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Wheels for the World 2020: A Win-Win

Laurie Kelley September 14, 2020

The pandemic has put a halt to in-person fundraisers for nonprofits like Save One Life, and yet we are extremely dependent on these events for overhead revenue. We receive program funding from grants, but for regular overhead, like salaries, rent, utilities and services, we often have to raise our own funds. Save One Life is starting to be known for its “adventure” fundraising, like cycling, mountain climbing and even hiking!

**Saturday’s riders: social distancing!**

This past weekend we honored Barry Haarde, a man with hemophilia and HIV who cycled across the US six times in as many years, raising over $250,000 for Save One Life. Barry passed away in February 2018, a huge loss personally and professionally to us. He was one of a kind, a deep, sensitive soul with a heart as big as the state he lived in, Texas. We decided to first do a bike ride in 2018 to remember him, and now we continue that tradition each year to honor him and his favorite cause, helping children with hemophilia in developing countries. Save One Life was one of his top favorite charities.

Our “Wheels for the World” went virtual this year, and we opted for an in-person cycling ride (with proper masks and social distancing) starting from historic Ipswich, Massachusetts. We had eleven riders participate on Saturday to do 23 miles, including an 11-year-old! Pretty gutsy!

With lots of water and donuts, we headed out into the crisp, New England air. The course took us through the lush green farmlands and horse stables that populate this area, and past solid, magnificent colonial-style homes, some dating back to the 1600s! Along the route a small grey vole darted madly across the road, just missing my tires; a white-tail deer stood like a statue in a golden field, ears alert like radar shields as it watched us pass; and sadly, I saw a very flat chipmunk, a victim of the waning sun and probably extra body fat as he readied for hibernation.

We all returned together, and after congratulations (no hugs!), we went to True North Ale House for a complimentary beer, or in our case, blackberry Izzy drinks. I especially enjoyed chatting with Oliver, a tall young man who just started working at uniQure three weeks ago, and who is responsible for making the viral vectors into which the human gene for factor VIII is placed. He is working on the cure for hemophilia and we surely hope he is successful!

On Sunday, we reconvened on another bright, warm day, with a smaller group. I was also supposed to ride this one, 62 miles, but had woken up Friday with a back spasm. I worked it out, and was able to ride the 23 miles (which was actually 27 for us, as we got a little lost) but today, it came back with a vengeance. No way could I do 62 miles when it felt like there was a little ball with spikes sitting in my lower right back. I showed up in my gear, ever hopeful, but bailed at the last minute, my back sending warnings. I watched the six riders shove off. Jodi, Karen and I were waiting for maybe two more, and after 20 minutes a car pulled up. Was it Dan Leonard of uniQure, or Scott?

I watched as a handsome man got out of the car and approached. He said, “You don’t recognize your own brother?” My brother! All the way from the Springfield area, two hours away. I hadn’t seen him in over a year and hadn’t known he had registered! Imagine if I had not had the back spasm, and took off on the 62-mile ride. I might never have seen him that day. Jim and I hugged and chatted, and then Dan Leonard arrived. So now they both had someone to ride with. They opted for the 23 mile one that we did yesterday. And off they went!

The day was successful and I know we raised a lot of money from the wonderful sponsors, and from the dedicated riders. I didn’t even know some of the riders—two from Worcester, Massachusetts—who were college student and friends of someone in our community. They loved the ride and pledged to participate next year.

In fact, everyone had a great time. I heard from quite a few people that this year had been so strange, that they had not had a chance to ride outside at all. Injuries, work, childcare issues, COVID… this fundraising ride gave them the boost to dust off the bike (my brother’s literally had cobwebs on it!) and get out in the glorious autumn sunshine to enjoy the beauty of this state. We made new friends, reconnected with old friends, and helped Save One Life.

As Doug and I drove into downtown Ipswich, to get a bite to eat on Saturday, a tall, lean cyclist pulled right in front of us, not even staying to the side of the road, but directly in front (legally fine). I was struck by his position and body type. Incredibly, this rider had Barry’s long torso, and thin, powerful legs. The way he held his handlebars and leaned over… I had ridden enough with Barry to know his stance anywhere. It was as if he materialized to ride with us once more time…. and then he was gone. But I managed to snap a photo.

His spirit is always with us, challenging us to be better versions of ourselves, and leading us into a future where every child with hemophilia will have access to medicine. That’s why we ride, and that’s what we work for at Save One Life. RIP Barry. And thanks to everyone who sponsored, rode and supported Wheels for the World 2020!

