US Hereditary Angioedema Association


IN THIS ISSUE
Protecting Financial Assistance 2023 US HAEA National Summit 2023 Legislative Update and Welcome to Congress Pam King HAEA Scholarship Support Groups Open Enrollment Season	HAEA Community Blog: Brandie HAE Speaks Podcast #BeyondHAE Youth Podcast Clinical Trial Updates HAEA Events Calendar Social Media Snapshots

Dear HAEA Friends,

As highlighted in a previous communication, we are facing unfortunate and unprecedented circumstances that threaten access to HAE medicine for some members of our community. The situation is driven by sudden changes in the availability of charitable financial assistance – until now, an uninterrupted resource for helping HAEA friends afford their medicine and insurance premiums for the past 15 years. These changes are due to (1) Accessia Health (formerly PSI) dramatically decreasing the amount of financial assistance they offer, and (2) The Assistance Fund significantly reducing the number of people with HAE that they support.

The HAEA has vigorously engaged in direct and urgent communication with these charitable organizations to understand the reason for the sudden changes in assistance that threaten access to life-saving therapies. Despite our best efforts, the only information we have been able to obtain was included in one of the letters sent to our community that stated “…patients seeking assistance have increased significantly…”

As your advocates, fellow patients, and caregivers, we are gravely concerned when an HAEA friend loses access to their medicine. The HAEA Health Team has contacted a variety of charitable organizations with the hope of finding alternative sources of financial assistance for our community. At this time, however, none of the charities have resources available for people with HAE.

We do have some important information and suggestions based on the work we have performed to date.

The most immediate threat appears to be the loss of charitable financial assistance for copays. It is very important to note that the rules governing copays for HAE medicines are very different for people who have (1) commercial, employer-based insurance (including the Affordable Care Act Exchange plans), versus (2) public programs such as Medicare and Medicaid. Below, we address each situation separately.

Commercial / Employer-Based Insurance Plans

If you can no longer afford your copay due to changes in charitable assistance provided by Accessia Health and/or The Assistance Fund, you are eligible for the manufacturer’s copay assistance program. We recommend you contact the manufacturer of the product you have been prescribed through their hub (contact information provided below) and request to be part of their copay assistance program.

BioCryst - Empower (866) 536-7693
CSL Behring - Haegarda Connect (844) 423-4273
Pharming - Ruconest Solutions (855) 613-4423
Takeda - One Path (866) 888-0660

Public Insurance Programs -- Medicare and Medicaid

Unfortunately, the situation is very different for HAEA friends that are insured through Medicare or Medicaid because federal law prohibits manufacturers from providing you with copay assistance. Financial assistance for HAEA community members on public insurance programs can only be provided by charitable organizations.

Below are some steps that can be taken by our fellow HAEA friends on Medicare/Medicaid.

Some pharmaceutical companies have programs available to provide medicines when one of their patients encounters a circumstance when therapy can no longer be afforded. Publicly available information doesn’t indicate which, if any, manufacturers of HAE therapies offer this form of assistance. It would appear that HAEA friends on Medicare and Medicaid who can no longer pay for their therapy would be eligible for this type of manufacturer program because it is a donation and not a copay assistance program. We recommend you call the manufacturer’s hub (see list provided above) and ask if the Company will help when THEIR patients, due to circumstances beyond anyone’s control, can no longer afford medicine.

