Fall 2023 The Vessel Newsletter

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THE VESSEL The Official Newsletter for The Bleeding Disorders Association of Northeastern New York

FALL 2023

BDA COMMUNITY EVENTS ~ LET’S WALK! Our walk is almost upon us! It’s a fabulous day to come out and show your support of the BDA, learn about what programs and services we offer, make friends and have fun! This year we will be walking in the beautiful Schodack Town Park, off Poyneer Road in East Schodack, NY. Activities include: bouncy basketball, lawn games and a DJ for all ages. We will have nurses demonstrate how to self-infuse, so don’t miss this hands-on training. Our theme is pajama party and we will be awarding prizes for the most creative, the most comfy and the most funny get-ups.

Any donation of $25 or more gets you a t-shirt, so that you can show your support year round. So grab your BFF, your know-it-all older sibling and your annoying kid brother and join our Pajama Party! ...fair weather predicted!

In This Issue • BDANENY Upcoming Events • UNITE Walk • Medicare News

• NHF Rebranding • Alternative Funding Models • Industry Update • Pathway to Cures

From the Executive Director As I have mentioned in previous columns, Fall is my favorite season. The nip in the air, the brightly colored foliage, the cinnamonspiced everything…plus Thanksgiving, the GOAT of holidays. But the one thing I certainly don’t look forward to during this autumnal time is reviewing my health insurance plan and gambling on a choice for the coming year. “Should I have gone for a lower deductible? Should I have picked a plan with more in-network specialists?” Some years these are questions that are thankfully, only rhetorical, but some years, they have extremely expensive consequences.

from healthcare.gov (the Exchange) and Medicare and Medicaid. In each of these sectors, consumers must choose among several tiers of coverage, high deductible plans, managed care plans, and fee-for-service systems. And, these plans may or may not include pharmaceutical drug insurance with its tiers of coverage, copays or co-insurance. This means dealing with myriad regulations about usage, coding, and billing; and these activities come with a large administrative cost. Finally, we don’t have enough health care professionals. Even before the “Great Resignation,” multiple industry analysts raised concerns about a shortage of physicians, nurses, and allied healthcare professionals. Because their services are in such demand and because there are so few high-level professionals, US doctors and nurses are paid more than in any other country in the world.

In 2023, the premium for my health insurance plan increased by 16% and it is expected to go up again in 2024. I will have to look hard at the available plans and decide whether or not I want to swallow that per month increase or shift the cost to my deductible. Or should I choose a new plan that excludes my long-time optom- And as someone has to foot the bill for that cost, much of it is etrist? These are hard choices that individuals and families are passed along to the end-user. For most Americans, right now is having to make in the face of escalating health care costs. the time to pick your health insurance. Medicare beneficiaries can choose a Medicare Advantage plan or a Part D prescription drug I used to do a lot of public talks on health care, and I would alplan. People with coverage at work can choose from the options ways start out by putting the healthcare revolution in perspective. their employers offer. People who buy their own insurance can Before World War I, most people died at home, a quick death that make a choice on the ACA marketplace in their state. In 2022, would be unthinkable in our modern world. They died of infectious there were over 60 plans available to individuals in New York diseases, edema, diarrhea, pregnancy, workplace accidents, and State. The range of choice is generally heralded as a good thing: a few succumbed to old age. Pneumonia was known as the old Not everyone wants the same plan, the thinking goes, so offering man’s friend. Strikingly, World Ward II brought about not only a multiple options helps people shop for the one that is best for new global power structure and economy, but a reduction in them. But it turns out in real life most people are terrible at picking premature deaths, and it was all due to widespread use of antibi- the health plan that is right for them. Health insurance is a compliotics. cated financial product, and study after study has shown that people routinely pick bad plans, even choosing options that leave Penicillin was discovered in 1928 – the same year that my father them worse off financially in every possible scenario. Picking an was born – and revolutionized the provision of health care. Flem- ideal health plan requires knowing what you might use, what it ing’s discovery of penicillin is revolutionary because it changed might cost you, and how those expenses combine with the plan’s the worldview of the way doctors treat patients with infectious monthly premium. diseases, and as a result of the antibiotic revolution, individuals are not as vulnerable to death by bacterial diseases. In addition to Picking a plan is hard, but some simple guidelines help a little. It’s treating infectious diseases, antibiotics made many modern medi- helpful to know whether a given plan covers the doctors and hoscal procedures possible, including cancer treatment, organ trans- pitals you use, for example. And if you’re willing to take more fiplants and open-heart surgery. nancial risk, you may prefer a higher-deductible plan with lower

