March 2021 Newsletter

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March 2021

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

ALBANY DAYS 2021 BDANENY is joining the New York State Bleeding Disorders Advocacy Coalition for virtual Albany Days. This year, on March 21 – 23, advocates from all over New York State mobilize and advocate in support of several key bills. Part of Albany Days includes Advocacy Skills & Knowledge (ASK) training, where attendees become more comfortable with the issues and receive various training tools and resources to enable them to meet with their legislators or representatives to express their views. Participants will have the opportunity to become more informed on critical issues that affect continued access to quality care and learn effective, grassroots advocacy techniques. This program offers a unique experience for members and families of the bleeding disorders community to learn more about the legislative process, “virtually meet” with other families navigating through the same health care issues and even in this pandemic environment, Albany Days affords the opportunity to be better, together. One exciting component of this year’s Albany Days program is the virtual READY session, developed exclusively for teens. The virtual Regional Education Advocacy Development for Youth (READY) Teen Retreat is a free leadership program that teaches teens the ins and outs of advocacy and gives participants the opportunity to use these skills at the State level. Teens will learn how to tell their story and will have the opportunity to utilize their advocacy skills when they speak to legislators. The retreat will be filled with interactive games and sessions. This program is for ages 13 - 18 and is FREE to all attendees. You must live in New York State and either have a bleeding disorder, be a carrier, or be a family member of someone with a bleeding disorder to attend. There will be two major issues we will be addressing this year: prior authorization reform and access to patient assistance programs (sometimes called accumulator adjuster programs). See our platform on page 2.

In This Issue •

Advocacy Priorities

Legislative & Regulatory Update

Industry Update

Resources for Help

Upcoming Events

LEGISLATIVE PRIORITIES 2021 PRIOR AUTHORIZATION REFORM THE ISSUE: Prior Authorization is a practice by payers (health insurance plans and PBMs) in which a patient, doctor, or pharmacy must seek special permission from the payer to provide a patient with a medication or treatment already covered by the patient’s health plan. Prior authorization rules are set by the payer and differ for each payer. The rules may require a patient, doctor, or pharmacy to submit additional information and receive approval before providing the medication or treatment. The original purpose of prior authorization was to limit unnecessary and potentially fraudulent insurance claims.

• Make prior authorization based on medical science, not just cost Create common, easy to understand electronic prior authorization processing It shouldn’t be a burden to get medication or treatment already covered by a health plan. Imagine having to ask for special permission every time you need a refill of a medication you must use for life even though the medication is already covered by your insurance plan.

Differing prior authorization rules, the extra information required, and the extra time needed to meet these requirements and receive authorization can cause several problems. Patients, doctors, and pharmacists may have trouble understanding the extra requirements. The extra work involved increases costs to payers, doctors, and pharmacists. It can also delay patients from receiving necessary care, which can harm the patient’s health and lead to complications (which require additional treatment).

ACCESS TO PATIENT ASSISTANCE PROGRAMS THE ISSUE: Many health insurance plans require patients to pay money out of their own pockets when the patient uses certain health care services and medications. These out of pocket costs may include copays, co-insurances, and deductibles. The original purpose of these out of pocket charges was to make patients mindful of which services and medications they use in order to help control costs. Some third party groups such as pharmaceutical manufacturers and patient asA growing problem for people with chronic medical sistance organizations offer financial assistance to conditions is the requirement to get prior authoriza- help lower income patients pay for these out of pocket tion for every medication refill. Many people with costs. bleeding disorders regularly use clotting medication to control bleeding and may need monthly refills. Be- Because of a murky ruling by the federal CMS cause there is no cure for bleeding disorders they may (Centers for Medicaid & Medicare Services), some need a prior authorization every month for medication payers (health plans and PBMs) are not allowing pathe health plan already promised to cover. Delays and tients to use 3rd party assistance programs. In other denials mean the patient can go without factor, risking cases the payer will accept the assistance payment and serious bleeding and lasting complications. then bill the patient for the out of pocket costs on top of it. Lower income patients and those with serious OUR POSITION: NYSBDC recognizes the need to medical conditions struggle to pay for their health create smart and efficient prior authorization stand- care and other expenses such as food, rent, utilities, ards which improve patient access to quality, afforda- etc. Being able to use third party patient assistance ble care. We support legislation as a key step toward means these patients can get the care they need and achieving that goal. pay their other bills. Without assistance, some patients may be forced to delay or skip treatment, causing A/PROPOSED SOLUTION: Prior authorization worse medical problems. rules which delay necessary medications and treatments and add to provider or payer costs must be re- People with complicated medical conditions may need formed. multiple doctors to help manage their condition and • Make a prior authorization approval good for the take special medication for which there are no generic length of a prescription (1 year) rather than requiring alternatives. Getting the right care and medication isn’t a matter of choice, it’s what they have to do. Peoapproval for each refill • Limit the time needed for approvals; 72 hours for ple with bleeding disorders need regular comprehennormal requests, 24 hours for urgent situations, and no sive care at a bleeding disorder specialty treatment center. They may also need to take clotting medica wait for emergency situations 2

