Sprin 2023 Newsletter

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

Spring 2023

Albany Days

March 18~March 20 March is advocacy month! The month of March sees multi- R.E.A.D.Y. Program The first part of our Albany Days was focused on the ple advocacy groups descend on our capital city and R.E.A.D.Y Program; the Regional Education Advocacy BDANENY is proud to be among those groups, bringing Development for Youth (READY) Teen Retreat is a free our message to legislators, staffers and regulators. This leadership program that teaches teens the ins and outs of year, we were happy to gather in person again with advoadvocacy and gives participants the opportunity to use cates from across the state as our fellow New York State these skills at the State level. Teens learned how to tell Bleeding Disorders Coalition members met for Albany their story and how to utilize the advocacy skills they Days 2023. Held at the Albany Hilton, one block away would find necessary when speaking to legislators at Albafrom the State Capital building, the two-day training continued the tradition ny Days. The retreat was filled with interactive games and of providing an an- sessions, including the creation of a laminated picture nual opportunity for board showcasing the unique journey of each family. the bleeding disorAdvocacy, Skills and Knowledge (ASK) Training ders community Sunday afternoon’s training focused on two key issues the from all parts of coalition is highlighting for this current legislative session: New York to bestep theray and prior authorization reform. Attendees come more educated learned about the background of these issues, why they are in critical issues that important to the bleeding disorders community, and the bill affect access to that we support which could provide for relief. The proquality care and to We also took our message to Washington! gram also provided a blueprint for meetings with legislalearn effective grasstors, including how to frame your unique family experience roots advocacy techniques. Prior to their legislative meetto illustrate the need for remedial legislation. The program ings, attendees received advocacy training to become more concluded with a dinner during which attendees were procomfortable and proficient when speaking about public vided with updates on advocacy at the national level. health issues. Throughout the event, families learned more Legislative Visits about the legislative process and met other families naviThe final day of Albany Days saw advocates in the Legislagating through the same health care issues. tive Office Building raising public awareness around bleed-

In This Issue • Snowflake Festival • World Hemophilia Day

• Legislative & Regulatory Up-

date • Industry Update

• Medicaid Unwinding • Scholarship Applications • Upcoming Events

ALBANY DAYS cont. ing disorders. We were able to secure a number of in-person visits with legislators and/or their staff. Lawmakers were open to our requests, with several adding their names as co-sponsors on the spot. Additionally, we were able to engage many individuals passing by our display table – with chocolate! – thereby enhancing the general public’s understanding of the challenges of living with a bleeding disorder.

We were fortunate in that the Senate has already passed the primary bill for which we were advocating, but our visits served to set the stage for future action on prior authorization reform.

Finally, these visits serve to cement the bonds between constituents and elected representative and to introduce those new legislators to the concerns of the bleeding disorders community.

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A Word from the Executive Director Spring is a time for renewal, growth, and expan- hearts to what we can accomplish with a little sion; we feel it in the air. Even though we can drive and determination. do this at any time during the year, it feels just Last year, New York State became one of a right to do it during Spring. We seem to have handful of vanguard states to pass legislation that extra energy and focus to get physically accounting all co-pays toward an individual’s out tive, take action, and create change. of pocket deductible. This is what we can acSpring is a great time to envision endless possi- complish if we look at each new season as an opportunity and a chance to educate and enrich bilities, and to use our creative gifts to reach new levels of achievement, wellness, happiness those around us. and success.

Spring is a good time to let go of the old and make room for the new. This can apply to the traditional spring cleaning but most importantly we need to apply it to letting go of old grudges, resentments, and anger that holds us back from enjoying inner peace and more happiness in our lives. When we hold on to the old we keep ruminating and re-visiting old wounds and they keeps us stuck. And sometimes we don’t even know it. When we choose to let go of the old we make room for the new and open the door to endless possibilities that are waiting for us. We all have struggles- it’s called life. While it is true that some challenges are harder than others, this only means that we have to work harder at letting go. We are all being guided through the learning process of figuring out life by overcoming its challenges; this is part of our earthly journey.

