Laurie Kelley

Snakes and Hemophilia

Laurie Kelley October 18, 2020

Just in time for Halloween, our amazing archivist, Richard Atwood of North Carolina, has found another reference to hemophilia in an unlikely place—a novel about snakes. The Snake, by John Godey, published in 1978, is a novel about… a snake.

It’s called a black mamba, though olive-slate in color, extending 11 feet, 2 inches. The black mamba is the largest and deadliest poisonous snake in Africa. The snake arrives in New York City in a plywood box with a burlap lining and air holes.

A Greek bought the snake in Elizabethville, then lost it in a poker game in Maputo. Matt Olssen, a seaman returning from Africa to the States, wins the snake and wants to sell it to a zoo. After docking in Brooklyn, Olssen takes his snake-in-a-box and begins drinking. In Central Park late that September night during a stifling heat wave, a thug named Ramon Torres mugs, then shoots Olssen dead. The box breaks open and the snake bites Torres twice in his thigh. Torres dies that night at East Side Hospital.

The next evening Roddy Bamberger leaves his date at the outdoor Delacorte Theatre. Walking past Belvedere Lake, Bamberger steps on the snake and is bitten three times in his left thigh. Bamberger dies at East Side Hospital. With two deaths to solve, and the city in a panic, Captain Thomas Eastman of the NYPD is put in charge of finding the unidentified snake in Central Park – an area covering 840 acres, of which 150 is water. The Reverend Sanctus Milanese of the Church of Purification proclaims that the snake is Satan, or Satan’s messenger.

Captain Eastman recruits Mark Converse, a young herpetologist, to locate the snake. Jane Redpath, a graduate student, takes photographs in the menagerie at night. She is accompanied by her boyfriend, Jeff, a former football player at Columbia. Jeff is bitten on his forearm, and dies at East Side Hospital. The snake then bites Alvis Parkins, a 15-year-old boy, twice in the neck, but Alvis is run over by a car before he can reach a hospital. Fortuitously, Redpath took a photo of the snake, that can now be identified for the correct antivenin.

Later, the snake chases a rat into an apartment and bites a family from Trinidad: Webster McPeak, wife Emily, son Webster Junior, and daughter Charlene. Even with black mamba antivenin, Emily and Charlene die at West Side Hospital. On the twelfth day of the heat wave, Converse spots the snake sunning on a rock in the East Meadow, but a police helicopter scares the snake away. A follower of Reverend Milanese named Graham Black is also searching for the snake. Black is bitten on his right leg and receives the antivenin. The police fire Converse. Reverend Milanese organizes Operation Pillar of Fire to burn the snake out of Central Park. The snake escapes and bites a couple three times in the back of a taxi. Another Milanese disciple is bitten by the snake after it is beheaded.

But it’s not over yet… the snake leaves 13 eggs in her burrow in Central Park!

It’s interesting to note that on page 56 in the book, a television newscast shows the milking of a Russell’s viper’s venom at a Brazilian snake farm. The film is narrated: “Not all snake poison is malign. The venom being taken from this Russell’s viper will be used as a coagulant for persons suffering from hemophilia.”

Richard comments, “The simplest of fears can sometimes be the most terrifying. The possibility of a single snake terrorizing an entire city seems realistic. The snake bites 11 individuals, killing at least six, plus she has the opportunity to bite numerous other New Yorkers during her short stay in Central Park. The revelation that Russell’s viper venom has been used to treat hemophilia as a topical hemostyptic is correct. The novel, however, does not reveal that the product has been commercially available from Burroughs Wellcome and Company as Stypven, and from Boots as Rusven. A Russell’s viper venom dilution of 1:10,000 clots hemophilic blood in 17 seconds. The commercial product uses a 1:100,000 solution, while the venom is still active at 1:1,000,000,000.”

I’ve heard of transgenic animals that are milked to collect factor, but never milking a snake for its venom to clot blood! Since Halloween is cancelled most everywhere due to the pandemic, read a good scary book this Halloween!

Is It Safe to Donate Blood in a Pandemic?

Laurie Kelley October 11, 2020

In the U.S., the first full week of September is designated National Blood Donation Week, to promote blood donations and the need for blood, for emergences and for therapeutic drugs. I normally post something about it, but with Covid-19 taking center stage, overlooked it this year. I just read The Source magazine, from the Protein and Plasma Therapeutics Association (PPTA), and found the welcome, called “Outlook,” by new president Amy Efantis, very compelling. Please have a read of this excerpt, and go to “the source” itself, to download your own copy here. Remember that some of our community members still use plasma-derived products, as do many in developing countries. Is it safe to donate blood in a pandemic?