Clinical Trials can offer free access to HAE medicines being studied until they are approved and commercially available. Please contact our HAE Health Advocates at (877) 839-4232 to learn more about one of the 3 clinical trials taking place. We can provide a referral to whatever trial you choose.
- The Ionis OASIS-HAE Plus trial is a phase 3, open-label (medicine is provided, there is no placebo) study testing the safety and effectiveness of donidalorsen, the company’s preventive HAE therapy that is delivered by subcutaneous injection. Trial participants must be 12 years of age and older. Data from clinical trials already completed showed that patients given donidalorsen once every 4 weeks had a mean reduction in attack rate of 98.3% after taking a second dose. No serious adverse events were reported and there were no abnormalities observed in laboratory measurements.
- The KalVista Konfident trial is a Phase 3 placebo controlled clinical trial, evaluating the safety and efficacy of two different doses of sebetralstat as an on-demand oral tablet for HAE attacks. The trial is open to adults and adolescents 12 years of age and older who have had at least 2 HAE attacks in the previous 3 months. Data from clinical trials already completed show that when compared to placebo, attacks treated with sebetralstat achieved symptom relief more quickly, and significantly reduced time to onset of symptom relief. There were no serious adverse events reported in trials and no patients withdrew due to adverse events.
- The BioMarin HAERMONY₁ study is a phase 1 / 2 trial testing an investigational gene therapy called BMN 331 to improve HAE symptoms. To qualify for the HAERMONY₁ study, participants must be 18 years of age or older; diagnosed with HAE due to C1-INH deficiency; have 6 months of a continuous prophylaxis or on-demand treatment regimen (prior to study enrollment); and a documented attack frequency of at least 1 attack per month on average.
If you are receiving Social Security you can register with the ‘Extra Help’ program HERE. According to the eligibility requirements, you can complete an application for ‘Extra Help’ if, “Your combined savings, investments, and real estate are not worth more than $33,240 and you are married and living with your spouse, or $16,660 if you are not currently married or not living with your spouse.”

Make sure you are on the waitlist for both Accessia Health and The Assistance Fund. Although these organizations are not accepting new applications at this time, you will be on the waitlist should additional funds become available, and they will be able to contact you directly.
- Accessia Health - (800) 366-7741
- The Assistance Fund - (855) 845-3663

Finally, your health is of the utmost importance. Please let your HAE physician know about the situation you are facing, especially if you are not able to access your medication(s). It is important you have a copy of your current treatment plan and a letter from your physician in case of an emergency.

The HAEA will keep in touch as we continue working feverishly on your behalf to resolve this truly awful situation. Please do not hesitate to contact our Health Team at (877) 839-4232 should you have any questions.

Warm regards,

Tony Castaldo
US HAEA President & CEO

APPLY FOR A 2023 US HAEA NATIONAL SUMMIT GRANT BY FEBRUARY 28TH!

As announced, the 2023 US HAEA National Summit will be held in Orlando, Florida, from July 20 - 23, 2023.

If you wish to attend the 2023 US HAEA National Patient Summit – Living Beyond Boundaries, you can apply for an HAEA Summit Conference Grant.

Grants will be awarded through a lottery process. HAEA Summit Conference Grants are available for hotel accommodation, air travel and/or ground transportation. The deadline for grant applications is Tuesday, February 28th. The HAEA will notify grant recipients by Tuesday, March 14th. If there are grants still available after this date, we will announce a second drawing.

Conference grants are available to cover travel and/or hotel expenses. A limited number of grants will be awarded through a lottery process that closes on February 28th.

NOTE: To receive a grant, you must agree to attend all SUMMIT sessions. Ushers at the SUMMIT will scan badges before each session to record attendance.

2023 Legislative Update and Welcome to Congress Webinar

Join us on Thursday, March 16th, at 7:00 PM ET / 4:00 PM PT for the Welcome to Congress Webinar! Learn about this year’s congressional agenda and how it could advance the HAEA legislative priorities.

This webinar will cover:

Congress's new priorities,
Issues that will impact the HAE community in 2023, and
What you can do to participate in the HAEA volunteer grassroots advocacy program.

You don’t want to miss this highly informative webinar! Register today to secure your spot!

Fall 2022 Scholarship Applications are now open!

Are you planning to attend a college or university in the Fall of 2023? The HAEA helps students with a confirmed HAE diagnosis pursue their academic goals by reducing the financial burden associated with higher education. Applications for the Pam King HAEA Scholarship Program are open twice a year. We invite interested individuals to take advantage of this opportunity, and complete your application before March 31st.

Apply HERE!

February HAE Heroes Connecting - Rare Disease Day

Tell your rare story to raise awareness on February 28th when we celebrate Rare Disease Day! In this month’s HAE Heroes Connecting support group, we will focus on ways to share our stories on Rare Disease Day.

HAE Heroes Connecting support groups are held virtually via Zoom. If you are unable to join us this month, we invite you to next month’s session when we will talk about patient safety.