So in this season of renewal and rebirth, let’s work on letting go of the past fears and pains that hold us back and open our minds and

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Our Advocacy Issues Increase Step Therapy Guardrails

changes their formulary · Plans/PBMs must accept letters of medical necessity from a prescribing doctor The issue · Step therapy overrides granted by a plan/PBM ust be Step therapy is a utilization management practice whereby honored for 12 months health plans or Pharmacy Benefit Managers mandate pa· Failure to follow these guardrails constitutes grounds for tients try less expensive medication options before “stepping up” to more expensive medications, if necessary. the patient to receive an automatic override of a step therapy ruling.

Step therapy is also known as “fail first” because patients must fail on the mandated medication before the patient S.1267/A.901 balances cost concerns with medical princican perceive the doctor-prescribed medication. For people ples. Health plans and PBMs will still be able to apply step with bleeding disorders, failing on a medication could lead therapy and patients will be protected from unsound step to spontaneous and uncontrolled bleeding into joints, mus- therapy policies. S.1267 passed the Senate in 2022. It cles ot other organs, potentially causing permanent dampassed again in 2023 by a vote of 59-3. We are now workage, chronic pain, or even life-threatening emergencies. ing with the Assembly to secure more co-sponsors and passage this session.

A 2021 Tufts University study of the 17 largest commercial payers found 38.9% of policies had step therapy, 55.6% of Prior Authorization Reforms step therapy rules were more strict than clinical guidelines, and 33.4% of the time patients were required to step up The issue: through more than one medication. Step therapy protocols Prior Authorization is a practice by payers (health insurwere found to be included in 77.3% of health insurance ance plans and Pharmacy Benefit Managers – PBMs) that policies for people with complex, chronic diseases like requires a patient, doctor, or pharmacy to seek special permultiple sclerosis and Hepatitus C. mission from the payer to provide a patient with a treatment already covered by the patient’s health plan. Prior What is being done: authorization rules are set by the payer and differ for each S,1267/A.901 “ An act to amend the insurance law and the payer. The rules may require a patient, doctor, or pharmacy public health law, in relation to requiring a utilization reto submit additional information and receive approval beview agent to follow certain rules when establishing a step fore providing the medication or treatment. The original therapy protocol,” has been passed in the Senate but repurpose of prior authorization was to limit unnecessary and mains in committee in the Assembly. potentially fraudulent insurance claims.

Under S.1267/A.901: · Patients can’t be required to try an off-label medication

Differing prior authorization rules, the extra information required, and the extra time needed to meet these require· Patients can’t be required to try and fail on more than one ments and receive authorization can cause several probmedication lems. Patients, doctors, and pharmacists may have trouble · Patients can’t be required to try a medication for more understanding the extra requirements. The extra work involved increases costs to payers, doctors, and pharmacists. than 30 days · Patients can’t be required to try and fail on a medication It can also delay patients from receiving necessary care, if they were already covered for a doctor-prescribed medi- which can harm the patient’s health and lead to complications (which require additional treatment). cation in the previous year · Patients can’t be required to try a medication if they already failed on it (even on a different plan) · Patients can’t be forced to try other medications if they’re already taking a covered medication and a plan/PBM 4

A growing problem for people with chronic medical conditions is the requirement to get prior authorization for every medication refill. Many people with bleeding disorders regularly use clotting medication to control bleeding and may

Our Advocacy Issues, cont. need monthly refills. Because there is no cure for bleeding disorders they may need a prior authorization every month for medication the health plan already promised to cover.

Delays and denials mean the patient can go without factor, risking serious bleeding and lasting complications.

er than the rate of inflation, and no one understands how the prices of drugs keep going up and up. There is no real mechanism on how high they can rise. Additionally, prices can randomly rise during the middle of the year, after an individual has selected their health insurance plan.

What can be done: The Governor has proposed “The Prescription Drug Price and Supply Chain Transparency Act of 2023” in the Health and Mental Hygiene Article VII as part of the State Budget.

