March 20, 2024

From the President’s desk

From the President’s desk

We recently celebrated International Rare Disease Day and are actively preparing for World Primary Immunodeficiency Week in April. This is an opportunity for IPOPI to take a look at our constantly evolving world, as we find ourselves at the crossroads of new thrilling developments and challenges. Exciting advancements are on the horizon. Newborn screening is showcasing its potential to diagnose the most severe treatable rare diseases before they become symptomatic. Targeted therapies and Advanced Therapeutic Medicinal Products offer new hope for access to care, and even cure, for previously untreatable conditions. Concurrently, it is imperative to reassess and reinvent models that have been successful in covering the costs of medicines, ensuring that treatments become accessible. There is nothing more tragic than having a treatment within reach but inaccessible to those in need.

Thanks to networks such as ERN RITA, the field of immunodeficiency is expanding, revealing valuable commonalities across rare immunological diseases.

Beyond recognising shared interests, we can also extend our collaboration to more prevalent conditions such as some secondary immunodeficiencies and even cancer, where research efforts sometimes overshadow our own. Artificial Intelligence offers unparalleled possibilities in our quest for improved healthcare. While navigating this dynamic landscape, we must remember that we, as a community, hold the power to drive positive change for the well-being of those we serve.

However, in parallel to these breakthroughs, we also need to consolidate our fundamentals. Raising awareness remains paramount to dramatically improve diagnosis in all world regions and we applaud our dynamic national member organisations who, with dedicated physicians and researchers, tirelessly commit in their mission, navigating the complexities of managing challenging conditions. Effective advocacy is essential to ensure decision-makers have a science-based understanding of our field’s intricacies and needs. Education empowers all stakeholders, from patients learning about their chronic condition and its management to healthcare professionals staying updated on advancements.

Above all, we need collective intelligence to tackle multifaceted challenges. Patients, healthcare providers, researchers and the tech and pharmaceutical industry must work together to form a robust ecosystem of progress, overcome challenges, and celebrate successes. Also, let us stand together against misinformation, whether unintentional or deliberately disseminated, ensuring that accurate information guides any stakeholders’ decisions and actions.

The future may seem overwhelming at times and it’s always easy to romanticise the past. However, at IPOPI, we choose adaptation as a strength, not only as a need. We believe that now, more than ever, we hold the power to shape a better future for patients with PID. Together!

Martine Pergent
IPOPI President