Copay Help? Thanks a Lot, Payers

Laurie Kelley September 7, 2020

Just when we need financial assistance the most, as we require to use one of the highest priced drugs on earth, insurance companies (payers) are negating the benefits of the copay-assistance programs from factor manufacturers. Well, it negates the benefit to us–it does benefit the payers.

Here’s a Q&A about co-pay assistance featuring Miriam Goldstein, Director for Policy, Hemophilia Federation of America.

1. “I’ve seen on Facebook that some insurance companies won’t honor manufacturer copay assistance programs. What’s going on?”

Drug manufacturer copay assistance programs help many in the bleeding disorder community. People who live with bleeding disorders rely on medications—clotting factor or other treatments—to control bleeding and preserve their health. These drugs are essential, but expensive, and are needed on an ongoing basis. As a result, many people with bleeding disorders face the prospect of hitting their health insurance out-of-pocket maximums each year, and can reach that limit all at once with one order of factor. Yearly out-of-pocket maximums include copays, coinsurance, and deductibles, with amounts varying from plan to plan. While the Affordable Care Act (ACA) places an annual cap on cost-sharing amounts, that ceiling—in 2020, between $6,900 and $8,150 for an individual, or between $13,800 and $16,300 for a family, depending on plan type—is high, and the prospect of hitting it every year is daunting, to say the least.

Manufacturer copay assistance programs protect patients from this financial burden, and sustain their access to essential medications, by covering patients’ drug-related copay, coinsurance, and deductible expenses. Many programs will cover $12,000 and up in cost-sharing expenses per year. Eligible patients typically must have third-party commercial insurance to participate in these programs.

Unfortunately, in recent years a growing number of health insurers and pharmacy benefit managers (PBMs) have begun claiming that copay assistance programs incentivize patients to demand pricey brand name drugs instead of using more cost-effective generics. Citing the need to counter these incentives and contain costs, some health plans have been taking steps to limit the value of manufacturer copay assistance to patients—while maximizing the amount that the health plans themselves collect from those programs—through the use of “accumulator adjusters.”

2. “I’ve heard that term, but don’t know what it means. What are ‘accumulator adjusters,’ and how do they affect me?”

Accumulator adjuster programs are used by health insurance plans to limit the value of manufacturer copay assistance programs. When an accumulator is in place, the health plan accepts the manufacturer copay assistance—which is supposed to pay for the patient’s out-of-pocket drug costs—but then doesn’t credit that amount toward the patient’s deductible or out-of-pocket maximum. The health plan draws down the full value of the copay assistance as prescriptions are filled; but then it “adjusts” or resets what is credited to the patient’s cost-sharing obligations back to zero. As a result, a person with a bleeding disorder will still have to personally pay deductibles, copays, and other out-of-pocket expenses, up to the yearly out-of-pocket maximum. At the same time, the health plan appropriates the full amount of the copay assistance—assistance that was supposed to help the patient!

Accumulators leave bleeding disorder patients in a tough financial bind. If this happens to you, please check out the options listed in Hemophilia Federation of America’s (HFA) online Resource Library.¹ You may be able to get financial help with your copays from a third-party, charitable nonprofit patient assistance fund.² Health plans sometimes will not apply accumulators to copay assistance provided by charitable organizations,and will still credit the charitable assistance to patient deductibles and out-of-pocket maximums.

HFA is working with other patient advocacy groups to educate health plans and PBMs about the dangers of accumulators. We urged federal regulators to ban the use of accumulators, where patients don’t have the option to choose a generic drug (as is the case with bleeding disorder patients). Unfortunately, the US Department of Health and Human Services (HHS) rejected this request. In May 2020, HHS finalized a rule3 that allows health insurers to continue using accumulator adjusters for the coming plan year.

HFA and allied groups will continue to advocate for federal and state policies that prioritize patient access to their prescription medications. In the meantime, we need to hear from you! If you learn that your copay assistance will no longer be credited toward your deductible or out-of-pocket maximum, please share your story with HFA’s Project CALLS.⁴ Collecting data on the impact of accumulators allows us to make a case for change when we ask lawmakers to take action to protect patients from these harmful health plan tactics.

Miriam Goldstein is director for policy at Hemophilia Federation of America, where her work includes monitoring and analyzing federal legislation and regulations impacting patient access to care; insurance, Medicaid, and Medicare issues; and blood and product safety. Miriam lives in Arlington, Virginia, and is the mother of two adult sons with hemophilia.

