Jim, that's a good question. In all that I've read, nothing has been specific as to what trial level a compound or drug has to attain before it reaches submittal qualification. I've only seen other criteria that it needs to satisfy, the life-threatening or debilitating nature of the condition; the medical plausibility of the proposed orphan indication; that the prevalence of the condition in US is less than 200,000; that it is unlikely that marketing the medicinal product in the US, without incentives, would generate sufficient return to justify the necessary investment; that no satisfactory method of diagnosis prevention or treatment exists, or if such a method exists, that the medicinal product will be of significant benefit to those affected by the condition, one would think that certain criteria would have to been met but I can't find anything
masa: I gave up drilling for an answer, zip, nada zero found. There is a lot riding on it if they go there sped up process, tax incentives, extended foreign and domestic patent life, notoriety and they wave the $740,000 filing fee and cash will be a concern by the end of 2014.
Jim Long and calm