ENCE-CF-LAM-LTX

"European Networks of Centres of Expertise for CF (Cystic Fibrosis), LAM (Lymphangioleiomyomatosis), and LTX (Lung Transplantation)"

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 Obiettivo del progetto (Objective)

'European Centres of Expertise Networks for rare diseases have been identified by the European Commission as one important area of future activity in the attempt of achieving one of the main objectives across the EC endeavour of “Optimising the delivery of health care to European Citizens”. It is obvious that especially in rare diseases the joined forces of the experts networking throughout Europe are more likely than national services to meet the needs and expectations of patients. The construction and implementation of European networks of centres of expertise (ENCE) call for input from many different stakeholders: patients and doctors and other care team members, clinical researchers, health administration, and health insurers will have to be heard to come to a model of such networks that can easily be used as blueprint for most of the rare disorders. To achieve this goal first a mapping exercise of existing networks and elements needed for such networking has to be done. We plan to do so for three different rare entities, i.e. cystic fibrosis (CF), pulmonary lymphangioleiomyomatosis (LAM), and lung transplantation (LTX), having in common that they all comply with the EC definition of a rare disease. From the information gathered during this process the areas of activity and a detailed set of criteria for such European networks will be developed. These construction principles will be brought together in a blueprint to be used for other rare disorders. These criteria can then be used for certification, quality control, and funding of a European system of networks. Networking in this European sense will make the best possible use of resources already existing and remaining in the responsibility of the individual member states by interfacing and complementing these present and future elements. This will help the health care system partners to learn from the best and improve the quality of care to the best European level of expertise.'

Introduzione (Teaser)

The treatment of rare diseases (RDs) requires experts and specialised infrastructure. EU-funded scientists developed a blueprint for establishing networks of expertise enabling effective treatment and improved outcome.

Descrizione progetto (Article)

Although expert opinions are always valued, many diseases and their therapies have become well established and most patients can be treated effectively in care facilities close to their homes. However, when it comes to RDs, the majority of the medical field is lacking experience. This is why the joined forces of experts from throughout Europe are obviously much more likely to bring about a positive patient outcome.

The EU has targeted the creation of the European Networks of Centres of Expertise (ENCE) for rare diseases as one way to achieve optimal health care for European citizens. In order to develop a blueprint applicable to all RDs, the EU-funded project ENCE-CF-LAM-LTX evaluated implementation for three different RDs.

Consortium members began by mapping available resources and structures separately for each of the three cases. They consulted with patients, health care workers, clinical researchers, administrators and insurers to determine needs and expectations regarding Networks of Centres of Expertise. From the research, scientists constructed a list of elemental requirements, including patient registries, clinical trials networks and the participation of patient organisations. Basic research and biobanking were also fundamental to optimised health care delivery. Each of the elemental components was then described in detail for the three RD case studies.

Scientists then extracted the common characteristics among the three case studies to come up with a generic blueprint that can be applied to any RD in the future. This guiding information was published in a groundbreaking document titled 'How to build European reference networks (ERNs) for rare diseases - Elements, considerations, and materials', also referred to as 'ENCE elements'.

Establishment of ENCE for rare diseases will foster even greater expertise among participants through shared knowledge and experience. This clustering of professional, support and technical resources will enable more efficient and effective patient care, improving prognoses and raising healthcare standards to the highest levels across the EU.