BDANENY December 2021 Newsletter

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

December 2021

BDANENY 2021 In-Person Events Although we spent most of 2021 hunkered down in our home offices behind our N-95 masks and bottles of hand sanitizer and Lysol, in the short window of opportunity afforded to us in 2021, the BDANENY held several in-person events!

pled ice cream treats while enjoying a screening of “Bombadier Blood” at the Jericho Drive-In on October 14. Sponsored by OPTUM and Novo Nordisk, we were able to sample all the snack bar had to offer.

We held our Annual Meeting at Liberty Ridge Farms in Schaghticoke on October 24, where we conducted As previously reported, we raised funds and polished official business, hosted two educational sessions— our beanbag tossing skills at the End of Summer Un”Dealing with Stress through Color Therapy,“ presentWined Event & Cornhole Tournament on August 28 ed by our friends at OPTUM and “Navigating School at Galway Rock Winery in Ballston Spa. Despite a with a Bleeding Disorder,” presented by our friends at smattering of rain, the competition was intense, but Takeda. Then, it was off to the corn maze and the 32+ friendly. other attractions the farm had to offer. All of our atWe dusted off our running (okay, walking) shoes for tendees were happy to interact with our 14 exhibitors, the UNITE for Bleeding and happier to sample the delicious brisket the farm Disorders at Gavin Park in served up. Wilton on September 24. Finally, we kicked up our heels at the Black & Blue The weather cooperated Bash at the Embassy Suites in Saratoga Springs on and our fabulous walkers November 6. Bidding on Silent Auction items was fast had a beautiful day in the and furious with many walking away with terrific barpark. Thank you to everygains! A huge thank you to the Chrzan Family for coone who came out, despite ordinating the event and securing the most amazing a year off and a new locaSilent Auction packages and wine pull bottles. tion. Although we’re back inside again for a few months, We gathered under a perwe made the most of the opportunities to meet face to fect starry night and samWalkers on 9/24 face. See the pics inside this edition!

In This Issue •

UNITE Walk, Movie Night, Annual Meeting, Black & Blue Bash

Farewell to 2021

Industry Update

Legislative & Regulatory Update

Resources for Help

Scholarship Applications



A Word from the Executive Director And so this is Christmas, And what have you done? Another year over, A new year just begun…

mental health, our mental habits and our social behaviors impact our health almost as much as diet and exercise.

Perhaps this has been why we have seen such an increase in bad behavior. Flight attendants seem to be taking the brunt of it. Every day a new story is reported, showcasing how airline passengers are assaulting flight attendants and other passengers over mask mandates. But NBA fans have also exhibited less than gracious behavior, screaming insults at players and throwing food and drink on the court. And many retail and restaurant workers have been verbally insulted and, at times, physically attacked just for doing their jobs. Not to mention certain politicians….

So my goal for 2022 is to be kinder—to everyone around, but also to myself. Here’s hoping that we can make our community a little nicer, a little friendlier, and make 2022 a little better than 2021.

So how can we escape this languishing stage and reYes, it’s that time again. Time to look back and take turn to a meaningful state of being? While finding stock, and then look ahead and set goals. As we close purposeful challenges, enjoyable experiences and enin on year three of the pandemic, and as we are faced gaging work are all possible remedies to languishing, with the Omicron wave- which is proving to be the it’s hard to get started when you can’t focus. most contagious strain of the virus- it seems as though The secret may be in the maxim taught to me by my we’re beyond worry or depression. Many of us feel kindergarten teacher, Mrs. Kleckner, “Life is hard by somewhat joyless and aimless. It turns out there’s a the yard. By the inch, it’s a cinch.” In other words, name for that: languishing. Languishing is a state of start small. Start with a meaningful conversation or a stagnation and emptiness. It’s the void between desmall act of kindness. Connect with your neighbors. pression and flourishing, the sense that you’re conReally listen when talking with someone, instead of stantly looking through a foggy windshield. thinking about what you’re going to say in return. Tip Early in the days of the pandemic, we were all on high the worker at the drive-thru. Let someone go in front alert. Every grocery bag or package that came into the of you in line. Call your mom or dad or kids just to home was a potentially deadly vehicle, a way for the say, “I love you.” My son came home from getting his unseen enemy to invade our happy homes. But the COVID booster to tell me that a stranger had told him virus continues to mutate in ways that are still threat- that his coat was looking pretty sharp. He told that ening, but no longer highly alarming. Our COVIDstory multiple times on Christmas and smiled every related stress has become a chronic condition. time he did.

