ICER looks for input on how to assess advanced therapies

Health systems globally are waking up to the fact that a method for assessing the value of treatments for chronic conditions is not necessarily equipped to assess cures or transformative therapies.

Medicines like Novartis’ (NOVN: VX) Zolgensma (onasemnogene abeparvovec-xioi) – a potentially curative one-off gene therapy to treat children with spinal muscular atrophy (SMA) that has become the world’s most expensive drug – cannot be considered in the same way as a drug that a diabetes patient takes as a maintenance therapy for 70 years.

For this reason, the USA’s unofficial cost-effectiveness body, the Institute for Clinical and Economic Review (ICER), has released a draft set of proposed adaptations to its value framework to be applied in the assessment of potential cures and other treatments that qualify as single or short-term transformative therapies (SSTs).