The new year presents a fresh slate and a chance for renewal. We asked leaders in patient advocacy what makes them optimistic about 2023. Hear about the goals and projects they’re planning to tackle:
Johan Prevot, Executive Director, International Patient Organisation for Primary Immunodeficiencies (IPOPI)
IPOPI’s busy 2023 program makes me optimistic about the impact our organization can have and the positive effects it will have for patients with primary immunodeficiencies in 2023. We are not only training patient representatives at our regional meetings in both Africa and Latin America, but we are also preparing for our 6th International Primary Immunodeficiency Congress (IPIC2023) which takes place in Rotterdam in November 2023.
Parallel to this, we will take advantage of any possibilities throughout the year to promote access to diagnosis, enhanced plasma collection as well as highlight the importance of newborn screening, among many other important topics. IPOPI is starting the year 2023 with a lot of enthusiasm, and we look forward to collaborating with our national member organizations and other stakeholders to achieve our goals for the next year.
Dr. Len Valentino, President & CEO National Hemophilia Foundation
I continue to be optimistic that this community will persevere and strive for a better life that is rooted in health equity by working to ensure access to care and treatment, providing education and advocating for what is needed for each community member to thrive and live their best life.
Danielle D., Primary Immunodeficiency Patient and new mom to twins
Personally, I am optimistic about 2023 as a first time mom to twin boys. I am excited to see them grow, learn and absorb their surroundings! Having these two little boys makes me want to take even better care of myself so that I can always be there for them!
Tony Castaldo, President & CEO, U.S. Hereditary Angioedema Association (HAEA) and CEO HAE International
Despite having very effective HAE therapies approved in the United States, clinical development of next generation continues apace and we expect a new medicine to be approved next year.
Alain Baumann, CEO, World Federation of Hemophilia
The recent FDA approval of a gene therapy drug is reason enough to be optimistic about 2023. It’s a sign that the hope so many people in the community have is getting closer to being a reality. We are also very optimistic about hosting our global Comprehensive Care Summit, taking place in Buenos Aires in May. This is another first for us and represents an opportunity for our community to get together again, in a multidisciplinary environment, where we can continue learning and sharing experiences. It’s going to be a busy year, and our whole team is excited to get started.
Jodi Taub, Licensed Clinical Social Worker and Primary Immunodeficiency Patient
After a very challenging few years, living as an immunocompromised individual during a global pandemic, I am hopeful that there will be new medical innovations that will provide greater protection, so that we can more freely live our lives.
Scott Santarella CEO, Alpha-1 Foundation
A renewed sense of engagement among patients and key stakeholders, the re-energized commitment of scientists and researchers to advance the field and the continued leadership of partners makes for a promising and very hopeful future for patients. Specifically, CSL’s investment in a second manufacturing facility, fully enabled supply continuity and with a third manufacturing site coming on-line in 2023, patients will have unlimited access to care.
Lisa Butler, Executive Director, GBS CIDP Foundation International
Through the past three years, I think we have learned about tolerance, flexibility and inclusion. I remain optimistic that we can deepen our understanding of these while serving a rare disease community.
Alexandra Nagler, CSL Vifor, Global Patient Advocacy Lead
The patients I recently talked to wished for a return-to-face to face relationships in 2023 as they are craving social contacts they have avoided since the beginning of the COVID-19 pandemic. So I share their optimism that the world will be a safe place again for immunosuppressed people to live a normal life with social interactions that are simply pleasure and not an added risk to their fragile health.
Emmanuelle Lecomte, CSL R&D, Head of Global Regulatory Affairs
As it was mentioned during CSL’s Investors Day, we are advancing and building our R&D capabilities and pipeline to contribute to our sustainable and profitable growth for the next decade. We are investing in CSL’s future by spending more on R&D than ever before and our late-stage pipeline is ready to deliver. It’s been inspirational to see recent successes, the persistence, resourcefulness, behaviors and learning ability that were applied throughout various innovative programs within R&D. I think it is really an exciting time for us to see just how much more we can accomplish together in 2023 and beyond!
Jorey Berry, CEO, Immune Deficiency Foundation
It's thrilling to see new coalitions and platforms to increase PI awareness – for both the general public and for health care providers. These new technologies and partnerships will improve health outcomes on several fronts by reaching those who don't know about PI, reducing the time to diagnosis, and providing opportunities to reduce health disparities.
Shane Fitch, President, Lovexair Foundation and Lovexair ambassador Arran Strong
Arran Strong: I am looking forward to qualifying as an Olympic surfer and raising awareness as an Alpha-1 Athlete.
Shane Fitch: Science and technology are becoming more mainstream in health, treatments, diagnosis and care and innovative therapeutics. Regulatory systems are now supporting fast-track approaches to discovering more about disease by accelerating approval of clinical studies for advanced therapies to be evaluated, along with personalization in treatment delivery, for rare diseases and chronic conditions. This means we can be more agile as a not-for-profit, engaged in digital health research and care support programs.
Where we have an active role connecting people with health care teams in more flexible and versatile projects. Our efforts, competences and resources are growing across a large number of patient communities and network partnerships. This presents us with exciting opportunities from a patient perspective, to help people understand and take a more pro-active role in their future health and care, wherever they are, and in an equitable manner.
Peter L. Saltonstall, President and CEO, National Organization for Rare Disorders (NORD)
In 1983, NORD was instrumental in the passage of the Orphan Drug Act. In 2023, NORD and this historic legislation are celebrating their 40th anniversaries! NORD’s 40th anniversary is an opportunity to reimagine the future for rare diseases – creating a world where every person living with a rare disease can live their best lives. It’s important to remember that our work is not done. As we reflect on our history, we are optimistic that together we can continue to innovate, support research, drive policy and improve care to create a better future for the rare disease community.