This bad mental territory is equally bad for our health and longevity. More and more studies show that our 3

Do You Need Help? Living with a bleeding disorder can be a substantial financial burden. The pandemic has made things worse. Where can you turn for help?


Hemophilia Federation of America (HFA) offers several programs for people with bleeding disorders:

PAN Foundation: Assistance with out-of-pocket costs

Helping Hands Emergency Assistance Program helps with housing, utilities and transportation. Helping Hands Item Assistance Program helps with medically necessary items that are not covered by insurance. Helping Hands Inhibitor Support Program provides a safety net for families affected by inhibitors. Pharmaceutical Corporations: Many pharmaceutical companies offer co-pay and insurance coverage support. Bayer: Jivi, Kovaltry, Kogenate CSL Behring: support-and-assistance Genentech: Hemlibra, Genentech Patient Foundation Grifols: support-and-resources/factors-for-health

Other Resources:

The Assistance Fund: Co-pay assistance; Premium assistance; Incidental medical expenses Patient Services, Inc.: PSI assists with patient travel expenses related to receiving medical care for the program diagnosis. This may include fuel, ambulance services, lodging, and public transportation. Patient Advocate Foundation Co-Pay Relief: Covers co-pay, co-insurance, deductible Colburn Keenan Foundation: The Foundation’s individual grants are meant to help individuals and families meet their urgent medical, basic survival, and quality of life needs. Social Services Supports

Supplemental Nutrition Assistance Program: IncomeMedexus: Custom Ancillaries (Supplies at no charge); based monthly benefit used to purchase food https:// IXperience Concierge Novo Nordisk: Product Assistance; Co-pay assistance HEAP: Help paying for your energy bill https:// Nuwiq; Wilate assistance-heap Pfizer: Sanofi/Genzyme: Alprolix; Eloctate

Child Care Subsidy Program: can help parents/ caretakers pay for some or all of the cost of child care services.

SAVE THE DATE! Albany Days will be held at the Renaissance Hotel March 26—28, 2022 4

2021 UNITE Walk—THANK YOU!!!

A HUGE Thank you to our fabulous Walk Teams: Chrzan Crusaders Carter’s Crew The Bruizers Blood Buddies HyperDuncs CAN’T CLOT THIS Bloody Awesome Coulombes Factor Endurance Network


Legislative & Regulatory Update—Federal the bundled payment rate did not adequately cover the cost of clotting factor therapies.

Supreme Court

American Hospital Association v. Beccera is a case pending before the Supreme Court of the United States. The case centers around a rule from the Department of Health and Human Services which reduced reimbursement rates for certain hospitals. Several hospital associations and hospitals affected by the rule sued the DHHS, alleging that it has exceeded its statutory authority. The rule being challenged deals with 340B hospitals and Medicare Part B insured patients. The government used to reimburse hospitals at a uniform rate if the hospital provided outpatient care to Medicare Part B recipients. The DHHS then changed the rule to reduce reimbursement rates for 340B hospitals, because they can get drugs at lower costs. Back in 1992, Congress created something called the 340B program to support health-care providers that serve poor and disadvantaged communities. Eligible providers get steep discounts on the drugs that they purchase — anywhere between 20% and 50% of the normal price. Initially, few hospitals qualified for the 340B program. Today, more than two-thirds of non-profit hospitals participate. HTCs are 340B entities. The Court heard oral arguments in November and are expected to render a decision in early 2022.

