At year’s end, we survey leaders in patient advocacy – including a few of our own employees – about key initiatives and accomplishments that happened over the last 12 months. As always, they had much to share about the impact they and their organizations made and how they are improving the lives of patients who live with rare and serious conditions.
Anthony Castaldo, CEO and Chairman of the Board of HAE International (HAEi) and of the U.S. Hereditary Angioedema Association (HAEA)
Training and motivating the future advocacy leaders is a key objective shared by the U.S. HAE Association and our HAE International organization. In 2023, we introduced LEAP, an educational program that helps youngsters learn new skills, develop as individuals and work on an advocacy project that benefits themselves and their local HAE Advocacy Organization.
Our entire community is beaming with pride because our inaugural LEAP class of 20 young people from 15 countries has “graduated” after they completed the program’s rigorous academic and project management requirements. These young advocates are now injecting their “home” organizations with a refreshing level of enthusiasm backed by newly acquired skills and a burning desire to improve the lives of people with HAE.
Karen Pinachyan, CSL Behring Head of Global Medical Affairs Hematology
Our 2023 accomplishments include an advisory board with 10 hemophilia patients from various countries and with various life experiences. We seek insights from patients because we ultimately design everything we do for them – who better to tell us what they need? Understanding patient needs also helps us plan for the future and design the way we want to partner with patients and patient communities – not only in hemophilia, but also in other disease areas.
Jorey Berry, CEO of the Immune Deficiency Foundation
Our Advocacy Day in early 2023 ultimately resulted in a new law in Texas that banned a health insurance policy was creating a financial burden to accessing treatment. It was important because it improved access to treatment and it was memorable because I spent that advocacy day with Carolyn Ann Demaret, the mother of David Vetter, who was affectionately known as the boy in the bubble, but who was born with Severe Combined Immunodeficiency. It was a pivotal moment to fully understand how our community channels their deeply personal experiences – even when so difficult – into advocacy and support for others, always with the hope that future generations don’t have to endure what they have endured.
Johan Prévot, Executive Director of the International Patient Organisation for Primary Immunodeficiencies (IPOPI)
In 2023, IPOPI made significant strides to improve the lives of primary immunodeficiency (PID) patients worldwide. Collaborations with the United Nations Institute for Training and Research (UNITAR) and the Asia Pacific Plasma Leaders Network (APPLN); the work at the International Coalition for Safe Plasma Proteins (ICSPP); adding to our advocacy efforts around the SoHO legislation aimed at optimizing plasma collection in Europe underscore IPOPI’s commitment to ensuring improved access to life-saving immunoglobulin therapies and plasma for PID patients.
Regional meetings in Africa and Latin America in 2023 provided vital capacity-building platforms for patient representatives and facilitating discussions on key PID priorities. With the addition of four new members, IPOPI’s global impact continues to expand. IPOPI launched the PIDetect Programme in Moldova, supporting local doctors to advance diagnostic expertise. The second edition of the Global Stakeholders Summit in 2023 explored critical areas such as newborn screening, genomic sequencing, and therapeutic advancements, emphasizing IPOPI’s role in shaping the future of PID care. The much-anticipated International Primary Immunodeficiencies Congress (IPIC2023) witnessed substantial engagement, with almost 900 participants actively contributing to the discourse. As IPOPI looks forward to 2024, its collaborative efforts and steadfast commitment to our community solidifies its leading position in advancing PID diagnosis, treatment and care on a global scale.
Arran Strong, Surfer, Lovexair Ambassador and advocate for people who have Alpha 1 Antitrypsin Deficiency
In 2023, I traveled around the world – Africa, Mexico, the coastline of Portugal – to surf and spread a message of positivity. I started filming and recording a podcast on Spotify, the Strong4Cast, where I interview special guests who all have unique stories. I absorb the best from each connection and it has been a really inspiring process that has brought me a lot in my daily life. I’m also celebrating two years of being a Dad!
Lisa Butler, Executive Director of the GBS/CIDP Foundation International
Strengthening our relationships with our international counterparts has been a key focus this year. Not only did we hold a successful multi-lingual chapter meeting in Barcelona, but at our biennial Symposium in Washington D.C., as I greeted patients and families, and watched the various interactions, I knew without a doubt that our mission had been accomplished! Over 15 countries in attendance, and we even planned a special "listening session" with our friends from Latin America. That moment of realization that we are a true global family was one of most memorable.
Paula Manchester, CSL Global Platform Leader, Plasma Product Development
As I reflect on 2023, one of the highlights was the Research & Development Summit in May where we were inspired to think and act as "One R&D". I really appreciated the opportunity to connect with so many colleagues from across the organization. It remains a critical feature in my day-to-day thinking as a "CSL citizen". Our future depends on each employee seeking out opportunities to don their "enterprise lens" and proactively find ways to collaborate.
Alain Baumann, CEO, World Federation of Hemophilia
There were many memorable accomplishments for the WFH in 2022. We celebrated our 60th anniversary, we had a successful WFH 2023 Comprehensive Care Summit, we launched the WFH Shared Decision Making Tool for hemophilia treatment and the WFH Gene Therapy Registry (GTR), just two name a few. But a major highlight for us was certainly the start of a new collaboration with CSL to support the WFH Humanitarian Aid Program. Your CEO Paul McKenzie’s belief in the program was very inspirational for us, and we had a very productive discussion with him in Montreal this year. The next years will see our organizations working even more closely together for the good of the global bleeding disorders community.
Shane Fitch, President of Lovexair, an advocacy organization for people with respiratory diseases
Seeing patients with diverse respiratory diseases and complex health issues (who enrolled with us on different care programs through equitable access) improve their confidence in managing their health. They experience the benefits for themselves, have been able to gain more independence and, for example, reduce anxiety, gain better physical fitness and have learned how to navigate complex health conditions better prepared.
Scott Santarella, CEO, Alpha-1 Foundation
The Alpha-1 Foundation experienced exceptionally positive and enthusiastic engagement with our patients and key stakeholders reinforcing the importance and value of personal interaction and connection in rare disease communities.
Jodi Taub, LCSW, private practice psychotherapist and a primary immunodeficiency patient
Over the last few years, I have been leading caregiver support groups for the Immune Deficiency Foundation. The caregiver experience can be incredibly isolating, especially for those who support loved ones living with rare disease who may never have had the opportunity to connect with other caregivers. Shared experience with peers can be so powerful. I am grateful that I was given the opportunity to support the primary immunodeficiency community in this very special way.
This year, the participants asked to divide the group amongst significant others and caregivers of children (including adult children). Those experiences were similar, but there were distinct differences that could benefit from having their own separate groups. The Immune Deficiency Foundation prioritized the patient experience and this feedback was taken seriously. I now run two separate groups. Both of the groups have been thriving.
