December 19, 2022
From the President’s desk
From the President’s desk
Enthusiastic and energised, this is how we left our recent Global Patient Meetings’ held in Gothenburg, back-to-back with our colleagues and friends from ESID and INGID. It was so good to reconvene together after 4 years behind a screen and enjoy the family feeling again. It was also very moving to celebrate our PID champions, Prof Surjit Singh from India for his tireless commitment to our community and Jerry Zheng from China for his steady efforts in raising awareness on PIDs. Both were respectively the winners of our LeBien and Luciano Vassalli awards. In addition to this, we launched our BIG award that for the first time celebrated a National Member Organisation for one of its brilliant projects and shed the light on SOPPI from Sudan. Bravo!
When listening to our delegates, as well as when travelling back to Asia for two successful meetings, one regional in Kuala Lumpur (Malaysia) and one national in Bangkok (Thailand), I can say that the keyword throughout the world remains ACCESS. Access to diagnosis and to treatment. This word covers different realities but, in many countries, it still means an acute need. This reinforces the sense of our mission to advocate for health systems that take into account chronic and rare diseases, that consider saving lives as well as offering their patients an optimal quality of life thanks to a timely diagnosis and an appropriate treatment, thus allowing them also to lead a productive life. A win-win situation!
Access to diagnosis is paramount for children and adults, including at the earliest stage of life thanks to the newborn screening of the most devastating conditions for which a treatment exists. This is the continued effort we lead together with the International Society of Neonatal Screening (ISNS) and the European Society for Immunodeficiencies (ESID) under the umbrella of Screen4Rare. As per treatment, we obviously focus our efforts on immunoglobulins which are life-saving and life-enhancing treatments for a majority of PID patients, and for which supply remains tight. We advocate for global sufficiency, through regional plasma collection efforts, namely thanks to plasmapheresis: how many lives could we re-enchant and even save when achieving this goal?
Of course, when addressing the many, we don’t put aside the rarest or even ultra-rare diseases. Existing treatments such as transplants, gene therapy or targeted therapies must also be made available and affordable. Speaking of ultra-rare, we are thrilled to prepare for a global IPOPI registry for WHIM syndrome, an ultra-rare disease, potentially underdiagnosed.
With science going ahead steadily, with the genomic revolution identifying more and more genes and specifying diseases, and with medical science identifying new medicines that precisely target dysfunctionalities, we know that the entire perspective that goes from bench to patient will need to be reconsidered. We certainly will have to reinforce stakeholder cooperation to meet everyone’s expectations and needs, with the patient’s health as the ultimate goal. Looking ahead, we have fabulous perspectives as well as important challenges to address, and more than ever this motivates us to always go ahead, duplicating our efforts with our partners, thanks to our sponsors so to change patients’ lives for the best.
We now approach the festive season, for many of us a symbol of family happiness and celebrations, as well as good resolutions for the future. Ours is well known, always working ahead, for long terms outcomes and sustainable improvement of the quality of life of patients with PIDs. I wish you a wonderful end of the year!