HAEA In Action Newsletter Facebook Twitter Instagram LinkedIn
IN THIS ISSUE


support Families4HAE
SAVE THE DATE – 2023 US HAEA NATIONAL SUMMIT

The HAEA is excited to announce that the 2023 HAEA National Summit will take place in Orlando, Florida from July 20 - 23, 2023. The Summit theme is Living Beyond Boundaries and will bring the HAEA community together in-person for the first time in three years. Our goal is to unite patients and caregivers, healthcare professionals, and industry partners once again for a transformative experience of learning and sharing.

The Summit will feature an engaging program that includes:
  • a Professional-Scientific Track,
  • a Youth Health Management and Advocacy Training,
  • Brady Club Kids Workshops for the very young members of our community, and,
  • general sessions covering
    • the latest information on HAE therapies,
    • treatments on the horizon,
    • results of HAEA advocacy research regarding insurance and other challenges,
    • grassroots legislative initiatives.
Registration will open on January 16, 2023, so stay tuned for more details and make sure to Save the Date!

 Watch the 2023 Summit Video Here! 


open enrollment

Dear HAEA Family,

I would like to personally wish you and your family a happy holiday season.

2022 has been an exciting year for our community, and as we look forward to 2023, we have an important announcement.

After many years of selfless and dedicated service, Lois Perry will be retiring at the end of this year. Lois has been an integral part of the community since the HAEA was founded in 1999. Her impact on all people with HAE and their caregivers is immeasurable. Lois has played an important role in transforming a catastrophic unmet medical need into a situation where most people can live a normal life. We cannot thank her enough for her invaluable contributions and commitment to the HAE cause.

Lois personifies the word “advocate” and will be greatly missed. The good news is that she will continue to stay active as a member of the HAE community and looks forward to seeing all of her HAEA friends at our July 2023 National Summit.

Moving forward, Christine Selva will take over as the Director of Health and Research starting in January 2023.

On behalf of our eternally grateful HAEA community, we wish Lois the best of luck and good health!

Warm holiday greetings,

Tony Castaldo
Tony Castaldo



support Families4HAE
The HAEA Shared Decision Making Tool is Now Available!

We are pleased to announce a valuable new resource available to the HAEA Community!

The Shared Decision Making Tool has been specifically designed to guide you and your healthcare professional in developing an HAE treatment plan that fits your needs!

The HAEA has partnered with key opinion leader physicians to develop a Shared Decision Making Tool designed to encourage a more collaborative approach to treatment decisions between people with HAE and their healthcare provider.

Determining the best treatment plan requires a partnership between the individual, healthcare, provider, and often caregivers or family members. The tool takes into account important factors like attack frequency, and HAE’s impact on your health and wellbeing. The Shared Decision Making Tool encourages an open dialogue to help you and your healthcare provider craft a treatment plan that reflects your preferences as well as best medical practices.

This free resource provides a printed summary of your responses to share with your physician before your next visit.

 Click Here to use the Tool! 


If you have any questions, feedback, or would like additional information on the Shared Decision Making Tool, please reach out to Christine Selva at cselva@haea.org.




US HAEA Initiated Research Accepted for Poster Presentations at the 2023 Annual Meeting of Specialist Physicians who Treat HAE

The results of three HAEA initiated research projects have been accepted for presentation at the February 2023 Annual Meeting of the American Academy of Asthma, Allergy, and Immunology.
Having our research selected for presentation provides an important opportunity to share information that affects the care of people with HAE.
  1. Consequences of insurance coverage delays and denials for patients with hereditary angioedema

  2. Modern therapies have dramatically increased quality of life for people suffering from HAE. Nevertheless, insurance changes, delays, and denials are becoming more common. Twenty HAE patients (type 1 and 2) who recently experienced insurance delays or denials completed an online survey, and nineteen participated in a follow-up focus group. The survey and focus group addressed the impact of insurance challenges on the use of healthcare services, work/school attendance, and anxiety. Insurance delays and denials have significant impacts on individuals with HAE including 1) increased urgent care and ER visits, 2) missed work/school days, 3) higher levels of anxiety and 4) a negative impact on family life.

  3. Comparison of Selected Health and Well Being Characteristics in Seniors with Hereditary Angioedema (HAE) and a Non-HAE Patient Matched Cohort

  4. HAE requires lifelong treatment. Yet, there is little information regarding how HAE affects Seniors. This study compared US adult HAE patients ≥60 years to a non-HAE patient matched cohort. Prior research revealed significant QoL and mental health differences between adults with HAE and the general population. With the exception of activity impairment, these data show relatively minor and statistically insignificant differences among the two groups of seniors. These results are likely associated with the significant advances in HAE therapeutic options that have significantly improved quality of life.

