On 4 November, HFE attended the 5th Primary Immunodeficiencies (PID) Forum organised by International Patient Organisation for Primary Immunodeficiencies (IPOPI). Chaired by MEP Dr. Cristian Busoi (EPP, RO) and MEP Dr. Boleslaw Piecha (ECR, PL) the meeting reflected on the development of a comprehensive framework of care and policy to deal with PIDs at the European level.
PIDs represent a group of more than 250 genetic rare disorders which affect the human immunity system. IPOPI is the Association of national patient organisations dedicated to improving awareness, access to early diagnosis and optimal treatments for primary immunodeficiency (PID) patients worldwide.
The 5th Primary Immunodeficiencies Forum brought together patients, health professionals, patient organizations, representatives of the European Parliament, the European Commission, and industry to hold a debate on the challenges facing patients with PIDs in Europe and define a common and concrete pathway to better address PIDs.
Mr. Johan Prévot, Executive Director of IPOPI, gave a broad overview of the definition of PIDs and patients’ needs. He pointed out the urgency to have a comprehensive framework for PIDs, “there is a need for a macro-social approach for these conditions” and he suggested building a bridge between policy and care. He reminded participants how early diagnosis can insure a very high quality of life to PIDs patients, but unfortunately PIDs are not easily to diagnose and in the European Member States there is poor knowledge of these conditions.
Given the specificity and need of access to the most modern technologies to treat these diseases, he also remarked on the crucial role of innovation and research to treat these diseases. Genetic screening, newborn screening and genetic therapies can facilitate early diagnosis and improve patients’ quality of life and the development of an efficient and well organized European Health Technology Assessment system is a priority.
Dr Mahlaoui as a doctor specialised in PIDs identified the main medical challenges of PDIs in evaluating outcomes of new research on therapies, improving treatments and care, creating an European register to measure therapy improvement and outcomes, establishing an European Reference Network of highly specialised healthcare providers which represents a clear added value for the European Union and will help to provide affordable, high-quality and cost-effective healthcare to patients with conditions requiring a particular concentration of resources or expertise.
Finally, Ms. Jose Drabwell gave an overview of the PIDs at the international level. She emphasized the unacceptable difference of treatments between continents, countries and hospitals in the same city. Based on her professional and personal experience, she stressed the economic burden of PIDS for the national health systems. In her opinion the situation can improve only developing and incentivising the already existing home therapies, which might represent an improvement in patients` life and cost reduction for the national economies.
In the closing remarks, the chairs MEP Dr. Cristian Busoi (EPP, RO) and MEP Dr. Boleslaw Piecha (ECR, PL)MEPs offer their time and their support to keep working on PIDs and pushing for European legislation which puts patients’ interest at the centre.