1. hemophiliafed.org/resource-library/additional-resources/navigating-patient-assistance-programs

2. Patient assistance funds that offer financial assistance to eligible bleeding disorder patients include the Assistance Fund, Patient Access Network (PAN) Foundation, and Patient Services, Inc. (PSI).

3. US Dep’t of Health and Human Services, Notice of Benefit and Payment Parameters for 2021, 85 Fed. Reg. 29164 (May 14, 2020).

4. projectcalls.org

Know Your Factor Products

Laurie Kelley August 30, 2020

A recent tweet by a doctor I follow had me realizing we could all do with reviewing all hemophilia products… or at least, just knowing which product we use, and why. Currently, there are so many products and options. Do you know what your product’s name is, what “class” is it, and why you are using it?

First, know that all products approved by the US FDA for treatment are considered safe (from infectious disease) and efficacious (does its job). We always start by talking about products by first saying that there are two basic classes for standard factor: plasma-derived and recombinant.

The major difference between the two types is the origin of the factor, called the source material. Plasma-derived factor originates from human blood plasma. Recombinant factor originates from genetically engineered mammalian cells containing the human gene for factor. Most people with hemophilia in the US are now using recombinant products.

Recombinants can even be further classified according to their manufacturing process: first generation, second generation, and third generation. Several recombinant factor products also have a prolonged half-life, allowing you to infuse less frequently. The first of these new products was introduced in 2014.

First generation products contain human/animal proteins in culture medium; albumin added in final formulation

Second generation products contain human/animal proteins in culture medium; sugar added in final formulation, no albumin added

Third generation products contain no human/animal proteins added at any stage; formulated with sugar, not albumin

There is mention of a fourth generation–those products that are made not from mammalian cells but from human cells, so there is no risk of animal cells in the formulation, which may limit the formation of inhibitors.

And of course, there are nonfactor products, like Hemlibra.

So, know your treatment and why you use it! Ask:

Who selected your therapy—you, your doctor, or both? Could you explain to another patient or parent why you chose that product over the others Don’t leave the decision making to your doctor only, and absolutely not to your payer only. Remember that your payer may prefer to reimburse the least expensive product and not necessarily the best for you. Decide with your physician which product is best for your child. One day you may need to justify your product choice to your payer. It will help if you can learn to speak the language of therapies!

And use our chart, which we update frequently on our homepage. You can download this by clicking on the image on our homepage.

Pondering Pain

Laurie Kelley August 23, 2020

All last week we at LA Kelley Communications were putting the final touches on the November issue of PEN, which is all about pain management. Ironically, in the middle of editing, I was off to see an orthopedic podiatrist for an extremely painful foot. For months I’ve been getting out of bed, unable to put pressure on my right foot, until I hobbled about enough to finally ease the arch down a bit. For a couple of months I thought it was just “old age,” and overuse from trying to run 6 miles in the morning, when I should be running about 2. The ortho said no; it was a too-tight tendon. Plus, my right leg is a bit shorter than my left! So he will make me orthotics. Take some Aleve®, wear sneakers and stop going about barefoot, and the orthotics will be in soon.

Then this morning…. stabbing back pain. It felt like someone knifed me; it felt like an animal was biting into my back. I had wanted to go on a 70 mile bike ride (to prepare for Save One Life’s Wheels for the World ride in 2 weeks), but it was a no go. A heating pad, more Aleve, and a day on bed rest.

It was a gorgeous day, too. Sunny, clear and the beach coast beckoned. From my bedroom I can see the gorgeous trees swaying, hear birds chirping. It was frustrating to be stuck inside, inactive.

Then I realized this is what our community members go through a lot. Even those on prophy; even those with long-acting factor. The backache could have been a bleed, causing constant aching, then sharp pain. Ice, rest and OTC pain-killers. No activities, even on beautiful days.

Pain can teach us a lot. I reviewed my week; why was I getting a backache? I checked my calendar: I had only done yoga once in the past two weeks! I know from experience I need to do yoga at least four times a week to stave off back spasms. I did a 32-mile bike ride on Friday, feeling great, and took yesterday off, and sat about all day, working on my computer and reading. In fact, I sat for four straight hours. Big mistake. Lastly, I didn’t drink any water. Not a drop. I had tea and Diet Pepsis, both diuretics. Lessons learned… again.