These group meetings offer opportunities for HAE community members to share their experiences and provide support to others. Interested in joining one of these monthly support groups? Contact the HAE Health Team for more information by calling 866-798-5598 or emailing an advocate at troyce@haea.org and adinaramsey@haea.org.

Only One Month Left to Enroll In A Medicare Advantage Plan!

We encourage you to take some time to review your options and compare them to determine which one is most attractive to you. The HAEA Health Team is available to help you navigate the selection process and find the best option available to you.

Medicare Advantage Open Enrollment Period: January 1st – March 31st each year, if you’re enrolled in a Medicare Advantage Plan, you can switch to a different Medicare Advantage Plan or switch to Original Medicare (and join a separate Medicare drug plan) once during this time. Note: You can only switch plans once during this period.

Employer sponsored plans: Each employer sets their enrollment dates so it is important to review and research the plans offered with your Human Resources department to ensure the plan you choose offers the best coverage for you.

Please reach out to your HAEA Health Team for assistance in your insurance review, research, and selection process.

The HAEA Health Team is always here for you! Please reach out to us at 866-798-5598 for assistance with: 1) insurance issues, 2) finding an HAE expert physician, 3) updating your member information, 4) joining a virtual support group, or 5) HAE-related questions.

Do you have questions regarding HAE and pregnancy? This month’s HAEA Community Blog article explores Lisa’s experience with managing both her pregnancy and HAE diagnosis.

“Despite my HAE attacks increasing in frequency during pregnancy, I was determined to be proactive in my care and treatment to ensure that my HAE was well managed.”

Access the HAEA Community Blog today and read A First Time Mom’s Experience Managing Her HAE While Pregnant: Lisa’s Story by clicking HERE!

Would you like to share a unique story or opinion about your HAE? The HAEA Community Blog is the perfect venue to share your story in your own words with other members of the HAE community. You can share your story HERE!

Podcasts

HAE Speaks Podcast - Episode 34: A Newly Diagnosed Perspective

Kayla, an individual with HAE, shares her perspective on being recently diagnosed with HAE, learning to advocate through the HAEA intern program, and her journey to a diagnosis.

Listen Now!

Do you have a topic, question, or idea that you would like us to cover? Send your suggestion to Mike Mallory at mikemallory@haea.org.

HAE Speaks and #BeyondHAE are available to download on Apple Podcasts, Spotify, or wherever you get your latest podcasts!

Clinical Trial Updates

What is a Clinical Trial?

Clinical trials are research studies conducted to evaluate whether a medicine to treat HAE is safe and effective in humans. Clinical trials are a critical part of drug development, and are required before a treatment can be approved by the Federal Drug Administration (FDA) and become available by prescription.

What are the benefits of participating in a Clinical Trial?

Clinical trials can offer an open label extension, which means free access to HAE medicines being studied until they are approved and commercially available. Participation in a clinical trial also provides regular touchpoints with an HAE physician throughout the trial period, and allows you to have a more active role in your health care.

Following is a list of clinical trials currently underway. If you are interested in receiving more information on clinical trials, or are interested in participating in any of the trials listed below, please reach out to the HAEA Research Team at research@haea.org.

The OASIS-HAE and OASIS-HAE Plus Studies

The US HAEA is currently assisting IONIS in recruiting people with HAE for the OASIS-HAE and OASIS-HAE Plus studies.

The OASIS-HAE and OASIS-HAE Plus studies are evaluating the safety and effectiveness of an investigational subcutaneous preventive medicine called donidalorsen, which is given either once every 4 or 8 weeks. The studies are enrolling participants 12 years of age and older. This investigational drug uses a technology called antisense, which targets the kallikrein generating system using messenger RNA to suppress the protein that produces kallikrein.

The OASIS-HAE Study is a Phase 3 worldwide placebo-controlled trial, and the OASIS-HAE Plus Study is an open-label extension study without placebo.

To qualify for the OASIS-HAE or OASIS-HAE Plus studies, participants must be 12 years of age or older with a documented diagnosis of HAE. Participants must experience at least two attacks during the screening period, and have access to, and the ability to, use at least one or more acute medications to treat HAE attacks.