Each year medical science advances at a pace our ancestors could only dream of. In the span of one man’s life, we have gone from preventing gangrene to CRISPR gene editing. It’s a truly miraculous human accomplishment – but one that does not come without costs. Economists estimate that $4,660 per person was spent on health care in 2000—an increase of 838 percent from the $497 spent in 1950, assuming constant dollars. Medical technology is expensive, as is the growing pharmacopeia available to the public. Additionally, the U.S. healthcare system is highly complex, with separate rules, funding, enrollment dates, and out-of-pocket costs for employer-based insurance, private insurance

premiums, while if you value more predictable expenses, a lowerdeductible plan may work better. But people’s actual health care needs and insurance fine print vary enough that those guides can lead you astray. Most of the research on plan choice looks at the financial design of the plan, yet that approach leaves out some other elements of health plans, like the choice of doctors, or whether the company offers good customer service. The bottom line is: just buying health insurance is not enough. You need to ensure that you have picked the right insurer and the right plan. So, think about attending our Insurance Forum on October 21 at the Marriott Hotel to arm yourself with the knowledge to enable you to select the plan that’s right for you and your family.


Taking Action Against “Alternative Funding Models” As the federal government launches into drug price negotiation, a controversial form of drug price discounting is under increasing scrutiny. The National Bleeding Disorders Foundation (NBDF, formerly NHF) and the Hemophilia Federation of America (HFA), have joined a petition demanding that Payer Matrix stop representing itself as a patient advocacy organization. Payer Matrix, based in Media, Pennsylvania, is what is known as an alternative funding company. On behalf of employers and payers it enrolls patients in manufacturers’ patient assistance programs (PAPs). Alternative funding companies are under fire because to qualify patients for PAPs and charity assistance, they ask payers to selectively disqualify patients for coverage. This enables the alternative funding company to obtain a drug for free by representing to a PAP or charity that the patient has no coverage for that medicine. Payer Matrix was sued this year by AbbVie, which alleged that such practices are misleading, harmful to patients, and fraudulent. AbbVie contended that one reason why the business model is fraudulent is that patients are still technically covered even if they fail to qualify for PAPs. Alternative funding companies effectively switch off commercial coverage for specialty drugs so patients can apply for PAP or charity care. If they don't get it, their commercial coverage is switched back on, AbbVie said. If successful in this effort, the AFP vendor charges the employer a percentage of the employer’s expected savings (20 to 30% of list price of the employee’s drug) or a substantial per-employee, per-month fee.

NBDF contends that Payer Matrix was very far from a good fit for the term “patient advocate.”“Payer Matrix profits each time it successfully enrolls a patient in a PAP. As such, we believe it is highly inappropriate and misleading to consumers for Payer Matrix to classify itself as either a patient advocacy company or a leading patient advocate. Payer Matrix is a for-profit business representing the interests of employers and itself, not patients,” the NBDF said. Drug pricing experts have called the alternative payment business “shady,” saying it nets high profits for companies such as ImpaxRx, PaydHealth, PayerMatrix, RxFree4me, SHARx, but depletes PAP and charity dollars more rapidly and potentially leaves truly needy patients without access to essential medications. NBDF and HFA joined with 34 other advocacy groups representing 14 disease communities including Arthritis, Autoimmune disorder, Bleeding and Clotting disorders, Cancer, Cystic Fibrosis, Liver Disease, Lupus, Hepatitis, Hemophilia, HIV/AIDs, Neurological Conditions, Multiple Sclerosis, Psoriasis, Rare Disorders, and Spondylitis to urge Payer Matrix to stop identifying as either a ‘Patient Advocacy Company’ or a ‘Leading Patient Advocate’.

