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On @Health_Affairs, me on how @novartis $NVS broke health economic analysis rules to justify Zolgensma’s price, rather than saying “Hey, @US_FDA gave us a monopoly and we are using it!” They say @icer_review supports $2.125MM per dose, not so fast. 1/n healthaffairs.org/do/10.1377/hbl…
4 places @Novartis strayed: cherry picking a sub-group, ignoring quality of life effects on value, comparing to over priced alternative rx, short pay over time horizon relative to long expected benefit time horizon, all detailed in @Novartis release novartis.com/news/media-rel… 2/n
Cherry picked sub-group: @icer_review found that a hypothetical treatment for pre-symptomatic SMA might justify $2.1MM price, but only at $150K per life year gained, and only if newborn screening and immediate treatment widespread. But today it is not. icer-review.org/announcements/… 3/n
1 price across all indicated populations necessitates cost-effectiveness across all indicated populations (Z approval covers all types of SMA). Closest to this is @icer_review price for type 1 SMA encountered clinically. That is $900K (at $150K/QALY, and $1.5M at $150K/LYG) 4/n 
Cost-effectiveness uses the QALY metric. @Novartis chose the $’s per LYG from @icer_review, perhaps because it makes Zolgensma seem to have more value. If you don’t consider quality of life effects, treatments that solely improve function or relieve symptoms have ZERO value 5/n
.@Novartis chose inflated price comparator. Says Z bargain because < 10-year Spinraza cost. But @icer_review says Spinraza overpriced, should cost around $64,000 per year at most. If Spin priced appropriately Z would cost > 3x Spinraza 10 yr price. 6/n nejm.org/doi/full/10.10…
That leaves out that projected Spin lifetime cost is not what @Novartis says. @icer_review said would be around $2.2MM, about same as Z. Spin will go generic at some point too lowering long term cost further, so @novartis claim misleading & wrong. 7/n
And about ‘pay over time’. Thesis is payments contingent on cont'd efficacy. But @Novartis plans 5 annual payments (presumably complete on day 1 of fifth year, not day 365). @icer_review says the benefit of Z has to go > 20 years to justify value, these are out of sync 8/n
Great that @phrma cos embrace cost-effectiveness to align prices with benefits. But the methods must be as strict as they are for effectiveness and toxicity evaluations to ensure the proper amount of society’s wealth is transferred to the company's shareholders. 9/9.
