Even at $2M+, ICER calls blue­bird's po­ten­tial gene ther­a­py for blood dis­or­der 'su­pe­ri­or' to stan­dard of care

The In­sti­tute for Clin­i­cal and Eco­nom­ic Re­view, a drug pric­ing watch­dog, gave a mea­sured thumbs up on Thurs­day morn­ing to blue­bird’s po­ten­tial­ly ex­pen­sive new gene ther­a­py for a rare but de­bil­i­tat­ing blood dis­or­der, which will go be­fore an FDA ad­vi­so­ry com­mit­tee next Fri­day.

While blue­bird raised some stick­er-shock eye­brows with its pro­posed price of $2.1 mil­lion per treat­ment course of what’s known as beti-cel, ICER said pre­vi­ous­ly in April and again to­day that the ther­a­py would be cost-ef­fec­tive, as long as there’s an 80% pay­back op­tion for pa­tients who do not achieve and main­tain trans­fu­sion in­de­pen­dence over a five-year pe­ri­od.

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