4 October 2018 - The ICER today released a final evidence report and report-at-a-glance on inotersen (Akcea Therapeutics) and patisiran (Onpattro, Alnylam Pharmaceuticals) for the treatment of hereditary transthyretin amyloidosis.

ICER’s report was reviewed at a September 2018 public meeting of the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), one of ICER’s three independent evidence appraisal committees. During the meeting, the Council found that both inotersen and patisiran provide a substantial net health benefit when compared to best supportive care alone, but evidence is insufficient to distinguish between the two treatments. However, current pricing far exceeds commonly cited thresholds for cost effectiveness.

During the deliberation, Council members weighed the therapies’ other benefits and contextual considerations. The Council unanimously recognised that the novel mechanism of action was an important other benefit for treating individuals with such a high lifetime burden of illness. A majority also recognised that the new treatments may reduce family and caregiver burden and may improve a patient’s ability to return to work. These Council members emphasised that the burden that a hereditary disease places on families cannot be understated, and that these new treatments may also have a positive psychological effect on multiple generations of a family.

Read ICER announcement