Medicare News The Biden Administration took a variety of actions in August to implement the Medicare Part D prescription drug reforms that were enacted as part of the 2022 Inflation Reduction Act. CMS released guidance detailing how Medicare Part D plans can structure new optional programs allowing seniors to spread out their out-of-pocket prescription drug spending over a 12-month period. These optional “prescription payment plans” will become available in 2025 and apply only to Part D prescriptions (most bleeding disorder products are covered under Medicare Part B). CMS also announced that it had selected the first ten Part D drugs that will be subject to price negotiation between Medicare and the drug manufacturer. This is only the start of the process: any negotiated prices will not be effective until 2026 and manufacturers have until October 1 to decide whether to participate in negotiations, pay an excise tax, or withdraw their products from coverage by Medicare. 3


The National Hemophilia Foundation is Now the National Bleeding Disorders Foundation The National Hemophilia Foundation (NHF) has officially changed its name to the National Bleeding Disorders Foundation (NBDF). Foundation leaders announced the change alongside advocates and supporters at the 2023 Bleeding Disorders Conference in National Harbor, MD. In addition to the new name, the National Bleeding Disorders Foundation unveiled a new visual identity and logo intended to represent a wide range of inheritable blood and bleeding disorders, as well as a new tagline: Innovate | Educate | Advocate. “Our new name is one that’s inclusive, trying to represent that entire blood and bleeding disorders community based on our past. We think that this really embraces what we’re going to offer into the future by bringing everybody in and offering them something. No matter what disorder you have, you will find a home in the National Bleeding Disorders Foundation,” said Dr. Len Valentino, CEO. In the near future, NBDF will seek ways to harness its resources and networks to help people facing an even broader array of inheritable blood and bleeding disorders, based on the understanding that bleeding disorders are blood

Pathway to Cures Announces First Venture Investment Pathway to Cures, an affiliate of the National Bleeding Disorders Foundation, is a venture philanthropy fund focused on early-stage companies developing cures, therapies, or enabling technologies in support of the inheritable blood and bleeding disorders community. Pathway to Cures, (P2C) announced last month its inaugural investment of $250,000 in Anvesana to develop therapies for treating inheritable blood and bleeding disorders. Anvesana is developing therapeutics that modulate mRNA biology to treat inheritable bleeding disorders such as hemophilia, as well as neurodegeneration and metabolic syndrome caused by insufficient protein production. The company’s RNA-sequence analysis platform provides insights into the underlying biology, rapidly prioritizing the right RNA species to target for a given disease. Anvesana’s platform consists of a distributed database that stores and searches thousands of RNA sequence datasets and a proprietary reference transcriptome containing over 250,000 annotations curated by a team of over 40 scientists. By leveraging the database and the reference transcriptome, Anvesana’s algorithms filter out many of the biological artifacts that doom otherwise promising RNA therapeutic programs. P2C’s goal is to bring investment dollars, knowledge, scientific, and community relationships together to facilitate development of new products to address the unmet needs of the blood and bleeding disorders community. P2C’s Scientific Advisory Group makes recommendations to the P2C Investment Committee regarding promising opportunities. Inheritable blood and bleeding disorders include rare and ultra-rare blood disorders, hemophilia A and B, von Willebrand disease, sickle cell disease, anemia, clotting disorders, and other hematological disorders, affecting more than 20 million people worldwide. In collaboration with other organizations, P2C invests in innovative therapies and technologies, leveraging the deep resources and scientific community relationships of its parent organization, the National Bleeding Disorders Foundation. By reinvesting proceeds from investments back into the Fund, P2C will amplify investment impact, support promising companies, and build a portfolio of investments that further the mission of the National Bleeding Disorders Foundation. 5

Industry Update Sanofi

pies manipulate a patient's genes at the cellular level to

The FDA has accepted a supplemental biologics li-

treat inherited diseases. But the therapies are among

cense application (sBLA) for ALTUVIIIO

the costliest treatments in the world. Hemgenix

(Antihemophilic factor; Sanofi) for managing bleeding (etranacogene dezaparvovec) gene therapy for hemoin patients aged younger than 12 years with severe he- philia B, has a price tag of $3.5 million, and Roctavian mophilia A.