This legislation would require prescription drug manufacturers to report price increases in advance, including the reasons for the increase and a requirement to provide consumers advance notice of proposed prescription drug price Unfortunately, this bill was not passed by the Legislature. hikes, and require disclosure of or a ban on ‘pay for delay’ We continue to work with the Legislature, as well as with deals by which brand name drug makers pay generic manuother stakeholders to craft and introduce a bill which would facturers to keep their cheaper generic versions off the marhelp to ensure that prior authorization requirements do not ket for nearly 17 months on average. The Governor’s plan would also empower the New York State Department of negatively impact patient care.

Financial Services to investigate drug manufacturers’ assertions as to reasons for price increases, building upon the Prescription Drug Pricing Transparency agency’s existing authority to investigate certain price The issue: spikes.

The cost of prescription drugs have always increased highWhat is being done: Last year, A.7129 (Gottfried) / S.6435a (Breslin) created common sense guidelines for the use of prior authorization by health plans and PBM’s. The guidelines were designed to allow for prior authorization to be used in a way which didn’t delay treatment for patients or add unnecessarily to the burden on medical staff.

National Advocacy Issues HELP Copays Act Although New York State has passed legislation ensuring that all health insurance programs overseen by the State mandate that all co-pay assistance be counted toward an individual’s out of pocket costs, this does not impact employer-funded health insurance (ERISA) plans. In order to extend these proctections to ERISA plan members, it is necessary to pass legislation at the federal level.

counts toward a patient’s cost-sharing limits.

In the House, HR 830 was introduced by Reps. Carter (RGA) and Barragan (D-CA). The Senate has yet to introduce a companion bill.

Federal Programs

We support funding for the following programs: The Help Lower Patient Copays Act (HELP Copays Act) is · The National Heart, Lung and Blood Institute at the National Institutes of Health (NIH). a bi-partisan, two-part solution that: Clarifies the ACA definition of cost-sharing to ensure pay- · The Maternal and Child Health Bureau at the Health Rements made “by or on behalf of” patients count toward sources and Services Administration (HRSA), which protheir deductible and out of pocket maximum. vides funding to HTCs and allows them to participate in the Closes the Essential Health Benefits (EHB) loophole to 340B program. ensure that any item or service covered by a health plan is · The Division of Blood Disorders at the Centers for Dispart of the EHB package so that all related cost-sharing ease Control and Prevention (CDC).

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Important Medicaid Information Medicaid “Unwinding” Is Coming Soon Do you or a family member currently have health coverage through Medicaid, the Essential Plan or the Children’s Health Insurance Program (CHIP)?

If so, you may soon need to take steps to find out if you can continue your coverage. Soon, states will resume Medicaid and CHIP eligibility reviews. This means some people with Medicaid or CHIP could be disenrolled from those programs.

However, they may be eligible to buy a health plan through the Health Insurance Marketplace®, and get help paying for it.

Here are some things you can do to prepare: -Make sure your address is up to date -Make sure your state has your current mailing address, phone number, email, or other contact information. This way, they’ll be able to contact you about your Medicaid or CHIP coverage.

-Check your mail New York State will mail you a letter about your Medicaid or CHIP coverage. This letter will also let you know if you need to complete a renewal form to see if you still qualify for Medicaid or CHIP. If you get a renewal form, fill it out and return it to the state right away. This may help you avoid a gap in your coverage.

What if you don’t qualify for Medicaid or CHIP? If you or a family member no longer qualify for Medicaid or CHIP, you may be able to buy a health plan through the Health Insurance Marketplace®. Marketplace plans are: - Affordable. 4 out of 5 enrollees can find plans that cost less than $10 a month.

- Comprehensive. Most plans cover things like prescription drugs, doctor visits, urgent care, hospital visits, and more.

LADIES CRAFT NIGHT Thank you to Cottrill’s and CSL Behring for a fun-filled evening!

INDUSTRY UPDATE BioMarin - ROCTAVIAN™ (valoctocogene roxaparvovec) ROCTAVIAN™ (valoctocogene roxaparvovec) is gene therapy for adults with severe hemophilia A. An application for licensing has been before the FDA for some time.