Cont. page 6

A Word from the Executive Director March is Bleeding Disorders Awareness Month In 2016, March was first declared Bleeding Disorders Awareness Month, to help raise critical awareness and funds to improve the lives of those diagnosed with a bleeding disorder. What originally started as the Red Tie Challenge has grown and evolved into a monthlong celebration of the bleeding disorders community. The Red Tie was introduced as the symbol for the bleeding disorders community in 2016, when NHF and the U.S. Department of Health and Human Services worked together to designate March as an official U.S. health observance month — Bleeding Disorders Awareness Month. The Red Tie reflects the fact the blood ties — embodied in the color red and the tie — are what bind the community together. Advocacy is a powerful and important catalyst for the change we want to see. We can work to improve the laws, policies and systems that affect the communities we live in.

community.) Tell Your Story: Personal anecdotes are the most effective way to show lawmakers how abstract policy impacts the daily lives of their constituents. Here are ways to make your story resonate; Don’t try to use fancy language. Your story is best when told in a conversational manner. Pretend you’re talking to a friend. Describe the challenge. Why is the situation particularly impactful? What are the factors that make it so? Why don’t existing safeguards work? Convey the struggle. Of course, you and your family want to do everything possible to live a full, healthy life; but what are the roadblocks you must overcome? Use vivid language and key details. Show resolution. Describe a key turning point; how the situation has been resolved or how it may be resolved. This is the point where you need to restate The Ask. Leave the Door Open: Give your contact information, then follow up with the staff in a day or so, in case they have any questions or (hopefully!) want to use you as a resource.

Grassroots advocacy is about finding strength in numbers — and harnessing that strength to influence public opinion and government action. The value of this form of advocacy is that it is driven by the people. It is grounded by the belief that people matter and that their collective voices are powerful in influencing elected officials and shaping policies at all levels of govern- Bottom line: YOU are the best person to tell YOUR ment. story. Our governmental representatives and aren’t able In recent years, the Internet and social media have to constantly craft solutions to issues affecting their transformed grassroots advocacy. Constituent commu- constituency if they aren't hearing from their own votnication with legislators is at record levels and all elect- ers. Your story is a powerful tool in shaping public poled officials use Facebook, Twitter and other social me- icy. Let us help you use it! dia platforms to dialogue with the public – it’s the way they raise funds for their re-election campaigns! So, if you can’t participate in Albany Days, social media will allow you to advocate at any time, from any place. Whether you are interacting in person or virtually, here are some tips to make the most of your effort: The ‘Ask’: Let your policymaker know why you are advocating and what you want from them. You want to be clear, concise and confident. Know Your Audience: Be familiar with whom you plan to speak. Consider the relevance to your policymaker’s district and how your story might reflect broader community issues. (For example, Prior Authorization issues impact a larger audience than the Bleeding Disorders


Do You Need Help? What’s standing between you and your career goals? Hemophilia Federation of America (HFA) is awarding Job Readiness Grants to help the bleeding disorders community pay for technical training and certification to support their career goals. Whether you want to secure a better paying position, find a new job that’s

easier on joints or transition to the career you’ve dreamed of, gaining a technical skill or certification may set you apart from other applicants. The grant may be used to cover the cost of technical training or certification up to $500 for a person with a bleeding disorder between the ages of 18 and 64. Job Readiness Grants could be used to support the applicant’s career goals, such as the careers in the sustainable fields of database management, graphic design, medical coding and more. See examples here and here.

About this Grant •

Applicant must be a person with a bleeding disorder between the ages of 18 to 64

seeking training and certification to aid in securing sustainable employment. •

Applicant must complete the Career Planning courses in HFA’s Learning Central by April

15 to be considered. Please register in Learning Central using the same name and email

provided in your Job Readiness Grant application.

