Navigating disruption in the PID landscape: embracing opportunities and anticipating threats in the next ten years

Lúcia Mamede¹Roser Cantenys Sabà^2,3Samya Van Coillie¹Johan Prevot¹Silvia Sánchez-Ramón⁴Maria Cecilia Poli^5,6,7Anne Barasa⁸Björn Wolfgang Schuller^10,11,9Ayal Hendel¹²Nicolas Garcelon^13,14Cornelis Boersma^15,16,17Pamela P Lee¹⁸Claire Booth^19,20Luigi Daniele Notarangelo²¹Josina Drabwell¹Nicholas L Rider²²Frank Staal^23,24Siobhan Oisin Burns^25,26Petrus MARTINUS VAN HAGEN^27,28,29Martine PERGENT¹Jacques G. Rivière^30,31,32,33Nizar MAHLAOUI^34,35*

• ¹International Patient Organisation for Primary Immunodeficiencies (IPOPI), Brussels, Belgium
• ²Catalan Health Service, Barcelona, Catalonia, Spain
• ³Digitalization for the Sustainability of the Healthcare System (DS3) research group – IDIBELL, Barcelona, Spain
• ⁴Department of Clinical Immunology, IML and Health Research Institute of the Hospital Clínico San Carlos (IdISSC), Madrid, Spain
• ⁵Facultad de Medicina Clínica Alemana de Santiago, Universidad del Desarrollo, Santiago, Chile
• ⁶Programa de Inmunogenética e Inmunología Traslacional, Instituto de Ciencias e Innovación en Medicina, Facultad de Medicina, Santiago, Chile
• ⁷Immunology and Rheumatology Unit, Hospital Dr. Roberto del Río, Santiago, Santiago Metropolitan Region (RM), Chile
• ⁸Immunology Unit, Department of Human Pathology, University of Nairobi, Nairobi, Kenya
• ⁹CHI – the Chair of Health Informatics, Technical University of Munich University Hospital, Munich, Germany
• ¹⁰Munich Data Science Institute, Technical University of Munich, Munich, Bavaria, Germany
• ¹¹Munich Center for Machine Learning (MCML), Munich, Germany
• ¹²Institute for Advanced Materials and Nanotechnology, Faculty of Life Science, Bar-Ilan University, Ramat Gan, Jerusalem, Israel
• ¹³Institut National de la Santé et de la Recherche Médicale (INSERM), Paris, Île-de-France, France
• ¹⁴Imagine Institute, Data Science Platform, INSERM, Université de Paris, Paris, France
• ¹⁵Health-Ecore B.V., Zeist, Netherlands
• ¹⁶Department of Health Sciences, University Medical Center Groningen, Groningen, Netherlands
• ¹⁷Department of Management Sciences, Open University, Herleen, Netherlands
• ¹⁸Department of Paediatrics and Adolescent Medicine, School of Clinical Medicine, LKS Faculty of Medicine, The University of Hong Kong, Pokfulam, Hong Kong, SAR China
• ¹⁹Department of Paediatric Immunology and Gene Therapy, Great Ormond Street Hospital, London, United Kingdom
• ²⁰Infection, Immunity and Inflammation Department, UCL Great Ormond Street Institute of Child Health, London, United Kingdom
• ²¹Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases (NIH), Bethesda, Maryland, United States
• ²²Department of Health Systems and Implementation Science, Virginia Tech Carilion School of Medicine, Roanoke, United States
• ²³The Department of Pediatrics, Pediatric Stem Cell Transplantation Program, Willem-Alexander Children’s Hospital, Leiden, Netherlands
• ²⁴Department of Immunology, Leiden University Medical Center (LUMC), Leiden, Netherlands
• ²⁵Department of Immunology, Royal Free London NHS Foundation Trust, London, United Kingdom
• ²⁶Institute of Immunity and Transplantation, University College London, London, United Kingdom
• ²⁷Division of Allergology and Clinical Immunology, Department of Internal Medicine, Erasmus Medical Center, Rotterdam, South Holland, Netherlands
• ²⁸Department of Microbiology, Faculty of Science, Chulalongkorn University, Bangkok, Bangkok, Thailand
• ²⁹Department of Internal Medicine, Clinical Immunology, Cipto Hospital, Universitas Indonesia, Jakarta, Indonesia
• ³⁰Universitat Autònoma de Barcelona (UAB), Barcelona, Spain
• ³¹Vall d'Hebron Research Institute (VHIR), Barcelona, Catalonia, Spain
• ³²Pediatric Infectious Diseases and Immunodeficiencies Unit, Hospital Infantil I de La Dona Vall d'Hebron, Vall d'Hebron Barcelona Hospital Campus, Barcelona, Spain
• ³³Jeffrey Modell Diagnostic and Research Center for Primary Immunodeficiencies, Barcelona, Spain
• ³⁴Pediatric Hematology-Immunology and Rheumatology Unit, Necker-Enfants malades University Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France
• ³⁵French National Reference Center for Primary Immune Deficiencies (CEREDIH), Necker-Enfants malades University Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France

The International Patient Organisation for Primary Immunodeficiencies (IPOPI) held its third edition of the Global Multi-Stakeholders’ Summit, gathering key primary immunodeficiencies (PID) stakeholders and experts to discuss and foment global collaboration. This edition focused on the impact of genomic medicine in PID treatment, the role of digital health, including artificial intelligence, in PID care, and how to anticipate and minimise risks to ensure optimal patient access to care. These discussions aimed to examine current hurdles and brainstorm feasible solutions and priorities for the PID community in these areas in the next ten years. These discussions led to recommendations for comprehensive approaches to care and access to treatment for PID patients, suggesting actions that will bring the community closer to treatments based on real-world evidence and adjusted to patient’s needs. To accomplish this, collaboration between academia, industry, regulatory authorities, and patients is crucial.