Federal Legislation Congress Passes Hemophilia SNF Access Act Starting October 1, 2021, SNFs can bill separately for hemophilia and other inherited bleeding disorder treatments administered to Medicare beneficiaries needing care in a SNF. This change allows the SNF to either provide the bleeding disorder treatment(s) themselves or contract with a third-party such as an HTC or specialty pharmacy. Medicare covers short-term stays (less than 100 days) in SNFs. SNFs are paid a prospectively determined daily rate for all SNF services provided to patients covered under a Medicare Part A stay. This bundled payment includes nursing and therapy components, drugs, supplies, and equipment, in addition to a room/board and administration component to cover the cost of a typical patient. Prior to the passage of the Hemophilia SNF Access Act, very few SNFs would admit patients with bleeding disorders because 6

Under current law, Medicare allows for certain costly, highly specialized services that SNFs do not typically provide to be billed separately. As of October 1, bleeding disorder treatments have been added to this list. Separate billing under Medicare Part B for bleeding disorder treatments for SNF patients will rectify a long-standing problem for our community and will facilitate placing an individual with a bleeding disorder in a SNF, provided they meet the clinical criteria for placement. The change in the law applies to treatments for all inherited bleeding disorders including but not limited to those needed to treat hemophilia A, hemophilia B, and Von Willebrand disease (VWD). While the bill included hemophilia in its name, it included all inherited bleeding disorders. This change allows for all treatment modalities used to treat an inherited bleeding disorder, not just clotting factor. Should a new treatment or therapy come to market, the Secretary of HHS has the authority to expand the list of products that can be billed separately. In addition, during the annual rulemaking for the Medicare SNF benefit, advocates can recommend additions or other changes as part of the comment process. This law applies to Medicare beneficiaries who are admitted to a skilled nursing facility following a qualified hospital stay of at least three days. Bleeding disorder beneficiaries are likely to use this benefit following surgery or a prolonged inpatient hospital stay. SNFs will be able to bill for bleeding disorder treatments the same way they bill for other Part B excluded services like chemotherapy and prosthetics. The SNF will know their billing methods for these excluded services. Co-pays for beneficiaries will be required. These may be higher than what the beneficiary is used to paying as most SNFs require that residents utilize their pharmacy services. Coverage under private insurers varies.

Federal Accumulator Adjuster Bill Introduced In November, the Help Ensure Lower Copays Act was introduced in the House of Representatives. This bill would apply additional payments, discounts, and other financial assistance toward the cost-sharing requirements of health insurance plans. The bill was introduced by Congressman A. Donald McEachin (D-VA) and Congressman Rodney Davis (R-IL). Currently, only 12 states have passed similar legislation.

Legislative & Regulatory Update—State 2021 NYS Public Policy Report PRIORITY ISSUES The use of utilization management practices like formulary controls, co-pay accumulators and prior authorization by health plans and Pharmacy Benefit Managers (PBMs) can create barriers to care. These practices remain a leading challenge for the bleeding disorders community. Mid-year formulary changes: also known as NonMedical Switching. A bill limiting NMS recently passed the Legislature after a pervious bill was vetoed in 2020. The New York State Bleeding Disorders Coalition (NYSBDC) has led a broad coalition of patient and provider groups to urge Governor Hochul to sign the bill into law. It was signed on 12/22/21. Pharmacy Access: the Rutledge decision handed down by the Supreme Court in 2020 has spurred state efforts to regulate PBM practices. Several bills expanding patient access to local pharmacy options and limiting forced mail order situations passed the legislature in 2021. The NYSBDC is working with pharmacists and other patient groups to get the bill signed into law by the end of the year. Co-pay accumulators: a bill requiring plans/PBMs to accept all 3rd party patient assistance easily passed the Senate, then stalled in the Assembly, due to cross-chamber negotiations. The NYSBDC is working with other patient groups to pass the bill in the Assembly and have it signed into law in 2022. Prior Authorization: a prior authorization reform bill was introduced last year and is currently on hold awaiting the results of a NYS Department of Financial Services Work Group. The Work Group of health plans, hospitals, clinicians and patients addressed topics including prior authorization and recently issued its final report NYSDFS: Report of New York’s Health Care Administrative Simplification Workgroup – October 3, 2021. The report makes several recommendations and we continue to review it in hope of seeing positive changes. We will also continue to work on the prior authorization bill should that prove to be a better route to