Deadline to apply is April 15, 2021

Recipients will be notified in early June, and grants will be distributed by June 30. CLICK HERE TO APPLY For assistance, contact with the subject “Job Readiness Grant”.

Job Readiness grants are part of the Helping Forward program. Visit Helping Forward for additional resources on employment and financial planning for the bleeding disorders community. 4


Legislative & Regulatory Update

News from New York State Two new bills of interest to the bleeding disorders community have been introduce in the legislature: A.1741/S.5299 “Patient Assistance Bill” Requires insurance companies or pharmacy benefit managers to apply price reduction instruments for out-of-pocket expenses when calculating an insured individual's cost -sharing requirement. News from Washington: ACA litigation. On Feb. 10, the Biden Administration advised the Supreme Court that the U.S. Department of Justice is switching its position in California v. Texas, the high-stakes lawsuit over the constitutionality of the Affordable Care Act. The Biden Administration now sides with the parties defending the law in court. In separate ACA-related litigation, the federal government asked courts to pause cases over the validity of Trump Administration rules (i) expanding non-ACA compliant “association health plans” and (ii) rolling back the ACA’s nondiscrimination protections, so the Administration can re-evaluate its stance in those cases. Still more litigation news! A federal trial court dismissed a case filed by hospitals and pharmacies against drug manufacturers who withhold 340B discounts from contract pharmacies. Separately, the Supreme Court agreed on Feb. 22 to hear a challenge to the Trump Administration’s “public charge” rule, which allows the federal government to count legal immigrants’ use of public benefits (including Medicaid) as a negative factor when they apply for permanent U.S. residency, or seek to enter/re-enter the country. Some observers expect the Administration to withdraw the public charge rule before the case works its way through the Court, mooting the issue. Federal health nominees. The Senate began hearings on various Biden Administration nominees for health agency offices: Xavier Becerra, nominated to head the Department of Health and Human Services; Chiquita Brooks-LaSure, nominated to head CMS; Vivek Murthy, nominated to serve as Surgeon General (a role he filled in the Obama Administration); and Rachel Levine, nominated to serve as HHS Assistant Secretary. 6

S.5299 (no same as) “Pharmacy Access Bill” Relates to the purchase of prescription drugs by pharmacies; establishes the same reimbursement amount shall be provided to all pharmacies, i.e. the same benchmark index, including the same average wholesale price, maximum allowable cost and national prescription drug codes to reimburse all pharmacies participating in the insurance network regardless of whether a pharmacy is a mail order pharmacy or a non-mail order pharmacy. Legislative Priorities, cont.

tion. One dose of the specific clotting medication a patient must use can cost $2,000 to $10,000; and many patients can easily use $200,000 or more of clotting medication in one year.

OUR POSITION: NYSBDC recognizes the need for qualified patients to use 3rd party assistance programs to help defray out of pocket healthcare expenses. We support legislation as an important way to help patients get assistance. PROPOSED SOLUTION: People should be allowed to use 3rd party financial assistance programs to help defray out of pocket healthcare expenses, and payers shouldn’t be allowed to limit patients from receiving such help, or to pocket help meant for patients. Help for patients who need it should go to the patients. Imagine finding out you are qualified for financial help with your medical expenses and being told you are not allowed to use it.

Industry Update Stimate Update On July 21, 2020, Ferring Pharmaceuticals issued a voluntary recall of Stimate (desmopressin) nasal spray due to “superpotency” – in other words, amounts of desmopressin being higher than specified. Stimate is used to treat von Willebrand disease (VWD) and mild hemophilia A. It is licensed and distributed by CSL Behring in the US. Ferring has halted production of Stimate while investigating the root cause. Upon investigation, the manufacturer has determined that production facilities would need to be retooled in order to correct the problem. This has proved to be a larger project than initially anticipated. We have recently learned that Stimate may not be available for perhaps three years. At last month’s MASAC (the Medical and Scientific Advisory Council for the National Hemophilia Foundation) meeting, participants explored alternatives. Several factors make producing an equivalent a complicated proposition. Barriers include: the costs of raw material; the stability of raw material; no standard formula for compounding; and laws and regulations governing compounding and repackaging. Additionally, Stimate is not on the FDA’s drug shortage list; being listed would allow the FDA to pursue strategies to mitigate the shortage. Hemlibra Update Genentech, the maker of Hemlibra, has made several changes to the Warnings and Precautions (sections 5.1 and 5.2) and Clinical Pharmacology (section 12.3) sections of the label, as requested by the FDA. The Warnings and Precautions sections have been updated to remind healthcare providers to consider the long half-life of Hemlibra (26.9 days) when prescribing aPCC after Hemlibra has been discontinued. Such a long half life presents the possibility of an interaction for up to 6 months after the last dose. Additionally, the label has been updated to make clear to healthcare professionals that Hemlibra has not been studied in people with severe hepatic or renal impairment and congenital hemophilia A with and without factor VIII inhibitors. COVID-19 Vaccine and Individuals With Low Platelets The Platelet Disorder Support Association (PDSA) recently recorded a session on the new COVID-19 vaccines and immune thrombocytopenic purpura