KONFIDENT Study

The US HAEA is currently assisting Kalvista in recruiting people with HAE for the KONFIDENT study, a Phase 3 worldwide placebo-controlled trial where doctors are studying two different doses of KVD 900-301 to assess which one may work best as a safe on-demand treatment for HAE attacks.

To qualify for the KONFIDENT study, participants must be 12 years of age or older; diagnosed with HAE type I or II; have access to an on-demand medication to use for HAE attacks; and be willing to use a study-approved method of birth control during the study.

HAERMONY 1 Study

The US HAEA is currently assisting BioMarin in recruiting people with HAE for the HAERMONY1 study, an investigational gene therapy called BMN 331 to improve HAE symptoms in adults 18 years of age and older diagnosed with HAE due to C1-INH deficiency.

To qualify for the HAERMONY1 study, participants must be 18 years of age or older; diagnosed with HAE due to C1-INH deficiency; have 6 months of a continuous prophylaxis or on-demand treatment regimen (prior to study enrollment); and a documented attack frequency of at least 1 attack per month on average.
*Additional study requirements apply.

For those who qualify, participation lasts about 5 years after receiving the study drug. There are weekly and monthly visits during the first year. After the first year, the visits occur every three or every six months.

If you have HAE Type I, Type II, or HAE with Normal C1-Inhibitor and are interested in learning about new clinical studies to prevent attacks, please contact an HAE advocate at 866-798-5598 to see if there will be an active clinical trial site in your area.

HAEA Events Calendar

Tuesday, February 14th, 2023 - 6:30 PM ET

HAEA Round Table: Relationships and HAE (VIRTUAL)

Watch on the US HAEA official Facebook page HERE!

Thursday, February 16, 2023 - 7:00 PM ET

HAEA Treatment Education Series Webinar: Taking The Next Step: Navigating Young Adulthood with HAE (VIRTUAL)

Date: Thursday, February 16, 2023
Time: 7:00 PM ET / 4:00 PM PT
Speaker: Dakota Fisher-Vance (BioCryst Associate Director of Global Patient Advocacy)
Sponsored by: BioCryst
Link Webinar: https://haea.zoom.us/j/82645538163
Phone: +1 646 558 8656 (Webinar ID: 826 4553 8163)

Upcoming HAE Heroes Connecting Support Groups:

Wednesday, February 1st, 2023 - 7:30 PM ET
Thursday, February 9th, 2023 - 11:00 AM ET
Tuesday, February 20th, 2023 - 10:00 PM ET

If you are interested in joining a virtual support group, please contact Troyce Venturella at troyce@haea.org or Adina Ramsey at adinaramsey@haea.org

Cincinnati, Ohio In-person Meet & Greet
Date: Thursday, March 30, 2023
Time: 6:30 PM ET
Location: Trio Bistro 7565 Kenwood Rd, Cincinnati, OH 45236
Guest Speaker: Jonathan Bernstein, MD

Register today and receive a FREE goodie bag for attending!

Social Media Snapshots

Don’t miss the latest HAEA news and announcements, connect with us on social media!

support Families4HAE

Update your HAEA membership information HERE!


10560 Main Street, Suite PS40 Fairfax City, VA 22030		CONTACT AN HAE ADVOCATE: (877) 839-4232
DONATE		JOIN HAEA
The HAEA is an advocacy and research organization committed to actively engaging our community in a wide variety of grassroots activities that promote HAE education and awareness. We provide personalized services to address the unique needs of people with HAE and their families, which includes helping them secure access to and reimbursement for modern HAE medicines. Our great success in supporting clinical research has resulted in a variety of FDA-approved therapeutic options. We work closely with expert physicians to continuously upgrade our community's quality of life through improving diagnosis and knowledge of the condition, and encourage an individualized approach to selecting an optimal treatment. The HAEA is product and company neutral, and continues to enthusiastically support drug discovery research aimed at the next generation of HAE therapies. Copyright © (2022) US Hereditary Angioedema Association All rights reserved. No part of this publication may be reproduced, stored in a retrieval system, or transmitted in any form by any means, mechanical, electronic, photocopying, recording, or otherwise without the prior written permission of the US Hereditary Angioedema Association. For information, send an email to info@haea.org.