(valoctocogene roxaparvovec) for hemophilia A, costs $2.9 million.

Patient Advocacy in Clinical Trials Members of the biopharmaceutical industry have

In addition to the lofty price tags of the therapies

teamed up with patient advocacy groups to form a

themselves, employers and payers are also confronted

consortium called the Patient Advocacy Leaders and

by the additional and substantial costs of administer-

Drug Development Industry Network (PALADIN).

ing the drugs, including hospital stays and follow-up

The past decade has seen a proliferation in the number visits.

of investigational therapies and the number of companies sponsoring active clinical trials.

Given the FDA pipeline of therapies targeting much

In recent years, the biopharmaceutical industry has

more common medical conditions, experts expect

elevated the role of the patient, acknowledging that

claim volume and severity to increase in coming

patients are the experts in what it’s like to live with

years. It is estimated that more than 1 million individ-

their disease or condition.

uals will be eligible for gene and cell therapy treatment within 12 years. Pharmaceutical companies

PALADIN, which has 23 member organizations,

claim that over time, the therapies will pay for them-

launched in March and aims to carve out meaningful

selves, as opposed to the cost of on-going prophylactic

roles for advocacy groups and patients in clinical re-

treatment, but failed treatments mean patients must

search trials, the regulated scientific process that ex-

return to the hemophilia therapies they were on previ-

amines the safety and efficacy of potential new treat-

ously, be it on-demand or prophylaxis bleeding con-

ments. The Tufts Center for the Study of Drug Devel-

trol products, potentially leaving payers on the hook

opment (Tufts CSDD), part of Tufts University School for the cost of multimillion-dollar gene therapies that of Medicine, is providing project management support did not work. for the Consortium. Outcomes-based therapy, also known as precision reInsurance Coverage for Gene Therapy

imbursement or “no cure, no pay,” has been discussed

Major insurers are eyeing and developing new strate-

for years as a way to ease the financial risk that payers

gies to help employers cover the cost of gene therapies must bear, but it has failed to gain widespread traction, priced at millions of dollars per treatment. Gene thera- for a number of reasons. The extraordinarily high 6

Industry Update cont’d. price of gene therapy, coupled with the lack of clini-

laxis, the cost of 1 mg of emicizumab would need to

cal data on durable outcomes and the limited patient

decrease by approximately 35% to be cost-effective

population for these therapies, appears to have

over a lifetime. Prior studies have analyzed the cost-

brought about a change.

effectiveness of prophylactic emicizumab in the context of patients with severe hemophilia A where emi-

BioMarin Pharmaceutical, the developer of Roctavi-

cizumab prophylaxis was found to be cost-saving

an, has announced intentions of reimbursing up to

among patients with severe hemophilia A

100% of the cost should patients fail to respond or cease to respond in the first four years after admin-

Spark Biomedical

istration. Meanwhile, CSL Behring, the developer of

Five Liters, a subsidiary of Dallas-based Spark Bio-

Hemgenix, reports to be negotiating with payers to

medical is conducting “first in-human” studies to

offer a similar deal. The promise of more gene thera-

demonstrate the effectiveness of noninvasive weara-

py treatments in the pipeline is sure to lead to more

ble neurostimulation in reducing blood loss, particu-

creative negotiations. A similar treatment for sickle

larly for individuals with blood disorders or undergo-

cell is expected to be approved in 2024.