Outlook by Amy Efantis

“… in those early days of this crisis, my committed and experienced colleagues recognized even before the scourge of Coronavirus SARS-CoV-2 was officially declared a pandemic that patients might have concerns about plasma-derived therapies (PDTs) and the safety of those therapies due to COVID-19. Our member companies’ leading pathogen safety experts came together to assure patients of the safety of PDTs. They outlined the extensive safety measures that industry follows for donor recruitment and validated pathogen removal/inactivation steps during the manufacturing process, providing assurance to patients and providers about the safety of PDTs. Our statement detailing these protocols was published on our website in several languages and has been frequently cited.

“PPTA sprang into action to make sure that plasma centers, center employees, and donors were deemed essential to perform their daily activities by authorities in the U.S. and in Europe. After appeals from PPTA, the European Centre for Disease Prevention and Control defined plasma as an essential substance of human origin and encouraged plasma centers to continue collection to ensure the continuity of production of plasma-derived therapies. PPTA also engaged in efforts to make sure that PDTs, and the essential goods needed in the collection of plasma as well as manufacturing of therapies, were considered essential to ensure the movement of goods between and among countries during the pandemic. This included appeals to authorities to make sure centers received priority for personal protective equipment and medical supplies.

“In the U.S., our efforts resulted in the President’s Coronavirus Guidelines for America noting the special responsibility of the pharmaceutical supply chain, including the important role of plasma and plasma donors. In fact, donors and staff were explicitly identified in every iteration of the federal government’s advisory list of essential critical infrastructure. But even as officials at the U.S. Department of Homeland Security and the European Commission issued guidance that deemed plasma centers, staff, and donors as essential, PPTA was called upon to engage directly with local, state, and national level authorities who were not aware of or did not understand the guidance, to ensure that centers remained operational. To assist in this effort, PPTA developed a repository of documented exemptions that plasma centers could have on hand if questioned by authorities during periods of restricted movement.

“We recognized early on that plasma donors, who are essential for addressing the clinical needs of patients for PDTs, might have concerns about their safety given anxiety surrounding the contagious nature of COVID-19. PPTA facilitated the development of a preparedness checklist for centers in the early days of the pandemic. PPTA member companies took immediate actions to accommodate the safety of donors in their centers with new protocols for increased screening, the use of personal protective equipment, social distancing, and the availability of disinfectants.

“Because of government-imposed restrictions in movement, our typical inspection paradigm was disrupted. In keeping with our historical commitment to standards, PPTA submitted a proposal to the European Medicines Agency (EMA) requesting remote good manufacturing practice (GMP) inspections and parallel inventory holds, which were agreed to by EMA’s GMP/Gross Domestic Product Good Distribution Practice Inspection Working Group. We adapted inspections in our International Quality Plasma Program to virtual models, and we advised auditors to match the U.S. Food and Drug Administration’s relaxed inventory hold period from 60 days to 45 days for purposes of PPTA’s Quality Standards of Excellence, Assurance & Leadership program. These efforts reflect our willingness to be nimble in a crisis with our abiding commitment to the highest possible standards.

“PPTA’s work with other stakeholders has meant increased attention to the urgency of plasma collection. Our partnership with the Platform for Plasma Protein Users and the European Plasma Alliance resulted in the European Commission launching a call for blood and plasma donations. It also prompted the EU Health Commissioner’s issuance of a statement recognizing for the first time the importance of plasma and its use in treating rare conditions, as well as its role in fighting COVID-19.

“In the U.S and in Europe, the patient community weighed in with local and national policymakers on issues supporting Source plasma donors and shined a bright light on the urgent need for plasma to treat rare disease patients. We are proud of our efforts during the COVID-19 crisis. PPTA has seen positive results in our responses during the pandemic. But this is not the end of the story.

“Concerns over plasma availability have now replaced initial anxiety around the safety of finished product. In March and April, plasma collections were down, understandably, compared to 2019 due to government-imposed restrictions on movement in the U.S. and Europe. But the expectation was that a recovery would occur as COVID-19 cases declined… in the U.S., as quarantine protocols were loosened, there was a resurgence of COVID-19 cases in several states, including Florida and Texas, states with a strong presence of plasma collection centers. Though a recovery continues, it is far less robust than expected. So now the real work begins.

