Traditionally payers hadn't restricted reimbursement to strictly clinical trial population in ultra orphan diseases. This is uncharted area in US as clinical trials typically limit enrollment to very small subset in ultra orphan diseases.
You can also see sentiment changes from patients in story linked back in this chain: they used to be outraged with insurance companies not covering approved drugs, now they are more or less resigned to limitations after hearing about price of drugs.
Register for free to join our community of investors and share your ideas. You will also get access to streaming quotes, interactive charts, trades, portfolio, live options flow and more tools.