September 2020

James’ Story: Why Preparedness and Support Matter

I’ve spent the last 24 years working in developing countries, helping families and nonprofits gain access to better healthcare for hemophilia. The story below touched my heart as I have been to the Philippines, and we now are partners in progress for better hemophilia care. Read James’s story below to see how his life changed with proper treatment!


Submitted by Takeda

James has hemophilia A. He’s found balance in the peaks and troughs of his life through profound relationships with both his wife and his care team.

This is James experience, but your experience may be different.

Learning, Living, and Growing

Born and raised in a rural village in the Philippines, James grew up in a careful household. Losing one brother to complications from hemophilia at a young age and living far away from any access to proper healthcare, his parents taught him to be cautious throughout his childhood. “My parents would limit my activities a lot. They loved me, but wanted to make sure I couldn’t hurt myself,” James recalls. When James did have a bleeding episode, the family would have to travel miles and miles to the nearest hospital to get him a blood transfusion.

James and his family moved to Los Angeles when he was 16 for one reason: his hemophilia. For the first time in his life, James learned more about hemophilia and the importance of maintaining factor levels. He began factor replacement therapy after his care team recommended a prophylaxis regimen.*

Learning and connecting with his hematologist, James realized his hemophilia was only a part of him, not who he was. “[He] helped me change my perception of hemophilia, my understanding of hemophilia. It gave me a little bit more confidence.”­

*James’ regimen was based on his needs as determined by him and his care team. This was his experience, but your experience may be different.

“Without her, the healing process would have been much slower.” —James, on Carla and why her support matters

Caring and Giving

Through surgeries, physical activities, and personal adventures, James found support in his wife Carla along with his care team. Having gone through surgery twice, he learned that preparedness is key. “Before both procedures there was a lot of discussion around how I’d be kept at a sufficient factor level. And for me, we decided to use factor prophylaxis during both surgeries, so they could continue to track my levels.” James could trust that his care team had his back in the operating room and Carla was there to help during recovery. “I wanted to be active again, to go out with her like we did before the surgeries. She was a big part of my recovery,” James remembers.

Post-surgery, Carla assumed the role of caregiver and ran with it. She partnered with James’ care team, including his physical therapist, planned his recovery, and learned how to help administer factor through a port.

Since recovering from his surgeries, James has been able to maintain an active lifestyle. Once he received the green light from his hematologist, James made a plan to stay fit. Through his preparedness and the support from his care team, James has been able to participate in bike events, including a charity ride with his friends.

“Crossing the finish line was such a great moment… tiring, but also rewarding. I was so proud of myself.” To James, crossing the finish line meant more than just finishing the ride. It was also a testament to the dedication of his care team and of Carla. Having their support, he learned he could trust his body again.

Today, James remains active and continues his adventures with Carla as they travel to many parts of the world. Together, they stay active in their local hemophilia community, sharing their story of recovery and resilience.

Adapted from Hello Factor magazine.

Visit bleedingdisorders.com to read more about patients like James and their stories.

Any factor and physical activity regimens mentioned were developed for James only after consultation with his care team, and may not be appropriate for all patients. Your factor regimen may be individualized after consultation with your care team.

This is a paid public announcement from Takeda and does not constitute an endorsement of products or services. When you click on the links in this blog entry, you will be directed to the bleedingdisorders.com website. LA Kelley Communications always advises you to be a savvy consumer when contacting any company; do not reveal identifying information against your will.

Copyright © 2020 Takeda Pharmaceutical Company Limited. 300 Shire Way, Lexington, MA 02421. 1-800-828-2088. All rights reserved. TAKEDA and the TAKEDA logo are trademarks or registered trademarks of Takeda Pharmaceutical Company Limited. US-NON-2025v1.0   08/20

Women, the community, and hemophilia B

There are many facets to our community. I think you could say there are as many facets as there are people with bleeding disorders. People may identify by the type of bleeding disorder they have, or the treatment they are on. But it’s important to remember this is a community in every sense of the word. That means everyone is important. And we should take a moment whenever we can to show our gratitude for the people who help hold everything together. Please read the public-service message below from Medexus to see what one company is doing to honor those people.