“Just as it has been challenging to predict what will happen next in the pandemic, the same holds for the future of PDTs. But we do know this much today — donations NOW will mean better outcomes in the future. To meet patient clinical need, we encourage you to:

• Contact policymakers about the urgent need for plasma donations.

• Speak out about ending outdated regulations and call for the coexistence of the private sector with the public sector to collect more plasma.

• Use your own social media channels to spread the word about the ongoing need for plasma donations.

• Donate Plasma if you are an eligible, healthy adult — patients are counting on you!

“Let’s come together to show our commitment to patients.”

The Plasma Protein Therapeutics Association (PPTA) represents the private sector manufacturers of plasma-derived and recombinant analog therapies, collectively known as plasma protein therapies and the collectors of source plasma used for fractionation.

Pain Is Not the Problem…It’s the Solution

Laurie Kelley October 5, 2020

by Corey Pierce , November PEN 2020

The bleeding disorder community has a pain problem, but it’s not the problem of too much pain. The real problem is a misunderstanding of pain and its importance for our survival. Many people with bleeding disorders have come to devalue pain, because pain is often characterized as a mysterious and uncontrollable inevitability. A fallacious concept like “spontaneous bleeding” has sowed a seed in many minds that the actions we engage in are divorced from how our bodies respond. If the pain we experience is seen as inevitable, or with clouded causes, then we’re left with no other option than to manage the best we can, or trick ourselves into thinking the pain doesn’t exist.

If given the option to never feel pain again, many people would take this deal in a heartbeat. But only later would they truly understand the horrors of living without pain. What if you took this deal? Well, rare medical conditions like congenital insensitivity to pain (CIP) suggest that you shouldn’t be too attached to your fingers and toes. And you’d better hope to never get an infection or have heart failure, because you’ll be without the best warning sign nature ever created: pain. In short, your life would end much sooner. Living without pain is not a blessing—it’s a tragic, brutal curse.

If you want to understand pain, know that it results from a set of factors. Pain doesn’t arrive out of nowhere, and it’s rarely attributable to a single cause. For example, the mild ankle pain I am experiencing as I write this is not only the result of countless past ankle bleeds, but also the result of (1) standing for two hours straight while making an elaborate meal yesterday; (2) wearing “cool” shoes with no heel drop yesterday; (3) running three days ago even though I was sore; (4) eating inflammatory foods yesterday; (5) being dehydrated today; (6) slightly twisting my ankle on a tree nut while intently looking at a mailbox on a recent run; (7) not being able to ice myself after physical activity (my roommate has needed the ice more after his recent surgery); (8) not yet knowing my mileage limit while running (running is new for me since COVID-19); (9) switching to a new running shoe (is it helping or hurting?); (10) walking a mile on uneven sand recently. And so on…

If you look critically at what contributes to pain, you’ll see that it’s possible to improve your underlying condition by changing how you navigate through this world. By examining the obvious—and subtle—contributing factors to pain, you can create a template in your mind for what you should be doing and also avoiding. In my earlier example, there are complex interactions between my physical awareness, my activity level, and my shoes that I can monitor and refine over time, as I get used to my new activity—running. This introspective, reflective process is often at odds with the objectives of using pain medications.

Sadly, many people with bleeding disorders have been offered pain medications as the only solution to their pain. Too much reliance on pain meds may be nothing more than a deception that obscures the true problem(s). While effective over the short term in tamping down pain signals (the effects), pain meds also disguise the complex set of underlying factors (the causes). If we don’t address the true causes of pain, and instead focus solely on the effects with pain medication, the condition or behaviors at the root of pain will get worse, not better.

So, is pain your enemy, or is it your collaborator? If you want to live a long and healthy life, pain can’t be reduced to your tormentor Don’t let your pain be in vain by seeking remedy only through pain medications. You can’t arrive at the underlying causes of your pain when you are literally numb to them. Our pain is not what ruins life; it is what prolongs life, but only if we listen and learn from it.

Corey Pierce is a PhD candidate in public health at Oregon State University. He is currently completing his PhD dissertation, which explores how pain attitudes are connected to the experience of pain and use of pain medication. Corey has severe hemophilia A. He is active in the bleeding disorder community as an advocate and teacher of yoga, and he serves on the board of Pacific Northwest Bleeding Disorders.

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