progress. Step Therapy: the national advocacy organization Aimed Alliance is studying ways to update NYS step therapy regulations enacted in 2016. The NYSBDC is working with Aimed Alliance and other patient and provider organizations to examine and advance options for step therapy reform in NYS for 2022. NYS MEDICAID Many people with bleeding disorders receive care through the NYS Medicaid program, making Medicaid a priority. Here are two Medicaid issues we will be following in 2022: The transition of Medication Pharmacy Benefits from managed care back to a fee-for-service program overseen by the State Department of Health has been pushed back to 2023. The transition is meant to give DOH greater leverage over medication costs. DOH has said clotting factor access should not be affected by the transition or the delay, but we will be watching closely to be sure this is the case. Additional federal funding from the COVID relief bill earmarked for NYS Medicaid expires on January 21, 2022. Prior to this supplemental funding, NYS Medicaid had a +$2 billion deficit by exceeding the Medicaid global spending cap. Whether the COVID funding was used to fill the deficit hasn’t been reported, nor is the future of the muchcriticized global cap. Since the cap was put in place at the outset of the Cuomo Administration – along with the Medicaid Redesign Team and all its initiatives – a new administration could take an entirely different tack in negotiating a 2022-23 budget. AREAS OF CONCERN Poverty: One in six Americans, including one in three children, live in poverty. Poverty has serious, lifelong impacts on people, especially those with a chronic illness. COVID has highlighted the need for robust support programs for those with low incomes and insecure work situations. Pain Management: Increasing numbers of patients Continued on p. 9 7

BDANENY 2022 SCHOLARSHIPS Are you a high school senior who plans to attend a vocational school or undergraduate program? The cost of a higher education increases every year, yet in today’s world, a degree is increasingly necessary. At the BDA, we are here to help. The Association designates funds for the higher education of its members at accredited vocational schools and undergraduate programs. Applicants must be individuals with or immediate family members of individuals with a congenital bleeding disorder and be dues-paying members of the Association. One award will be granted per year for members living within the same immediate household. Depending upon available funds and the degree program in which enrolled, the BDANENY will award up to two $1000 scholarships per academic year. Applications must be submitted on or before March 15, 2022.

Click here to download an application and click here to download the rubrics used for evaluating each scholarship.

Legislative & Regulatory Update—State (cont.) struggle with chronic pain, yet treatment options are limited and payers reluctant to cover new or long-term pain treatments. The lack of treatment options and coverage is exacerbating a growing crisis in our community. Mental Health: As more patients experience chronic pain and other impairments, there has been a serious rise in the rates of depression, substance abuse and suicide. Again, the pandemic has only exacerbated a bad situation. Age-Related Needs: As more individuals with bleeding disorders are living longer than ever before, their need for specialized long term care increases. Will our health care system be able to provide the individualized care they need? HTC Viability: Utilization management and the growing use of non-factor products are impacting HTC revenue. A serious reduction of HTC revenue will undermine the patient care that HTCs provide. Many HTCs are closing or curtailing services. TRENDS TO WATCH Telemedicine: COVID sparked a surge in the use of telemedicine, likely to continue post-pandemic, as clinicians, patients and payers seek more efficient healthcare tools. In a state like New York, where many do not have access to a full-service HTC, telemedicine could fill the access gap. Medication Costs: Patients with high-cost conditions have largely avoided debate over how medication prices drive healthcare costs. Several factors are coming into play which may change this situation and impel consumers toward action. These include: The efforts of the Institute for Clinical and Economic Review (ICER), an independent non-profit organization that seeks to place a value on medical care by providing comprehensive clinical and cost-effectiveness analyses of treatments tests and procedures. ICER has placed a monetary value on a number of prescription drugs since 2014. Those evaluations have been used by insurers to justify which drugs are approved or denied. The controversy over Aduhelm, the first new

