ISSUE: Assessing the robustness of clinical plausibility when survival curves are extrapolated to a lifetime has been debated in HTA submissions as it may lead to inappropriate decision-making. Although the challenges in how to best demonstrate the long-term survival benefit of new technologies to HTA bodies and payers are well discussed, these currently remain unresolved. The panel will debate whether the use of a structured process in the format of a framework or other toolkits that attempt to maximise clinical plausibility in the estimation of lifetime clinical effectiveness of new products could mitigate the methodological challenges in this area. Would a standardised process provide enhanced transparency and consistency in HTA decision making? This issue panel will also discuss the value of real-world data (RWD) and visual tools in this context.

OVERVIEW: In HTAs, validation of lifetime extrapolations of key outcomes for assessing comparative clinical and cost-effectiveness between the new technology and standard of care has been critical in decision-making. Providing robust lifetime clinical evidence requires large trial sample sizes and often long follow-up, which can potentially deny or delay critically ill patients access to life-extending therapies. The moderator will introduce this topic and propose the following questions (5 min): how can industry produce accurate long-term survival extrapolations that lead to unbiased treatment effect estimates with short-term trial data? What is the current HTA guidance on this topic? Can improvements in these extrapolations be made? Can RWD be used to support the validation of extrapolations? Each panelist will present a different perspective (consultancy, industry, HTA) debating the need for and applicability of an explicit framework to address this guidance gap (each lasting 10-12 min). 15min interactive discussion will follow with the use of online polling format. Stakeholders (health economists, industry, payers) involved in HTA submissions will benefit.