Supporting International Research for Rare Diseases

Supporting International Research for Rare Diseases

April 2011

Two recent workshops sponsored by the European Commission, Health Directorate, DG Research and Innovation, and the US National Institutes of Health Office of Rare Diseases Research (ORDR), focused on the development of international research initiatives of rare diseases.

The first workshop, Fostering Trans-Atlantic Cooperation on Rare Diseases, was held held on October 26 through 28,2010, in Reykjavik, Iceland. Participants explored international coordination in rare disease research. A recommendation was made to launch an International Rare Disease Research Consortium (IRDiRC) that will link funding agencies and organizations, researchers, patient advocacy groups, the pharmaceutical industry, and regulatory agencies from around the world. Click here to read a full report.

The goal of the IRDiRC is to deliver 200 new therapies for rare diseases and diagnostic tests for most rare diseases by 2020. The keys to to success in the research of rare diseases and the development of orphan products and diagnostics are:

Utilizing available resources
Adhering to coordinated research efforts are keys
Global sharing of information, data and biospecimen samples to support research
Fostering links between teams working on similar issues
Avoiding duplication of research efforts

Additionally, there is a need for appropriate rare disease classification, standard terms of reference and common ontologies; and harmonized regulatory requirements.

The second workshop, Fostering International Collaboration on Rare Diseases Research: Launch of the International Rare Diseases Research Consortium, was held in Bethesda, Md., on April 6 through 8, 2011. Participants discussed how the international initiative could be framed. A policy document will be developed before the end of this year, describing goals, research objectives, policies and the governance structure that will help foster international collaboration. Working groups on important issues were suggested. A summary statement from this 2nd workshop can be found here.

Source:  Spotlight on Rare Diseases Newsletter of the NIH Rare Diseases Clinical Research Network, Volume 2, Issue 1, Spring 2011