Approximately a year and a half ago, the FDA put a pause on review, citing the need for more data. Consequently, BioMarin submitted a three-year data analysis from the ongoing Phase 3 GENEr8-1 study. Earlier this month, BioMarin announced that the FDA has set a new PDUFA Target Action Date of June 30, 2023. PDUFA dates are deadlines for the FDA to review new drugs. The FDA is normally given 10 months to review new drugs. If a drug is selected for priority review, the FDA is allotted 6 months to review the drug. The company received Breakthrough Therapy designation for valoctocogene roxaparvovec in 2017.

spective, non-controlled, international, multicenter phase 3 trial that investigated the efficacy and safety of wilate® prophylaxis over 12 months in people of age 6 and older with severe VWD of any type except type 2N. The clinical trial's primary purpose was to investigate whether prophylaxis with wilate® lowered the mean total annualized bleeding rate (ABR) compared to the six months of ondemand treatment by more than 50%. Secondary goals were to measure spontaneous ABR and treatment-emergent adverse events.

Researchers reported an 84% reduction in ABR compared with on-demand treatment during a prior study. The median spontaneous ABR decreased by 95%. No serious drugrelated adverse events or thrombotic events occurred during prophylaxis with wilate®.

Sanofi

BioMarin has been aggressively marketing in the EU, espe- On February 23, 2023, the U.S. Food and Drug Admincially to health insurance groups in Germany, and expects istration (FDA) approved ALTUVIIIO™ [Antihemophilic to sign outcomes-based agreements (OBAs) providing for Factor (Recombinant), Fc-VWF-XTEN Fusion Proteincompanion diagnostic testing and reimbursement of ROC- ehtl], previously referred to as efanesoctocog alfa, a first-in TAVIAN with German health insurance groups in the com- -class, high-sustained factor VIII replacement therapy. ALing weeks. BioMarin's continued progress of the European TUVIIIO is indicated for routine prophylaxis and ondemand treatment to control bleeding episodes, as well as launch of ROCTAVIAN include plans to hold meetings with authorities in France and the submission of the reim- perioperative management (surgery) for adults and children with hemophilia A. bursement dossier in Italy.

ALTUVIIIO is a novel recombinant factor VIII therapy that is designed to extend protection from bleeds with once Octapharma USA, Inc. has submitted a Biologics License -weekly prophylactic dosing for adults and children with Application Supplement to the FDA to expand the approval hemophilia A. ALTUVIIIO has a 3 to 4 fold longer halfof wilate®, von Willebrand Factor/Coagulation Factor VIII life relative to standard and extended half-life factor VIII Complex, to include routine prophylaxis to reduce the fre- products. In contrast to other FVIII replacement therapies, quency of bleeding episodes in children and adults with it is independent of von Willebrand factor, which imposes a any type of von Willebrand disease. half-life limitation on earlier generation factor VIII therapies.

Wilate® is currently indicated in children and adults with VWD for on-demand treatment and control of bleeding Hemab Therapeutics episodes and perioperative management of bleeding. Additionally, wilate® is indicated in adolescents and adults with Hemab Therapeutics, a clinical-stage biotechnology comhemophilia A for routine prophylaxis to reduce the frepany, announced in January the first patient has been dosed quency of bleeding episodes and on-demand treatment and in a Phase 1/2 study evaluating HMB-001 for the treatment control of bleeding episodes. of the severe bleeding disorder Glanzmann Thrombasthenia. HMB-001 is a bispecific antibody that binds, stabilizOctapharma conducted a study designated WIL-31, a pro- es, and recruits endogenous factor VIIa (FVIIa) to the site OctaPharma

INDUSTRY UPDATE, cont. of vascular injury to overcome the body's inability to form healthy clots.

The Phase 1/2 first-in-human open-label study in patients with Glanzmann was designed to evaluate HMB-001 for safety, tolerability, biomarkers such as FVII levels and bleeding time before and after HMB-001, and efficacy based on assessment of changes in bleeding frequency.

