Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare.

BACKGROUND: Advanced therapy medicinal products (ATMPs) are beginning to reach European markets, and questions are being asked about their value for patients and how healthcare systems should pay for them. OBJECTIVES: To identify and discuss potential challenges of ATMPs in view of current health technology assessment (HTA) methodology-specifically economic evaluation methods-in Europe as it relates to ATMPs, and to suggest potential solutions to these challenges. METHODS: An Expert Panel reviewed current HTA principles and practices in relation to the specific characteristics of ATMPs. RESULTS: Three key topics were identified and prioritised for discussion-uncertainty, discounting, and health outcomes and value. The panel discussed that evidence challenges linked to increased uncertainty may be mitigated by collection of follow-on data, use of value of information analysis, and/or outcomes-based contracts. For discount rates, an international, multi-disciplinary forum should be established to consider the economic, social and ethical implications of the choice of rate. Finally, consideration of the feasibility of assessing the value of ATMPs beyond health gain may also be key for decision-making. CONCLUSIONS: ATMPs face a challenge in demonstrating their value within current HTA frameworks. Consideration of current HTA principles and practices with regards to the specific characteristics of ATMPs and continued dialogue will be key to ensuring appropriate market access. CLASSIFICATION CODE: I.

A comparison of patient and population values for health states in varicose veins patients.

Cost utility analyses typically incorporate preferences based upon the mean values for health states generated from a sample of the general population. The main argument for using general population values rests upon the premise that in a publicly funded health care system the main objective of the health care system is a societal one, namely to maximise health subject to equity concerns. Alternatively, it can be argued that patients themselves should be asked to value their own health state given that they have first hand experience of the state being valued. This paper investigates the relationship between patient and general population values within the context of a randomised controlled trial to compare alternative treatments for varicose veins. It was found that patients' own health valuations tended to be higher than those of the general population sample. The results from this study have important implications for economic evaluation. The incremental QALY gain for patients receiving treatment for moderate varicose veins would be almost halved if patients' own values were used to estimate QALYs in contrast to the general population values. The results also suggest that the variant of elicitation technique may be more important than the source of those values in revealing discrepancies.