RARE-BESTPRACTICES

Platform for sharing best practices for management of rare diseases

 Partecipanti

Mappa

 Word cloud

Esplora la "nuvola delle parole (Word Cloud) per avere un'idea di massima del progetto.

 Obiettivo del progetto (Objective)

'RARE-Bestpractices will develop a sustainable networking platform, supporting the collection of standardized and validated data and efficient exchange of knowledge and reliable information on rare diseases (RD). RD are characterized by low prevalence (EU – 5:10000 persons). There are more than 5000, overall affecting about 30 million citizens of all ages in the EU. RD are often life-threatening and chronically debilitating, and healthcare is impaired by limited knowledge. Collaborative efforts are needed to tackle RD to prevent significant morbidity, perinatal or early mortality, to reduce socio-economic burdens and to improve an individual’s quality of life. RARE-Bestpractices aims to improve clinical management of RD patients, narrowing the existing gap among EU MS and other countries, also considering the application of patients’ rights in cross-border healthcare (EU Directive 2011/24). The platform deals with RD as a global health issue, exploiting and integrating contributions from all EU MS and other world areas (Caucasus, Europe, America, Oceania, PAHO/WHO) and will identify additional research needs to further improve clinical practice. Fostering synergistic collaboration among experts, patients representatives, policy makers, institutions, agencies, and other organizations experienced in systematic reviews and guidelines production, RARE-Bestpractices will focus on: a) collection, evaluation and dissemination of existing best practices; b) an agreed methodology suitable to develop and update best practice guidelines; c) training activities targeted at key stakeholders to spread expertise and knowledge; and d) a forum for exchanging information, sharing lessons learnt, and facilitating collaborations. The platform is conceived for health care providers, experts, patients, policy makers and best practice guideline developers with outcomes that support closure of healthcare gaps among countries and improved clinical management of RD patients globally.'

Descrizione progetto (Article)

The EU-funded 'Platform for sharing best practices for management of rare diseases' (http://www.rarebestpractices.eu (RARE-BESTPRACTICES)) project is a consortium of 15 partners. These include a range of academic institutions, health care services, systems analysts, health sector research groups and the European Organisation for Rare Diseases (EURORDIS).

The main aims of RARE-BESTPRACTICES are to develop a methodology for guidelines for RDs that will populate a comprehensive public database. The specially tailored databases will house guidelines and prioritised research recommendations respectively to help professionals, patients and policymakers. Both databases are in the user testing phase, and a prototype for a forum-based web community is ready for testing.

Great emphasis is being placed on training and there is already a schedule for a series of events on guideline development and evaluation. Two workshops have explored the issues involved in using the Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach when developing guidelines for RDs. The draft guidelines will be applied and tested on the RD catastrophic antiphospholipid syndrome.

RARE-BESTPRACTICES is mapping out policies on orphan drug appraisals in eight European countries. Critical factors leading to positive or negative recommendations for pharmaceutical use are being assessed and how these feed into clinical practice. Under study are 17 case studies on 17 drug-indication pairs.

Dissemination is critical and is being addressed through newsletters, a leaflet, Twitter (http://twitter.com/rarebestp (@RAREBestP)), press releases, the project website and several papers in journals. The project is also creating a new science open access journal, http://rarejournal.org (Rare Diseases and Orphan Drugs).

RARE-BESTPRACTICES will help to ensure that the RD community has access to clear, quality guidelines that they can trust. A unique, first-time event, the project has brought together experts in all fields of RDs and this stands to ensure uptake of the innovative findings by the RD community, including RD national plans, centres of expertise and European reference networks in compliance with EU legislation.