Facilitated in collaboration with the UK-based clinical research organization Richmond Pharmacology, the trial is expected to expand into the US and other EU countries with initial data are expected in the second half of 2023.

Registry is supported by Biomarin, CSL Behring, Pfizer, Spark (Founding visionary partners) and Takeda (Collaborating partner).

Gene editing

Hemab is a clinical-stage biotech company developing next generation therapeutics for serious, underserved bleeding and thrombosis disorders. It is based in Denmark and the US and backed by Novo Holdings, RA Capital, and HealthCap.

Scientists gathered at the Francis Crick Institute in London, for the Third International Summit on Human Genome Editing earlier this month. Five years after the last summit, CRISPR technology has continued to mature. The advent of CRISPR gene-editing technology, short for “Clustered Regularly Interspaced Short Palindromic Repeats”, has raised a number of ethical considerations, including the delivery of equitable access to genome editing therapies, ongoing research to optimize delivery systems for genome editing apparatus and delivery of measures to foster discussions regarding regulation, governance, public and patient engagement.

Gene Therapy Registry

Many new advances in genome editing techniques were presented.

The World Federation of Hemophilia has developed a registry to monitor the long-term safety and efficacy of gene therapy for people with hemophilia: the WFH Gene Therapy Registry (GTR The launch of the WFH GTR coincides with the approval of gene therapy for hemophilia by both the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

American chemist and biologist David Liu reported on findings to use “prime editing” to treat genetic conditions such as Huntington’s disease and Friedreich’s ataxia. Unlike CRISPR, which makes a double stranded cut in the DNA, prime editing induces a single stranded cut. This makes it more versatile and precise for targeted deletion and insertion of genetic sequences.

This worldwide endeavor aims to collect important data on all patients who receive gene therapy, whether through a clinical trial or through a post-marketed product. Clinical data from around the world will be combined in this registry and used to answer critical questions on the long-term safety and efficacy of gene therapy in hemophilia.

A somatic genome editing treatment for sickle cell disease is set to obtain regulatory approval in the US later this year. “Somatic” genome editing (which makes changes that are not heritable) is different to germline and heritable genome editing (which makes heritable changes). There were also reports of research using CRISPR technology to treat diseases including Duchenne muscular dystrophy, cancer, HIV/AIDS, heart and muscle disease and inborn errors of immunity.

The WFH GTR was developed in collaboration with the International Society on Thrombosis and Haemostasis (ISTH), the European Haemophilia Consortium (EHC), the US National Hemophilia Foundation (NHF), the American In our area, Regeneron, with headquarters in Tarrytown and a facility in North Greenbush, is working on gene editThrombosis and Hemostasis Network (ATHN), Patienting for hemophilia.

Reported Outcomes Burdens and Experiences (PROBE) study group, manufacturers, and experts in the field of gene therapy. The WFH also partners with the United BioSource Corporation and Lifelink Systems for the technological development of the WFH GTR. The Gene Therapy

It takes 1200 plasma donations to treat one patient with hemophilia for one year.

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BDANENY SCHOLARSHIP 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 May 1, 2023.

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

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BDANENY Board of Directors and Staff President …………………………………………………………………………………..………….Bruce Weiner Vice President:………………………………………………………………………………………...Chris Huskie Treasurer:…...…………………………………………………………………………………………..Cindi Keeler Secretary:……………………………………………………………………………………. Christina Mastrianni Member:…………………………………………………………………………………….. Juliemar Abreu Boria Member:……………………………………………………………………………………………….. Matt Delaney

Member:…………………………………………………………………………………………….. Linda Peacock Member: ……………………………………………………………………………………………...Jessica Smith Advisor:……………………………………………………………………………………………….. Dave Huskie Executive Director:…..………………………………………………………………………………. Beth Mahar Operations Manager:………………………………………………………………………….. Patricia Bengraff Social Media & Special Events:………………………………………………………………….. Melissa Deeb