Journal Article

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

Prévot, Johan, Mahlaoui, Nizar, Barasa, Anne, Boersma, Cornelis, Garcelon, Nicolas, Van Coillie, Samya, Notarangelo, Luigi D., Staal, Frank J. T., Rivière, Jacques G., Burns, Siobhan O., Booth, Claire, Drabwell, Jose, Schuller, Björn W., Poli, Cecilia, Van Hagen, Martin, Lee, Pamela, Pergent, Martine, Hendel, Ayal, Sabà, Roser Cantenys, Sánchez-Ramón, Silvia, Rider, Nicholas L., Mamede, Lúcia

2025

Overview

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.

• Publisher: Frontiers Media SA,Frontiers Media S.A
• Subject: Artificial Intelligence / Clinical trials / Cost analysis / CRISPR / Digital health / Gene therapy / gene therapy (GT) / Genetic engineering / Genome editing / Health care access / Hematology / Humans / immunoglobulin replacement therapy (IGRT) / Immunologic Deficiency Syndromes - diagnosis / Immunologic Deficiency Syndromes - therapy / Immunology / inborn errors of immunity (IEI) / Leukemia / Lymphocytes / Manufacturing / Mutagenesis / Nonprofit organizations / Patients / Primary immunodeficiencies / primary immunodeficiencies (PIDs) / Primary Immunodeficiency Diseases - diagnosis / Primary Immunodeficiency Diseases - therapy / Product development / Rare diseases / Reimbursement / targeted therapies / Vectors (Biology)