ing surgical procedures. Past studies have shown that electrical activation of neural pathways to the spleen


can prepare the body for clotting in case of a wound,

A study recently published in the American Journal of leading to a 50 percent reduction in bleeding time and Hematology reports that at current pricing, the price

volume. Should this new study show positive results,

of prophylactic emicizumab (Hemlibra) would need

Five Liters said it would prioritize Von Willebrand

to decrease by >35% to become cost-effective in US

Disease (VWD).

adults with moderate or mild hemophilia A without inhibitors. “Prior studies have analyzed the cost-

Five Liters is set to launch a clinical trial later this

effectiveness of prophylactic emicizumab in the con-

year, focused on utilizing Transcutaneous Auricular

text of patients with severe hemophilia A,” the re-

Neurostimulation (tAN) therapy to alleviate menor-

searchers wrote in their report. “The cost-

rhagia in females with VWD and the company said it

effectiveness of emicizumab in patients with non-

is optimistic about the potential for a non-invasive,

severe hemophilia A without inhibitors is not current- non-pharmacological treatment option. ly known.” Sanofi Though patients who received emicizumab prophy-

Two Fitusiran Phase 3 studies, published in The Lan-

laxis experienced 62% fewer treated bleeding events

cet and The Lancet Haemotology highlight its poten-

and 64% fewer hemarthrosis events over their lifetime tial to address unmet needs across all types of hemothan those who received recombinant FVIII prophy-

philia. Fitusiran is an investigational small interfering Cont. pg. 10


Industry Update, cont.d RNA (siRNA) therapeutic for hemophilia A and B. It works by making more thrombin available for clotting. In the recently published phase 3 studies, Fitusiran was able to significantly reduce bleeding in hemophilia A and B (89.9%-90.8%), even in patients with inhibitors to clotting factor VIII or IX (FVIII, FIX). The drug “silences” antithrombin to allow background thrombin to produce clots more readily. For patients who de-

pend on FVIII and FIX replacement therapy administered several times a week, fitusiran demonstrated potential as a once-monthly prophylactic treatment able to control bleeding without peaks or troughs in active drug levels. Sanofi is currently investigating the efficacy and safety of fitusiran under a revised regimen which includes lower doses and less frequent dosing (as few as six subcutaneous injections per year), maintaining an antithrombin target range of 15-35% in all ongoing studies. Hemab Therapeutics Hemab Therapeutics announced results of pre-clinical research of HMB-001 to treat Factor VII deficiency. HMB-001 has the potential to transform treatment in several serious bleeding disorders and the company has already initiated a Phase 1/2 study in Glanzmann Thrombasthenia as well. HMB-001 is a bispecific antibody that binds to and stabilizes endogenous activated FVII (FVIIa) with one antibody arm and localizes FVIIa to the surface of activated platelets by binding to TLT-1 with the other arm. This allows for accumulation of FVIIa in the body and recruitment of FVIIa directly to the surface of activated platelets at the site of vascular injury where FVIIa is known to facilitate the formation of protective hemostatic plugs to stop bleeding. Hemab is targeting the development of therapies to treat underserved bleeding and thrombotic disorders, including Glanzmann Thrombasthenia, Factor VII Deficiency, Hemophilia B with inhibitors, Bernard Soulier Syndrome, Von Willebrand Disease, Hereditary Hemorrhagic Telangiectasia (or Osler-Weber-Rendu dis-

ease), Congenital Antithrombin III Deficiency, and others.


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BDANENY Board of Directors and Staff President…………………….…………..…Bruce Weiner Vice President…………………….………..Chris Huskie

Treasurer………………………………….....Cindi Keeler Secretary……..…….………….…..Christina Mastrianni Board Member…………………..Juliemar Abreu Boria Board Member ….……………...Mary Anne Cecchetto B

Board Member………..……...…....…Matthew Delaney Board Member………………………..….Linda Peacock Board Member……………..……………..Jessica Smith Executive Director……………..…….….…..Beth Mahar Operations Manager………………....Patricia Bengraff Special Events & Social Media…..…......Melissa Deeb

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