  5. Critical Analysis Highlights Methodological Flaws in a Study Using Insurance Claims Data to Assess Cost Effectiveness of Prophylaxis Therapies for the Rare Disease Hereditary Angioedema

  6. In a 2021 pilot study updating an earlier analysis of HAE prophylaxis medicines, the Institute for Clinical and Economic Research (ICER) concluded that these therapies are far less cost-effective than previously estimated. We evaluated ICER’s methodology, including the use and interpretation of insurance claims data and the sources for determining values associated with Quality Adjusted Life Years (QALY) calculations.

    Fundamental flaws in the base level attack rate and utility used to calculate QALYs raise serious questions regarding the validity of ICER’s methodology.


open enrollment
It’s the Holiday Season…And Open Enrollment Season!

We encourage you to take some time to evaluate your health insurance options and compare offerings to figure out which one best fits your needs. The HAEA Health Team is available to help you navigate the Open Enrollment selection process.

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.

ACA (Affordable Care Act): Open Enrollment runs from Monday, November 1st through Sunday, January 15th with coverage beginning on January 1, 2023.

If you don’t enroll in a plan by January 15th, you will be unable to get 2023 coverage unless you qualify for a Special Enrollment 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.



support Families4HAE
IT’S NOT TOO LATE TO MAKE A DIFFERENCE IN THE LIVES OF KIDS, TEENS, AND YOUNG ADULTS AFFECTED BY HAE TODAY!

Dear HAEA Family,

Imagine the joy of helping a child with Hereditary Angioedema (HAE) feel a sense of connection, comfort, and reassurance.

“The HAEA Care Kit meant the world to my daughter. We recently moved from Costa Rica to the United States and all the materials provided in the kit helped Sami to better understand her HAE diagnosis. She loved the HAEA Children’s Book Series because she can learn how to advocate for herself.” Diana M. (caregiver)

Your tax-deductible charitable donation of $20, $50, $100, or more, will make a difference in the future of kids, teens and young adults with HAE.

 Click HERE to make a difference 


Or send a check to:
Hereditary Angioedema Association
10560 Main Street Suite PS40
Fairfax City, VA 22030
*Please write “Families for HAE” in the memo!*



Community Blog
“When I first heard the term “caregiver,” I thought it didn’t apply to me. While I administered emergency medicine and took her to the emergency room, I wasn’t under the impression that I was doing anything special.”

Access the HAEA Community Blog today and read the rest of Will’s journey as a caregiver 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!



Newsletter icon Podcasts
HAE Speaks Podcast

HAE Speaks Podcast HAE Speaks Podcast - Episode 32: In this episode of the HAE Speaks podcast, the HAEA staff talks about their accomplishments in 2022. Listen now to hear what resources are available!

 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.


#BeyondHAE Youth Podcast - HAEA Round Table: Young Adults (Kayla, Jack, and Morgan)

Youth Podcast series As we wrap up the year, we are thrilled to welcome three young adults with HAE who share their experiences living with a rare chronic condition. This episode is part of the HAEA Round Tables and covers topics ranging from overcoming challenges at school, coping with HAE through puberty, dating and relationships, and more. Listen now to hear directly from young adults who share stories about their personal triumphs and challenges as they navigate life with HAE.

 Listen to the Podcast here! 

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

Clinical Trial icon 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.




CSL312 Study

The US HAEA is currently assisting CSL Behring in recruiting people with HAE for the CSL312_3002, a worldwide clinical research study to investigate the safety and effectiveness of Garadacimab, an investigational subcutaneous medication given once a month to prevent Hereditary Angioedema (HAE) attacks. In this study, doctors will be evaluating the long term use of Garadacimab.

The trial is currently fully recruited for adult participants, however, a few more adolescent participants (ages 12 - 17) are needed to meet the required numbers.

CSL312_3002 is an open label study with no placebo phase required, which means that all participants will receive Garadacimab for at least one year.




The OASIS Study

The US HAEA is currently assisting IONIS in recruiting people with HAE for the OASIS-HAE study, a Phase 3 worldwide placebo-controlled trial, which aims to determine whether its study drug (called donidalorsen) is safe and effective at reducing or preventing HAE attacks.

Participants may be eligible to participate in an open-label extension study at the end of the OASIS-HAE study, where all participants receive donidalorsen.

To qualify for the OASIS-HAE study, 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 HAERMONY
1 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

10560 Main Street, Suite PS40
Fairfax City, VA 22030


  
CONTACT AN HAE ADVOCATE:
(866) 798-5598


 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.