The International Rare Diseases Research Consortium: Policies and Guidelines to maximize impact

The International Rare Diseases Research Consortium: Policies and Guidelines to maximize impact
In rare disease research, data are scattered and the number of patients is often small, making critical the need for collaboration. The IRDiRC Policies and Guidelines were set out to address best practices in term of data/expertise sharing, integration, use of resources in rare diseases research. A change in how science is performed in addit [...]

Measuring what matters to rare disease patients – reflections on the work by IRDiRC taskforce on patient-centered outcome measures

Measuring what matters to rare disease patients – reflections on the work by IRDiRC taskforce on patient-centered outcome measures
The aim of clinical trials is to evaluate the effectiveness and safety for patients of a medical intervention, based on comparable results: the outcome measures. Despite guidance documents on outcomes, many trials, on rare diseases in particular, still do not include standardized outcomes. This situation leads to failures in late-stage of orp [...]

The 6th IRDiRC Consortium Assembly meeting

The 6th IRDiRC Consortium Assembly meeting
Dr Makoto Suematsu and the Japan Agency for Medical Research and Development (AMED) hosted an IRDiRC Consortium Assembly face-to-face meeting on November 10-11, 2017 in Tokyo, Japan. Over two days, IRDiRC members exchanged on the progresses made by their organizations since the last IRDiRC Conference in Paris, France (February 2017) that cont [...]

The ECRD 2018 will take place on 10-12 May 2018 in Vienna, Austria

The ECRD 2018 will take place on 10-12 May 2018 in Vienna, Austria
The European Conference on Rare Diseases & Orphan Products is the unique forum across all rare diseases, across all European countries, bringing together all stakeholders – patients’ representatives, academics, researchers, health care professionals, industry, payers, regulators and policy makers. “Rare Diseases 360° – collaborativ [...]

The RE(ACT) Congress will take place on March 7-10, 2018 in Bologna, Italy

The RE(ACT) Congress will take place on March 7-10, 2018 in Bologna, Italy
The RE(ACT) Congress – International Congress on Research of Rare and Orphan Diseases was initiated in 2012 by the BLACKSWAN Foundation to create a human platform to scale up scientific cooperation on rare and orphan disease. The 4th edition is co-organized in collaboration with E-RARE ERA-NET for research programs on rare diseases. Tthe RE( [...]

European Commission launches its Horizon 2020 Health Research Programme 2018-2020 including rare diseases

European Commission launches its Horizon 2020 Health Research Programme 2018-2020 including rare diseases
The last 27th October, the European Commission published the last Health programme of the Horizon 2020 research strategy. The programme has the ambition to face the most important challenges on health, demographic change and wellbeing in Europe, including those that are of main importance for rare diseases: the rising and potentially unsustai [...]