaz has adeptly done it in an entertainingly dark way. Four stars.

Saving Elephants in Florida

I had an experience on Saturday that really speaks to this community and its dedication. For some strange reason, I was reminded of a scene from Animal Planet in which a baby African elephant was stuck in some mud. Her entire herd, including her mother, rallied around the calf to assist her until she was freed. Naturalists were amazed by how communal and compassionate elephants are.

So a 30-year-old man, not a baby elephant, called my cell phone at 10 am Saturday. “Jack” has mild hemophilia and has never self-infused. He also has no insurance and was in the middle of a raging psoas bleed. My son has had those and they are excruciating. I felt for him. I could not ship him donated factor through Project SHARE. The best I could do was to provide numbers: PSI, ACCESS, Hemophilia Hotline (Do you have those numbers handy??). He went to an HTC on Friday (his first time, I think) and was given some free factor, but not enough to last him a full week. Worse of all, there he was at home, still bleeding, and he needed an infusion that day. And he didn’t know what to do.

No way would I coach him over the phone on that. So I called some friends, and fellow “elephant” mothers: Natalie, long time friend, on one side of Florida; Linda, executive director of the Florida Hemophilia Association, another long time friend, closer to where the young man was located; Kim Madeiros, executive director of Factor Foundation of America, also close to where he lives. What happened was amazing!

Natalie took my call and wanted to help, but was too far away. Kim returned my call– from the beach. She was out having fun with her son and her 14-month-old daughter. No matter. Bleeding patients come first. I could hear the roar of the waves as she spoke, her children squealing in the background. And Linda called. She works now for Walgreens, and so could not infuse Jack, even though she has a 26-year-old son with hemophilia and lives close by. But, her brother lives only 10 minutes from where Jack lives and he is a doctor. Problem solved! Our hemophilia herd rallied around this man, and within one hour we had located a way for him to avoid the ER and get an infusion.

For Jack, this was the wake-up call of a lifetime. Talk about stuck in the mud. I can understand how those with mild hemophilia can go their childhood never learning to infuse. They bleed so infrequently that it’s not practical sometimes to teach them. But as these guys age, their past catches up with them. Jack confided that he had some joint problems. We know now that even one bad joint bleed can set us on the road to arthritis.

So Jack knows he has to make a plan: register with the Floria hemophilia organization; visit an HTC, not just any hematologist; learn how to self infuse; get newsletters and books and read about hemophilia; meet some of his community brothers with hemophilia; and find a way to solve his insurance problem. You just cannot be without insurance if you have hemophilia! Even if it means going on Medicaid. Fortunately, there are many programs to get help in our community.

Jack was lucky, but I wonder how many other young men in our country have hemophilia and who live on the fringe, not aware of the community around them. They are like baby elephants with no herd to care for them. I am proud of Natalie, Linda and Kim, who went out of their way to help, and thank them for showing so much compassion and dedication. These traits are what make our community as special as it is. And sorry for referring to you ladies as elephants; you know what I mean.

Book I Just Read: The Cure, by Timothy Brantley

“The Cure” alludes to us as self-healing organisms: “You are the cause, you are the cure.” Nothing here is new… at all. Brantley (who is not an MD, though he has “Dr.” in front of his name, as in PhD, obviously trying to lure us into thinking he is an MD) has consolidated material from Harvey and Marilyn Diamond, who had a great book in the 70s called Fit for Life (four stars!), from Pamela Secure’s Three Day Fast, from John Robbins’s Diet for a New America… ground-breaking books published years ago which already laid out his principles, which are not new or earth-shattering. Drink water. Avoid table salt. Consume mostly fresh foods. Stop eating pastries, bread, sugar, colas. Chew your food more. Drink water. See what I mean? Nothing original. He himself has a compelling story: his father was an exterminator, and his mother eventually died of cancer, leaving him determined to unlock mysteries of cancer and illness as they relate to our diet. Problem is, people have already done that. He’s endorsed by several Hollywood stars (hmmm, no doctors? No nutritionists?) and appears on TV talk shows. Why not? He’s handsome and hip. But original? Naw. Strangest of all, after spending 90% of the book telling us to eat food as “Creation” made them, he promotes his herbal pills and concoctions by asking you many, many times to visit his website, which is at least 2 years out of date. If you have never read any book on diet and illness, you might like this and I think what he says is true. But I’ve read better. One star.

