[Mvandenbergh]

NICE, which makes value for money decisions for the NHS, uses a threshold of £25k per Quality Adjusted Life Year for assessing cost effectiveness of treatments.

At that threshold, to pay for an £800k drug treatment you'd need to give someone 32 extra years of life (or 64 years at double the quality of life and so on). This is enough to pay that kind of amount for life saving gene therapies, especially if given to young children but not enough to treat something that can be largely managed through diet control.

Since the majority of the world's patients are in Quebec, I don't understand why the provincial government doesn't cut a deal for this drug. I'm sure they'd rather sell it $250k a dose to a nice big patient population than at $1m to nobody. Quebec has a lot of pricing power here as the only large potential buyer.

[xxpor]

The fact that women can't have children without the drug, but apparently can with it adds a new complication to the QALY calculation I think.

[freeone3000]

RAMQ [Quebec Health] does negotiate separately from the rest of Canada... but they don't have the money. They don't have the money for top-line cancer treatments, and they so severely underpaid optometrists that the optometrist union opted out entirely. There is very little chance that even at $250K a dose (which is an imaginary figure! the sellers have said they will not reduce the price at all!) a negotiation could be reached.