Alzheimer’s drug approved since 2003. Of the two large studies of Aduhelm submitted to the FDA, one found that the drug was able to delay the loss of memory and thinking, while the other found no clear benefit. Combined with a $36,000 per year price tag, insurers, including CMS, are reluctant to approve it. Gene Therapy clinical trials are progressing rapidly, but the ultimate costs can be staggering. Near the top is the first approved one-time gene therapy Luxturna developed by Spark Therapeutics, which cures an inherited retinal disease that leads to blindness; its price tag: $850,000. Artificial Intelligence: Hospital systems, pharma, payers and brokers are collecting more data than ever in an effort to manage care costs. Now, they’re applying A.I. to the data, which soon may mean employing an algorithm to decide who gets what care. The Walmart Model: Retail giant Walmart is using their enormous footprint to deliver discount drugs and a growing number of medical services through their stores. They’re also joining Costco, Microsoft, Boeing and 40 other companies in a venture called Purchaser Business Group on Health to provide members with cheaper employee health coverage and a PBM called EmsanaRx. While ideas like association health plans and purchasing pools aren’t new, rising costs are driving employers to innovate more. Employer provided healthcare covers half of all Americans and strategies such as paying patients for certain health behaviors are increasingly common. BDANENY ADVOCACY PROGRAMS Albany Days (on-line) – March was once again Bleeding Disorders Awareness Month and we held our annual training (including our READY program) and advocacy day online for the second year. Virtual Advocacy Day #1- As the legislative session wound down in June, we took to emails and social media in a final push for our bills.

INDUSTRY UPDATE Bespoke Gene Therapy Consortium Launched

funding which it plans to use to further develop AMA005 an investigational gene therapy for individuals with hemoOn Wednesday, October 27, 2021, the NIH announced philia B. Amarna Therapeutics is a privately held, prethat they will be partnering with the FDA and 15 private clinical biotech company developing gene therapies for a organizations to increase the effectiveness of gene thera- range of rare and prevalent diseases. The company is piopies for rare diseases. The 15 private organizations inneering the development of gene therapies based on simian clude 5 non-profit organizations and 10 pharmaceutical virus 40 (SV40), a virus which normally infects a certain companies. Five major pharma companies have signed up species of Asian monkeys and to which humans are immuto be part of the BGTC – Biogen, Janssen, Novartis, Pfizer nologically naïve. Amarna has created a proprietary proand Takeda – plus smaller companies like REGENXBIO, duction cell line (SuperVero) that for the first time makes it Spark Therapeutics, Taysha Gene Therapies and Ultrapossible to produce SV40-derived vectors suitable for thergenyx, as well as Thermo Fisher Scientific. The non-profit apeutic use. Modified viruses are effective at inserting gepartners include the Alliance for Regenerative Medicine, netic material into cells, since that is the mechanism by the American Society of Gene and Cell Therapy and the which they normally infect cells. National Organization for Rare Disorders. Amarna and its manufacturing partner Halix are both headThe newly launched Bespoke Gene Therapy Consortium quartered in the Netherlands. (BGTC) which is part of the NIH Accelerating Medicines Partnership (AMP) program aims to develop platforms and standards that will speed the development and Findings of several gene therapy clinical trials were predelivery of customized “bespoke” gene therapies that sented at the 2021 American Society of Hematology Annucould treat the millions of people affected by rare diseas- al Meeting and Exposition, held in Atlanta, Georgia on Dees. BGTC aims to develop a “standardized” pathway for cember 11 – 14, 2021. developers to follow, which include assessing the basic AMT-061 (uniQure/CSL Behring) biology of adeno-associated virus (AVV), simplifying the path of translation from animal model studies to hu- Data from the HOPE-B Phase 1/2 Clinical Trial of AMTman clinical trials, developing a standard set of analytic 061 shows the therapy can prevent bleeds in patients with tests to apply to the manufacture of viral vectors and moderate to severe hemophilia B for at least 18 months and streamline the regulatory requirements surrounding the possibly for several years. The HOPE-B study is evaluating preclinical studies (e.g., toxicology safety studies). AMT-061’s five-year safety and effectiveness in 54 men. To start, the BGTC plans to initiate preclinical and clinical trials for four to six rare diseases that currently lack any treatment. The Consortium will provide $76 million over five years from NIH and private partners to fund a range of research. Efforts will be made to leverage methods and data from one application to another to reduce prohibitive R&D costs that threaten product development and patient access.

