RARE-Bestpractices
RARE-Bestpractices has developed a networking platform to support the collection of standardized and validated data and the efficient exchange of reliable knowledge and information on rare diseases (RD).
The project was funded by the European Union: Grant Agreement – FP7-HEALTH-2012-Innovation-1-305690
The specificities of rare diseases, including the limited number of patients, the large number of rare diseases, and their diversity in terms of origin and clinical manifestations, create challenges that make research progress and knowledge creation very difficult.
Building on the demand for greater knowledge and the need to share it, the four-year European Union-funded project RARE-Bestpractices – Platform for sharing best practices for the management of rare diseases (www.rarebestpractices.eu) – Grant Agreement FP7-HEALTH-2012-Innovation-1-305690 – worked to create a sustainable networking platform to collect, evaluate, and disseminate Best Practices to improve the management of patients with rare diseases and promote a consistent level of care services across European countries.
A collaborative network was established with experts in guidelines, systematic literature reviews, technology assessment, rare disease epidemiology, and public health from nine European countries. The main results were:
– A methodology for the production and evaluation of rare disease guidelines was agreed upon.
– A systematic collection of guidelines and research recommendations on rare diseases was carried out (these documents are collected in two specific databases available on the project website).
– An analysis of the criteria for defining the value of orphan drugs in 8 member states was conducted.
– A peer-reviewed, open-access journal on rare diseases was created (http://rarejournal.org/rarejournal);
– Two editions of the international course “Health care guidelines on rare diseases. Quality assessment” were organized.
– Training tools were produced to assist guideline developers and evaluators.
– A collaboration with the International Rare Diseases Research Consortium (IRDiRC) was established.
– Scientific coordination and networking were ensured.
The role of the CNR (represented by IRPPS and the Institute of Clinical Physiology) is reported:
– Project Management;
– Healthcare Process Modelling – graphical representation of care processes using standard languages;
– Policy Analysis – analysis of health technology value determination policies, especially in conditions of limited evidence.
CNR prepared the following deliverable: Method of representation of BP guideline through Standardised notations/ languages for graphical representation of healthcare processes
(http://www.cnr.it/peoplepublic/peoplepublic/index/downloadprodotto/i/119746)
CNR participated in the following publications:
Mincarone P, Leo CG, Sabina S, Sarriá-Santamera A, Taruscio D, Serrano-Aguilar P, Kanavos PG. Reimbursed Price of Orphan Drugs: Current Strategies and Potential Improvements. Public Health Genomics, 2017. DOI: 10.1159/000464100. https://www.karger.com/Article/FullText/464100
Mincarone P, Leo CG, Sabina S, Tordrup D, Taruscio D, Kanavos P. Reimbursed Price of Orphan Drugs: A Value Based Framework. Value Health. 2015;18(7):A678.
Sejersen T, Del Giovane C, Filippini G, Leo CG, Meerpohl JJ, Mincarone P, et al. Methodology for production of best practice guidelines for rare diseases. RARE DISEASES AND ORPHAN DRUGS. 2014; 1(1); 10-19. http://rarejournal.org/rarejournal/article/view/35
Pai M, Iorio A, Meerpohl J, et al. Developing methodology for the creation of clinical practice guidelines for rare diseases: A report from RARE-Bestpractices. Rare Dis. 2015;3(1):e1058463. doi:10.1080/21675511.2015.1058463. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4590012/
Taruscio D, Morciano C, Laricchiuta P, et al. RARE-Bestpractices: a platform for sharing best practices for the management of rare diseases. Orphanet J Rare Dis. 2014;9(Suppl 1):O14. doi:10.1186/1750-1172-9-S1-O14. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4249596/