I don’t regret the pain, because it reinforces what I already know I must do to keep it away. It teaches and reminds. It also makes me more compassionate towards others who suffer, like our community with bleeding disorders. Even if just for a day, I get a small dose of what it’s like to be laid up, watching the world go by on a sunny day. It makes you appreciate the good days all the more, and the medicine we have to alleviate bleeds and pain.

Black Lives Matter in Hemophilia History, Too

Laurie Kelley August 16, 2020

This is an essay from our esteemed writer Richard Atwood, of North Carolina, who writes quarterly for PEN, reviewing books and movies. Today he offered this research on the history of a very current topic. He writes:

We’re reminded daily, rightly so, of persistent systemic racism in the US in many aspects of our lives.

Historically, the obvious example in the bleeding disorders community was the segregation of blood, as practiced by the American Red Cross and the U.S. military, at least until after World War II, when, quietly and unannounced, blood became integrated.

Another example has been the underreporting of bleeding disorders in minority populations. There are myriad of reasons for this. Note, a few of the sources have not been confirmed for reporting hemophilia in African Americans. For now, just acknowledge that underreporting of bleeding disorders has occurred in minority populations.

Evidence-Based Prevalence of Hemophilia in Black Americans Before 1950

Richard J. Atwood

The underreporting of hemophilia in Black Americans has a long history that mimics their social history in terms of discrimination and suppression. And the few cases that have been reported in the medical literature tended to be either ignored or dismissed. Two texts from the 1940s might have altered the narrative if they, too, had not been overlooked. Julian Herman Lewis authored The Biology of the Negro (1942) at the beginning of the decade, and then, at the end of the decade, R. Ruggles Gates wrote Pedigrees of Negro Families (1949).

The Biology of the Negro (1942)

Julian Herman Lewis, PhD, MD, was an Associate Professor of Pathology at the University of Chicago. He investigated comparative racial pathology of Black Americans, loosely defined as people having a decided African origin found in the United States. Hemophilia is listed in the subject index, and the text contains a two-page summary of hemophilia (pp. 250-1). The author states: “It appears that hemophilia is an extremely rare disease in Negroes.” (p. 250). He mentions that Bulloch and Fildes reported, in 1911, that out of over 900 cases, only three papers documented four cases of the disease in Negroes, all from America. Lewis cites Hadlock (1874), Steiner (1900), and Buck (1900), all from Bulloch and Fildes (1911), and adds Crandall (1936), Pachman (1937), and Campbell (1939). His assessment of hemophilia’s rarity seems accurate, but the steady reporting of new cases in Black Americans needs to be acknowledged.

Pedigrees of Negro Families (1949)

R. Ruggles Gates, FRS, was an emeritus professor of Botany at the University of London and a research fellow in Biology at Harvard University. He collected over 200 pedigrees of black families from around the world, but mainly from the United States, Canada, West Indies, and British Guiana. The index lists hemophilia, parahemophilia, and pseudohemophilia. The text includes 245 figures to display the pedigrees. The section on hemophilia encompasses 15 pages (pp. 121-35) and 12 figures (Fig. 131-42). The author states: “This hereditary disease is believed to be less frequent in Negroes than Whites, but there are no definite statistics on the subject and it is possible that most cases of hemophilia in colored families have been derived from a white source.” (p. 122). Gates summarizes the literature, omitting Bulloch and Fildes (1911), but citing Hadlock in Cincinnati (1874), Steiner in Baltimore (1899), Buck in Portland, Oregon (1900), Taylor in South Carolina (1923), Bailey and McAlpin (1935), Crandall in Philadelphia (1936), Pachman in Durham, North Carolina (1937), and Campbell in Washington, D.C. (1939). Gates adds six black pedigrees having hemophilia, some from North Carolina and Baltimore. One pedigree (Fig. 132) has three pairs of identical male twins with hemophilia, one pair of dizygotic female twins with one being a carrier, plus one pair of normal male twins, a situation that surely should be noted for its exceptionalism. The author notes several issues that complicate any analysis. Many of the pedigrees involved interracial couples, thus mixing the genes from black, white, native American, and Creole families. A misconception was that any hemophilia would arise from white blood, and a pure black family was difficult to identify. Also, hemophilia itself was being differentiated into various types, such as true hemophilia, parahemophilia, pseudohemophilia, and purpura.

