RARE-Bestpractices is a four year project (January 2013-December 2016) funded by the European Commission under the FP7 Cooperation Work Programme: Health-2012. The project focuses to the specific action HEALTH.2012.2.4.4-3: Best practice and knowledge sharing in the clinical management of rare diseases.
The specificities of rare diseases (RD), including the limited number of patients, the large number of RD and their diversity as regard origin and clinical manifestations, result in challenges which make research progress and knowledge creation in RD more difficult.
Stemmed from the demand for an increased and shared knowledge on RD, the 4-year EU- 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 for collecting, evaluating and spreading best practices to improve the management of RD patients and promoting a consistent level of healthcare services in European countries.
A collaborating network was set up with experts in the area of guidelines, systematic reviews, health technology assessment, health policy, RD epidemiology and public health coming from 9 countries across Europe. The main results are:
- – Methodology was agreed for production and appraisal of guidelines on clinical management of RDs.
- – A collection of Bestpractices and research recommendations on RD was done (two databases were build and are available on the project website).
- – Value assessment criteria for orphan drugs across 8 EU Member States were identified and synthesized.
- – Dissemination initiatives
- – A new scientific international peer-reviewed open access journal was created (http://rarejournal.org/rarejournal);
- – Two editions of the International Course “Health care guidelines on rare diseases. Quality assessment” were delivered;
- Training tools were produced to assist in RD guideline development and appraisal.
- – Collaboration with the International Rare Diseases Research Consortium (IRDiRC) was activated.
- – Scientific coordination, networking was ensured.
The specific role of CNR (involved with two Institutes: Institute of Clinical Physiology in addition to IRPPS) is reported:
– Project Management;
– Healthcare Process Modelling – graphical representation through standard languages of healthcare processes;
– Policy Analysis – analysis of policies for value assessment of health technologies, especially in conditions of scarce evidence.
- Pierpaolo Mincarone, Giuseppe Ponzini.
CNR was in charge of the following deliverable: Method of representation of BP guideline through Standardised notations/ languages for graphical representation of healthcare processes
Moreover, CNR contributed to the following papers:
- 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/