Stepping Over the Line

I read a great article today in the Columbia Tribune about step therapy… an insurance cost cutting measure, meant for the common good, but which has caused great concern in the hemophilia community. So far it has been attempted in the hemophilia community, but our advocates have fought and won protection against this. Read on… (edited)

Who’s the Doctor?
Insurers reject prescriptions to save money.
Published Sunday, January 11, 2009

Two years ago, Tracey Joyce of Webb City showed up at her local pharmacy expecting to get a refill for an anti-arthritis medication. She’d been taking the drug, Celebrex, since she had a shoulder surgery two years earlier and says it made her pain manageable.

But at the pharmacy counter she got a surprise.

“They told me, ‘Express Scripts has said they won’t pay to fill this,’ ” Joyce recalled. “‘They want you to do a step program.’ “

Joyce was referring to Express Scripts, the St. Louis-based pharmacy benefit management (PBM) company that administers prescription benefits for more than 50 million people nationwide. The company is contracted through Joyce’s insurer, Mercy Health Plans, and is paid to lower drug costs.

Sometimes this means asking patients to try generic drugs instead of name-brand drugs and at other times to try alternative medications or alternative treatments for their illnesses. This is commonly called “step therapy” or the “fail first” approach.

Joyce, though, didn’t want to fail first. She had been prescribed Celebrex in part because she has a history of ulcers, and the medication doesn’t damage the stomach lining. She and her doctor agreed she needed that drug and that drug only. But the step therapy required that she try three months of Advil, Aleve or Naproxen before Express Scripts would authorize payment for Celebrex.

Joyce said she had no choice but to try the step therapy because of costs. After three months of taking the alternatives, she was “allowed” by the PBM to go back on Celebrex. But something still didn’t feel right, she said.

Soon afterward, chest pains sent her to the emergency room, and doctors discovered she had six ulcers. Her primary-care physician, Andrew Roudebush, blamed the step therapy drugs, which, he said, can actually harm the stomach lining and make ulcers worse.

Above, Tracey Joyce battled with her health insurance company after a pharmacy benefit management company changed her medication to a lower-cost drug. Below, Andrew Roudebush, Joyce’s personal care physician, believes the changes worsened Joyce’s ulcers.

“My view is they’re practicing medicine without a license,” Roudebush said of the way the PBM intervened in the doctor-patient relationship.

Joyce said the decision ultimately cost her insurer more money than the drug ever would have. “I ended up with a $14,000 hospital overnight stay because they wouldn’t pay for the $100 bottle of pills.”

“It’s been an issue with a lot of people, and it seems to be getting progressively worse,” Nold said. “In general terms, this is what happens all day long.” Unlike most other patients, Joyce was in a position to do something about this problem. She works as the legislative assistant to state Rep. Bryan Stevenson, R-Webb City, and she asked her boss to change the law.

“My thing is right is right, and wrong is wrong,” Joyce said. “Doctors went to medical school, doctors did all the training and they have to keep a license. Let’s let them practice medicine instead of the clerks that are on the other end of the 1-800 line.”

Only a doctor can write a prescription, but doctors say insurance companies are second-guessing their decisions.


Step therapy grew in popularity earlier this decade as costs of pharmaceuticals skyrocketed. Express Scripts, for example, said the use of step therapy among its members grew from 4.5 million at the end of 2002 to 11.7 million in 2004.

The company touts the savings of the programs. A 2004 study by the American Journal of Managed Care indicated implementing step therapy programs for three main drug classes–drugs used to treat ulcers, depression and arthritis, respectively — reduced total costs for those classes by 38 percent during a two-year period.

Express Scripts labels drugs as “front-line” and “back-up” drugs for certain chronic conditions like high blood pressure and arthritis. Front-line drugs are typically cheaper generics, and back-up drugs are the name-brand versions.

“Step therapy programs are only developed in therapy classes where the generic and brand-name options are considered highly interchangeable,” Palumbo said. This is “based on clinical practice guidelines, primary literature and/or feedback from practicing physicians.”

Palumbo added that in “rare instances” where it’s necessary, the company has an exceptions process that allows doctors to override the PBM step therapy. But for physicians like Roudebush, the exceptions process is frustrating. Roudebush said he has had little luck with the override option and has found the back and forth wrangling with PBM clerks and nurses takes several days and usually ends with a “no” from the PBM.

