The rare variants found in isolated populations are not necessarily rare disease targets. For example, the very rare mutations in SCN9A led to a novel class of analgesic (one of the broadest medical indications). However, the combination of new therapeutic modalities like cell and gene therapy, together with a more flexible regulatory and payor environment for rare disease does allow companies to economically pursue treatments and cures for rare conditions.
We believe a private entity is the best way to create value for both participants and industry for two reasons: (1) public projects haven’t succeeded in delivering value to participants themselves - the benefits accrue mainly to researchers and secondarily to public health; (2) to really create value from a population cohort you need more than just data - you need to produce valuable insights like drug targets and real world evidence. As a private entity, we can invest to create that value. The ethical requirements are the same for public and private entities, and as mentioned, we spent 5 years helping build the right ethical framework to ensure Newfoundlanders, and the province as a whole, benefit from our research.