Ed. note: This is the third post by Dr. Kim Hunter-Schaedle recapping the Conference on Clinical Research for Rare Diseases to see the first post please click here.
There are many positive aspects to setting up a research consortium. Not least, funding agencies like them, because they usually mean ‘bang for the buck’ – the researchers have already self organized and come up with a collaborative plan. However setting up a rare disease clinical research consortium presents challenges, especially for running clinical trials. Rare disease clinical trials must be kept small because there will always be a limited access to patients. Despite this one cannot underestimate the cost of a rare disease trial – many of the set up costs for a trial are fixed and must be spent whether the trial includes 100 patients or 1,000.
There are some practical issues that need to be considered up front. The consortium head must recognize this commitment as a top priority. All sites that participate in the consortium must have a committed team – not just a sole investigator at a site. Participating researchers must recognize that it might take a long time before seeing productivity or success. In terms of publishing results when they do come, senior established investigators should consider the opportunity to give more visibility to junior investigators who are still establishing their careers.
Funding is also a key issue. Although funding is committed for the consortium, the researchers should be on the lookout for additional funding that can be brought in to expand the programs of the consortium. This may be federal, foundation or institutional funding.