Step therapy, he said, is often not a choice between two equal options. “They’re just making a financial decision, which I respect, but I don’t feel like I frivolously prescribe medicines that are the most expensive,” he said. “I think I use what’s best for the patient.”

Nold agreed. He said he regularly has to give his patients repeated, medically unnecessary endoscopic procedures simply to “prove” to insurance companies they need stronger medication. “They’ll end up coming back to get another scope just to prove” the drugs are “not adequate, and that’s sad,” he said.


During the last legislative session, Joyce was among several people pushing for PBM reform. The language to regulate the practice was eventually written into legislation, House Bill 1332.

Among other things, the bill would have given physicians the power to override step therapy decisions when treatment is expected to be ineffective. It also caps the time limit on step therapies at 14 days.

The House and Senate overwhelmingly passed similar, but not identical, versions of the bill. The session ended before the differences in the two versions could be reconciled. Joyce said Stevenson was at work on a new PBM oversight bill last week and that he planned to submit it soon.

Keele testified before a state Senate committee in support of HB 1332 last year.

Joyce said she will continue to strongly advocate for the legislation until it passes. It has become her cause, and she has been getting thumbs-up from people everywhere she goes.

“You’ve got people with business degrees telling people with medical degrees what to prescribe,” he said. “Did you go to business school to know which stomach medicine is best for me? I don’t think so.”

Reach T.J. Greaney at (573) 815-1719 or

Read carefully. Step therapy assumes that two drugs are interchangeable, so the question begged is this: are factor VIII drugs interchangeable? Can an insurance company switch your factor brand to another to save on expenses? That’s what some have tried to do, and most like will try to do. Stay tuned… and watch your step.

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

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!

In Praise of PSI

So last Wednesday night I walk into an Irish pub in Haverhill, a city about 20 minutes from my home. “The Peddler’s Daughter” is a wonderful little piece of the old country, in a cozy cellar location, with great food and a lovely Irish atmosphere, run by real Irish people. It’s one of our favorite places to eat. Great fish and chips; homemade ketchup.

About an hour later a young man walks into the pub and heads to the bar, and I feel like i know him. Sure enough, I do. It’s Walter, the young man who tiled my cellar last fall. At that time was part of a work crew from a local contractor, and he surprised me, as he was putting in the tile, when he said, “Does your son have hemophilia, too?” Apparently, he figured it out from the photos I have of Paul Newman and me at Camp Hole in the Wall (can’t miss that autographed picture, front and center as you walk in). We chatted and I learned he has mild hemophilia and lives in Haverhill. I have people from all over the world on my mailing list but not someone who lives 15 minutes away? Well, he and his family were not active in the community. I added him to my mailing list, gave him a free copy of my book and sent him on his way, leaving me to enjoy my new floor.

Two months later Walt stopped by my office with news. “I quit my job,” he said hesitantly. I treated him with the same care and concern I would my own child. “What are you, crazy?” I said. No job, no insurance; no insurance, no factor. What was he thinking? He didn’t like his boss. And with no college education, it would be tough to find another job. I lectured him a bit and then armed him with a mission: call your hematologist; call your home care company and very quickly, call PSI. Tom had never heard of PSI. When I explained to him that it could save his life, I guess he listened.

Off he went and months went by. I didn’t hear from him. And then, in all the bars in all the world, he walks into mine.

“Hi, Walt?” I asked, eyeing him in the dim light.

“Mrs. Kelley!” he exclaimed, recognizing me. Then, “I got a job!”

Poor kid: now he’s treating me like his mother!

I was thrilled for him. Of course, there is a waiting period for those with pre-existing conditions… not to worry. Walt amazed me when he said, “I called PSI like you said and they are covering me until my new insurance kicks in.”

Wow. Here was a kid who never attended a hemophilia meeting, went to camp once, doesn’t know anyone else with hemophilia, quit his job not realizing the dire consequences, couldn’t name his factor brand… and he did it. He got himself back on track. I was so happy! He was too.

Kudos to PSI: you don’t know Walt but you have changed his life. Thank you, Dana, thank you everyone at PSI!

(To learn more about PSI and how they help people with chronic disorders who lose their insurance, go to

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