Henderson N., Errea M., Skedgel C. and Jofre-Bonet, M.
In light of concerns that not all medicines for ultra-rare (also known as ultra-orphan) conditions are appraised under the same NICE process, a new OHE Consulting Report discusses the distinct ethical and economic challenges faced by medicines for ultra-rare conditions, with particular reference to the challenges of HTA in the UK. A failure to consistently consider all ultra-rare disease medicines under the HST process could lead to inequalities in access and health outcomes for patients with ultra-rare conditions.
Cubi-Molla, P., Mott, D., Shah, K., Herdman, M., Summers, Y. and Devlin, N.
Cancer survival rates have improved dramatically in recent decades due in part to pharmaceutical advances, with a growing range of increasingly effective and targeted medicines being developed, such as immunotherapies. In the economic modelling of such treatments, the question arises of which utilities should be assigned to patients who show a long-term, durable response.
In recent critiques of economic models in this area by the National Institute for Health and Care Excellence (NICE), the idea that long-term cancer survivors (LTCS) who have received such treatments could report quality of life (QoL) scores which are similar to, or higher than, those of equivalent general population samples has not been viewed as credible. This literature review examines whether there is evidence to support the assumption that the QoL of LTCS can be similar to that of age/sex-matched population samples.
Just published are two new reports that explore the use of real world evidence for coverage and formulary decisions. The publications represent 1) the background paper to the 2017 ICER Membership Policy Summit and 2) a follow up paper based on the discussion at the Summit.