ICER Evidence Report questions long-term efficacy and safety of PNH treatments

US health technology assessor the Institute for Clinical and Economic Review (ICER) has posted its revised Evidence Report assessing the comparative clinical effectiveness and value of iptacopan, from Novartis (NOVN: VX) and danicopan from AstraZeneca’s (LSE:AZN) rare diseases unit Alexion, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).

The ICER noted that, while important health benefits for patients were shown in clinical trials for both agents, there is uncertainty about long-term efficacy and safety.

Using a shared-savings approach, the ICER’s model for iptacopan, which is already US Food and Drug Administration (FDA)-approved, suggests that the drug would need to be priced 70% lower than the current list price to meet commonly accepted thresholds.