Here's what's happening in the hemophilia B space!

Medexus Pharma, the people behind IXINITY® [coagulation factor IX (recombinant)], has launched a webpage specifically to honor the remarkable women they are calling the Heroines of Hemophilia B. These are women who advocate for themselves—as women with hemophilia B—or have made an impact lending their support to others.

The work women do as healthcare professionals is absolutely essential. And many times, the key provider of care in the family setting is also a woman. Then there are the women who have hemophilia B. As a patient population, these women are often overlooked. It's time they got the care and support they deserve.

One of the great things about the Heroines of Hemophilia page is that it gives you a way to show your gratitude by nominating someone in your life. If you know of a woman who has courageously taken a stand to advocate for herself, or gone over and above in the care of someone else, go to www.HeroinesofHemophiliaB.com and nominate her as a Heroine of Hemophilia B.

This is a community in every sense of the word. Here’s a way to show to it.

INDICATIONS AND IMPORTANT SAFETY INFORMATION

What is IXINITY®?

IXINITY [coagulation factor IX (recombinant)] is a medicine used to replace clotting factor (factor IX) that is missing in adults and children at least 12 years of age with hemophilia B. Hemophilia B is also called congenital factor IX deficiency or Christmas disease. Hemophilia B is an inherited bleeding disorder that prevents clotting. Your healthcare provider may give you IXINITY to control and prevent bleeding episodes or when you have surgery.

IXINITY is not indicated for induction of immune tolerance in patients with Hemophilia B.

IMPORTANT SAFETY INFORMATION for IXINITY®

  • You should not use IXINITY if you are allergic to hamsters or any ingredients in IXINITY.
  • You should tell your healthcare provider if you have or have had medical problems, take any medicines, including prescription and non-prescription medicines, such as over-the-counter medicines, supplements, or herbal remedies, have any allergies, including allergies to hamsters, are nursing, are pregnant or planning to become pregnant, or have been told that you have inhibitors to factor IX.
  • You can experience an allergic reaction to IXINITY. Contact your healthcare provider or get emergency treatment right away if you develop a rash or hives, itching, tightness of the throat, chest pain, or tightness, difficulty breathing, lightheadedness, dizziness, nausea, or fainting.
  • Your body may form inhibitors to IXINITY. An inhibitor is part of the body’s defense system. If you develop inhibitors, it may prevent IXINITY from working properly. Consult with your healthcare provider to make sure you are carefully monitored with blood tests for development of inhibitors to IXINITY.
  • If you have risk factors for developing blood clots, the use of IXINITY may increase the risk of abnormal blood clots.
  • Call your healthcare provider right away about any side effects that bother you or do not go away, or if your bleeding does not stop after taking IXINITY.
  • The most common side effect that was reported with IXINITY during clinical trials was headache.
  • These are not all the side effects possible with IXINITY. You can ask your healthcare provider for information that is written for healthcare professionals.

Please see click here for full Prescribing Information.

You are encouraged to report side effects of prescription drugs to the Food and Drug Administration. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Aptevo BioTherapeutics LLC, Chicago, IL 60606

IXINITY® [coagulation factor IX (recombinant)] and any and all Medexus Pharma, Inc. brand, product, service and feature names, logos, and slogans are trademarks or registered trademarks of Aptevo BioTherapeutics LLC and Medexus Pharma, Inc. in the United States and/or other countries.

© 2020 Medexus Pharma, Inc.    All rights reserved.     MP-IX-0295

Wheels for the World 2020: A Win-Win

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!

The Antonio Boys!

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?

Laurie with brother Jim

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.

Barry's Spirit

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!

Barry Haarde

Copay Help? Thanks a Lot, Payers

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 costsbut 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

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