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New HTA Decisions in England
October 2020
Drug name
WAYLIVRA® (volanesorsen)
Company
Akcea Therapeutics
Decision date
18/09/2020
Therapeutic area
Blood and immune system conditions
Therapeutic sub area
Blood conditions
Decision
Recommended
Indication
Volanesorsen is recommended, within its marketing authorisation, as an option for treating familial chylomicronaemia syndrome in adults with genetically confirmed familial chylomicronaemia syndrome who are at high risk of pancreatitis, and when response to diet and triglyceride-lowering therapy has been inadequate.
Decision Detail
It is recommended only if the company provides volanesorsen according to the commercial arrangement (simple discount patient access scheme).
Summary
The clinical evidence for volanesorsen was provided by two double-blind RCTs (APPROACH and COMPASS), an ongoing single-arm open-label study (APPROACH OLE) and the Early Access to Medicines Scheme (EAMS). The committee concluded that the clinical trial evidence showed that volanesorsen would likely provide benefits in lowering triglyceride levels and on clinical outcomes (acute pancreatitis and abdominal pain) in the short term. They also concluded that volanesorsen would likely provide some long-term benefits, although this was associated with substantial uncertainty, particularly at the licensed dose. Because of uncertainties, the company revised its commercial offer for volanesorsen, bringing the ICER down to £98,013 per QALY gained compared with best supportive care and using the committee's preferred assumptions. Acknowledging the uncertainties and taking into account other benefits of volanesorsen that may not be fully captured in the analysis, the committee concluded that volanesorsen can be considered a cost-effective use of NHS resources for highly specialised technologies. Therefore, the committee recommended volanesorsen as an option for treating FCS.