ISSUE: Recent developments in regenerative and immuno-oncological (IO) therapies, such as CAR-Ts and gene replacement across diverse indications have raised the tantalizing promise of a “cure”. Plateaus have emerged in the progression-free and overall survival curves following IO treatment in some cancers with clinicians interpreting these as indication of cure in some patients. Cure has appeared in statistical analysis and clinical discussions as well as in economic models, yet assumptions around cure fractions remain among the most controversial topics in the HTA assessments. There seems to be no consensus on the definition of cure, nor on how it should be modelled in economic evaluations. In this context the panel would discuss if there should be a standard definition of cure and how it should be defined or shall we continue with definitions from various stakeholders, 2) what are the ways that potential cure can or should be included in economic evaluations and the 3) impact of patient population and disease means when discussing cure.

OVERVIEW: The moderator, Dr Ishak, will introduce the topic and present current statistical methods to estimate cure; he will also challenge the panellists to what extent a consensus can be reached. Dr Latimer, as member of a NICE Appraisal Committee will address how cure has been and could be included in economic evaluations and what impact it has on HTA decisions; Dr Ryll will cover the patients’ and clinicians’ point of view; Ms Benedict will cover the perspective of pharmaceutical companies based on a set of interviews with representatives of companies developing CAR-T, cell and gene-therapies across a diverse set of diseases, impacting different age groups. The moderator will present for 5 minutes and each presenter will present for 10-12 minutes, with 20-25 minutes for panel discussion with audience participation.