The document discusses challenges in accessing rare disease drugs in Canada. It notes that Canada approves only around half of orphan drugs approved in the US and EU. Several provinces have established specialized programs to review rare disease drugs on a case-by-case basis. The document advocates for a "lifecycle approach" where patients are engaged throughout the drug development and review process to improve access and appropriate use. This could include managed access programs combining early approval with ongoing data collection to expand access while managing costs and uncertainties.