Subsequently, additional reports of hemophilia in African Americans, and also of hemophilia on the African continent, appeared in the medical literature. Yet the misconception has continued that hemophilia in Black Americans is uncommon. Perhaps if both texts by Lewis and Gates had been acknowledged, starting from their publication dates, this misconception would not have persisted. Any recognition would be found in medical journal articles.

A more comprehensive account of the medical literature on hemophilia in the black community had to wait until Stephen Pemberton wrote The Bleeding Disease: Hemophilia and Unintended Consequences of Medical Progress in 2011. In a footnote on page 312, Pemberton cites the texts of Bulloch and Fildes (1911) and Lewis (1942), plus the journal articles for Hadlock (1874), Koch (1890), Steiner (1900), Buck (1900), Taylor (1923), Crandall (1936), Pachman (1937), Birch (1937), Campbell (1939), while adding Rosenbloom (1923), Muir (1928), Kugelmass (1932), Rypins (1934), Prip Buus (1935), and Nesbitt and Richmond (1949). Clearly, Pemberton conducted a more thorough search of the literature than did other reviewers before 1950.

References:

Bailey FR, McAlpin KR. Familial purpura. American Journal of Medical Sciences, 1935; 190:263-8.

Birch CL. Hemophilia: Genetic and clinical aspects. Illinois Medical and Dental Monographs, 1937; 1:1-151.

Buck L. Haemophilia in the Negro. Medical Record, 1900; 58:149.

Buerk MS, Tucker HA. Penicillin in the treatment of early syphilis complicating hemophilia: Report of a case treated with 48 million units of oral penicillin G. Bulletin of Johns Hopkins Hospital, 1949; 84:24-8.

Bulloch W, Fildes P. Hemophilia. In Pearson K, editor, Treasury of Human Inheritance. London: Cambridge University Press, 1911.

Campbell EP. Hemophilia in the Negro: Report of a case. Medical Annals of the District of Columbia, 1939; 8:294-5.

Crandall NF. Hemophilia in the Negro. American Journal of the Medical Sciences, 1936; 192:745-51.

Forbes CD, MacKay N, Khan AA. Christmas disease and hemophilia in Kenya. Transactions of the Royal Society of Tropical Medicine and Hygiene, 1966; 60:777-

Forbes CD. The early history of hemophilia. In: Forbes CD, Aledort LM, Madhok R editors. Hemophilia. London: Chapman & Hall Medical, 1997.

Gates RR. Pedigrees of Negro Families. Philadelphia: The Blakiston Company, 1949.

Hadlock. Hemorrhagic diathesis, ‘Clinic’ of Cincinnati. Transactions of the Academy of Medicine, 1874; 7:241.

Koch WVM. Haemophilia occurring in malaria. British Medical Journal, 1890; 1:1301-2.

Lewis JH. The Biology of the Negro. Chicago: The University of Chicago Press, 1942.

Kugelmass IN. The diagnosis and management of hemophilia in childhood. New York State Journal of Medicine, 1932; 32:660.

Muir J. Heredity in hemophilia in South Africa. Journal of Medical Association of South Africa, 1928; 2:599.

Nesbitt RD, Richmond JB. Hemophilia in the Negro. Journal of Pediatrics, 1949; 34:315-21.

Pachman DJ. Hemophilia in Negroes. Journal of Pediatrics, 1937; 10:809-16.

Pemberton S. The Bleeding Disease: Hemophilia and the Unintended Consequences of Medical Progress. Baltimore: The Johns Hopkins University Press, 2011.

Prip Buus CE. Articular changes in hemophilia. Acta Radiol., 1934; 16; 503.

Rosenbloom J. Warning against the use of arsphenamin in the treatment of syphilis in a hemophilic. Journal of Laboratory and Clinical Medicine, 1923; 9:57.

Rypins EL. Roentgen ray as aid in diagnosis of hemophilia. American Journal of Roentgenology & Radiation Therapy, 1934; 31:597-602.

Sanford HN et al. Action of intravenous injections of histamine on the blood of hemophilic children. American Journal of Diseases of Children, 1948; 76:609-15.

Steiner WR. Haemophilia in the Negro. Johns Hopkins Hospital Bulletin, 1900; 11:44-7.

Taylor JH. A case of haemophilia in a Negro. Journal of the South Carolina Medical Association, 1923; 19:665-8.

Younge WA, Perry JC. Hemophilia in the Negro. Journal of the National Medical Association, 1950; 42:299-302.