ICER Says Gene Therapies For Sickle Cell Likely To Improve Lives, But Points To Uncertainties

With the US Food and Drug Administration likely to soon have two applications for gene therapies to treat sickle cell disease (SCD) on its plate, the Institute for Clinical and Economic Review (ICER) is assuming a price of $2m with potential outcomes-based reimbursement agreements for each while emphasizing the need to balance their potential to improve patient’s lives with ongoing uncertainty about their durability as well as concerns about adverse events, particularly from myeloablative conditioning.

The drug pricing watchdog released the draft evidence report on 12 April for the two therapies, bluebird bio’s lentiviral vector-based lovotibeglogene autotemcel (lovo-cel) and Vertex Pharmaceuticals Incorporated and...