(ITP), a disorder characterized by low platelet levels. Individuals affected by bleeding and platelet disorders continue to voice concerns and questions related to media reports of vaccine side effects, particularly those related to ITP. It should be noted that there have been an extremely small number of verified cases of severe ITP post vaccination, amongst the millions of individuals who have been immunized against COVID-19, however, the PDSA webinar answers many questions that concerned individuals may have. View the webinar for free here. BioMarin’s Update on Gene Therapy Last August, the FDA refused to approve BioMarin’s hemophilia A gene therapy valoctocogene roxaparvovec (Valrox) over concerns about the durability of the therapy after factor VIII levels seemed to fall off after 12 to 18 months, raising the possibility that patients might need to be re-dosed to maintain protection against bleeds. The FDA then said BioMarin must finish off phase 3 and then submit two-year follow-up safety and efficacy data on all study participants. The late-stage test was fully enrolled November 2019, but the last patient will not have completed two years of follow-up until November 2021. On March 8, 2021, BioMarin announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to Valrox. RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies that are intended to address an unmet medical need in patients with serious conditions. The RMAT designation is complementary to Breakthrough Therapy Designation, which BioMarin received in 2017, allowing early, close, and frequent interactions with the FDA. In addition to the RMAT Designation and Breakthrough Therapy Designation, BioMarin’s Valrox also has received orphan drug designation from the FDA and EMA for the treatment of severe hemophilia A. The Orphan Drug Designation program is intended to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. The bottom line: Although BioMarin’s investigational gene therapy for hemophilia A has not been approved for use, it is back in ongoing clinical trials evaluating its safety and efficacy, with positive feedback from the FDA. 7

Medical and Scientific Advisory Council Update NHF’s Medical and Scientific Advisory Council (MASAC) met on February 20 and developed several recommendations and guidelines for the treatment and management of bleeding disorders. This meeting took an in-depth look at von Willebrands and focused their attention on updating guidelines for establishing protocols for the correct diagnosis and treatment of inherited bleeding disorders for women. MASAC states, “Inherited bleeding disorders are under-recognized as causes of bleeding in girls and women. …Establishing the correct diagnosis of inherited bleeding disorders for women has important implications for management of bleeding, recommendations for invasive procedures, pregnancy and delivery management, and family planning and testing.” The documents developed include: MASAC Document 266: Recommendations regarding the treatment of von Willebrand disease MASAC Document 265: Guidelines for pregnancy and perinatal management of women with inherited bleeding disorders and carriers of Hemophilia A or B MASAC Document 264: Recommendations regarding diagnosis and management of inherited bleeding disorders in girls and women with personal and family history of bleeding The Council also discussed the pending new von Willebrand disease ICD-10 codes. This revision would potentially increase the diagnosis code from one to ten separate codes. The effective date of the new codes is October 1, 2022.

SAVE THE DATES! Upcoming Events - Mark your calendar. Check in on our Facebook or Website for details.

MARCH 2021 Tuesday Talks 3/23/21

Journey to Jivi


Wednesday Meditation and Mindfulness

APRIL 2021 Tuesday Talks 4/13/21

Von Willebrand’s


Jewelry Making

MAY 2021 Tuesday Talks 5/11/21


AUGUST 2021 8/26/21 – 8/28/21 NHF Virtual Conference

SEPTEMBER 2021 Saturday

2021 UNITE Walk


Gavin Park, Wilton NY

NOVEMBER 2021 Saturday

Annual Meeting


location TBD

Talking Points for Advocacy Bleeding Disorders Concerns About Affordability

chronic conditions need co-pay assistance programs to help them afford their high OOP costs.

People with bleeding disorders must have access to comprehensive health insurance that covers their lifesaving treatments. And expert care provided at hemophilia treatment centers (HTCs).

An increasing number of private health insurance plans are implementing co-pay accumulator adjustor programs that disallow co-pay assistance from counting toward a patient’s OOP maximum.