70% of participants had had a bleed in the six-month period prior to the trial, despite prophylaxis. Data from the study proves AMT-061 is statistically superior at reducing the patients’ annualized bleeding rate when compared to standard prophylaxis.

AMA005 (Amarna Therapeutics) Amarna Therapeutics has secured €5 million ($5.6) in new

A clinical trial of investigational gene therapy SB-525 found that a single dose prevents bleeds and the need for

AMT-061 was developed originally by uniQure; in May, CSL Behring acquired commercial and licensing rights to the therapy. uniQure remains responsible for completing This public-private collaboration is particularly timely in the HOPE-B trial, which is expected to be completed in the wake of recent patient deaths and safety issues linked March 2025. CSL Behring plans to seek approval of AMT061 in the US and the EU by June 2022. to experimental gene therapy involving AVV delivery, particularly liver damage. There is also a need to follow study participants long term to discern any side effects. SB-525 (Pfizer & Sangamo)

prophylaxis for at least two years in men with severe hemophilia A. The ongoing Alta Phase 1/2 Clinical Trial showed a sustained increase in the activity of clotting factor VIII (FVIII) which remained in the therapeutic range through two years post-dosing. SB-525 is being developed by Sangamo Therapeutics in collaboration with Pfizer.

detected in some participants, thus putting them at greater risk of blood clots. FLT180a (Freeline Therapeutics) B-AMAZE, a Phase 1/2 study of FLT180a found sustained levels of factor IX (FIX) for more than three years, in study participants with hemophilia B. The next step in the process for maker Freeline Therapeutics is their B-LIEVE dose-confirmation study, launched on December 6, to identify a dose and prophylactic immune management regimen to keep hemophilia B patients in the normal range of FIX expression. The B-LIEVE study is expected to yield interim data in mid-2022.

The on-going study is assessing the five-year safety and efficacy of four different doses of SB-525. Results show that SB-525 is generally well-tolerated; the most common adverse effects were infusion-related reactions and higher than normal levels of liver enzymes, which were fully resolved with oral corticosteroid treatment. At last assessment, no patient had experienced FVIII inhibitors, blood clot events or liver masses – side effects which were preFLT180a is generally well-tolerated; the most common sent in earlier gene therapy clinical trials. A previous clini- side effect is a temporary increase in certain liver enzymes. cal trial of SB-525 called AFFINE was placed on clinical hold in November after abnormally high FVIII levels were




Novo Nordisk: Lunch Sponsor


Sanofi Genzyme: Premier Exhibitor Octapharma: Premiere Exhibitor BDRN: Premiere Exhibitor

CSL Behring Genentech

Takeda: Premier Exhibitor Accredo: Premier Exhibitor

Cottrill’s Phramacy


Pfizer: Virtual Sponsor Optum Rx: Gold Sponsor Medexus Pharma: Exhibitor Genentech: Exhibitor

Hemophilia Alliance ◊ Takeda BioMarin ◊ Pfizer ◊ Sanofi Ascella ◊ Health ◊ Bayer ◊ Grifols ◊ Optum

BioMatrix: Exhibitor Bayer: Exhibitor CSL Behring: Exhibitor




Save the Date!

Contact Us BDANENY Office 333 Broadway Suite 320

April 9

Troy, NY 12180 (518) 729-3577

April 19 To promote awareness and support for... BDANENY Board of Directors and Staff President…………………….…………..…Bruce Weiner Treasurer………………………………….....Cindi Keeler Board Member…………………..Juliemar Abreu Boria Board Member………..……...…....…Matthew Delaney

Board Member……………………...…....…Dave Huskie Board Member…….………….…..Christina Mastrianni Executive Director……………..…….….…..Beth Mahar Operations Manager………………....Patricia Bengraff Administrative Assistant……….……......Melissa Deeb

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