One of the key findings of this analysis is that manufacturers tend to implement changes to the development programme based on both regulatory and HTA advice with regards to the choice of primary endpoint and comparator. It also confirms the challenging choice of the study comparator, for which manufacturers seem to be more inclined to satisfy the regulatory advice. Continuous research efforts in this area are of paramount importance from a public health perspective.
attracted over 80 delegates from 24 countries, nearly 60% of whom considered costutility analysis to be a necessary component of a Value Assessment Framework. There was little agreement on whether a single transparent threshold for costeffectiveness was appropriate, with many respondents suggesting a threshold that is much higher than the current cost/QALY. Over half (55%) of respondents considered surrogate endpoints to be acceptable for reimbursement decisions, whilst two thirds (64%) felt that MCDA might be a replacement for the QALY. Over half (53%) also felt that patients should play a more active role in health technology assessments, and risk sharing was considered to be an acceptable method of cost containment by over 70%. Finally, the majority of respondents (74%) felt it appropriate for manufacturers to command high prices for orphan drugs. In a country analysis, albeit limited by small numbers, there was general congruence between Europe and the US, apart from the need for cost-effectiveness analysis (60 vs 36% in favour, respectively). CONCLUSIONS: The Special Task Force has now recommended a refinement of the QALY to reflect societal values, and consider MCDA to be a viable alternative, whilst value assessment should incorporate both patient and population perspectives. The findings of this survey of ISPOR delegates were consistent with these recommendations, which illustrates the interest in the need for a Value Assessment Framework to aid reimbursement decisions.
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.