Annual treatment costs exceed $350,000 per year for a person with severe hemophilia. Therefore, people with bleeding disorders hit their annual out-of-pocket (OOP) maximum each year. For 2021, the OOP limit is $8,550 for an individual and $17,100 for a family.

When co-pay assistance is not allowed, many patients cannot afford their treatment and stop taking them or reduce the prescribed dosage. This often leads to complications and has unintended consequences (i.e. Increased ER visits, joint bleeds/damage, and missed days from work/school) that harm patients and increase overall costs.

Thus many community members worry about: Having access to and affording comprehensive insurance coverage; - Paying their OOP costs for treatment; - Hitting their OOP limit, often in the first month; - Needing financial assistance to afford their OOP costs.

What are Copay Accumulator Adjustor Programs and How Do They Impact Patient Access? Many people with bleeding disorders and other

Policies to Improve Access to Treatment and Prohibit Accumulator Adjustor Programs Plans should be required to count all co-pays (regardless of who pays) toward a person’s OOP maximum. This can be done via regulation by CMS at the federal level and by legislation at the state level.

Blood of the Paladin: A Graphic Novel Based on Jonathan Hill’s memoir, “Blood Brothers: A Memoir from the Lost Generation of Hemophiliacs” this new graphic novel showcases the triumphs and struggles of a young boy living with hemophilia and how Dungeons and Dragons provided a surprising source of inspiration and support through his journey. In 1971, at age 2, Jonathan was diagnosed with severe hemophilia A. As a young man, he faced “The Three H’s” that affected so many members of the bleeding disorders community in the 1980s and 1990s: hemophilia, HIV and hepatitis C. Blood of the Paladin is based on real events in Jonathan’s life. The graphic novel explores how he learned to understand, accept, manage, and finally embrace his bleeding disorder and the other medical challenges he encountered on his journey. The powerful story showcases the important role his family, friends, and doctors played in helping him become his own best medical advocate and champion. Two artists were brought on to illuminate the story of how Dungeons and Dragons, the iconic fantasy role-playing game, provided an escape from the real world, helping Jonathan learn to process life and death situations both real and imagined.

The novel was produced by BelieveLimited, and made possible by BioMarin. The first edition was launched through NHF’s HemAware magazine and is available for direct order or download. A podcast and audiobook will be available this fall.

St Patrick's Day is celebrated in countries with people of Irish descent. According to the US Census, 32 million US residents claimed Irish ancestry in 2018. This is more than six times the population of Ireland


color of St. Patrick’s Day was originally blue. Wearing green has become a staple of St. Patrick’s Day, but the holiday was originally associated with the color blue. It’s thought that the shift to green happened because of Ireland’s nickname “The Emerald Isle,” the green in the Irish flag and the shamrock, or clover. Green ribbons and shamrocks were worn as early as the 17th century. -Shamrocks are the national flower/emblem of Ireland. Legend says that each leaf of the clover has a meaning: Hope, Faith, Love and Luck. A shamrock is not a four leaf clover, contrary to popular belief. Your odds of finding a four-leaf clover are about 1 in 10,000. -Corned Beef and Cabbage Was an American Innovation. - Pinching The tradition in America is to pinch anyone who isn't wearing green on St. Patrick's Day. Legend has it that wearing green makes you invisible to leprechauns, who pinch anyone they see.

2021 UNITE Walk—Save the Date! Get ready to lace up your shoes...we’re walking to UNITE for Bleeding Disorders September 25, 2021

Where? Gavin Park, Wilton NY Stay tunes for more details!


Contact Us BDANENY Office 333 Broadway Suite 320 Troy, NY 12180 (518) 729-3577

Colorful Stories


Hosted by Linda Peacock, the My Journey to Jivi program brings the community together to hear inspiring firsthand stories of the defining moments and milestones that have shaped journeys from all walks of life.

Fresh Perspectives.

Each My Journey to Jivi special event features members of the community sharing stories about their life experiences and transition to Jivi.

Many Voices, One Community.

We all are on our own journey – our own path that we forge when we live with hemophilia A. Some journeys may be similar, while others are remarkably different. Here’s a chance to hear fellow patients and caregivers alike, who share their own journey, their own unique story. Join us on March 23 *** Register here

BDANENY Board of Directors and Staff President……………………….. Bruce Weiner Secretary……………………..…... Don McKay Treasurer…………………………. Cindi Keeler Board Member…………………. Bill Brownell

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

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