Presentation (CTI)

OBJECTIVES: A new generation of therapeutics (next-generation therapeutics (NGT)), including cell and gene therapies, advanced drug delivery systems, and novel medical devices, offer transformative health potential but present unique challenges in their development and evaluation. This study identified published early model-based cost-effectiveness analyses (CEAs) of NGTs to understand how they addressed scientific, regulatory, and economic uncertainties and to inform best practice for future evaluations.
METHODS: A systematic review (PROSPERO ID: CRD42023425881) using Ovid (Medline and Embase; Jan 2012 to Feb 2024) identified published early-model-based CEA of NGTs (medicines and medical devices). Data extraction included aspects such as disease area, therapeutic type, decision- analytic model type, parameter inputs and approaches to understand uncertainty and the use of ‘value of information’ (VOI) methods. Studies were assessed for adherence to CHEERS reporting criteria.
RESULTS: Twenty-seven studies were identified: 14 evaluated medicines and 13 evaluated medical devices. Most studies used Markov or decision-tree models; one-third relied on expert opinion or author assumptions for effectiveness and cost inputs due to limited clinical data. Single-arm trials, animal studies,and analogues from previous-generation products were frequently used to inform inputs. Eight studies used VOI methods including estimation of the expected value of perfect information (EVPI), to quantify the potential benefit of additional research. Models were designed to inform pricing, trial design, or go/no-go decisions, although reporting of assumptions and stakeholder engagement was variable.
CONCLUSIONS: Early model-based CEA can contribute to subsequent development of the next generation of therapeutics by identifying key uncertainties and informing future research priorities. Sensitivity analyses and especially VOI techniques are essential in shaping future studies by pinpointing the high impactparameters needing additional evidence generated by specifically designed future research studies. Recommendations include enhancing transparency,applying structured expert elicitation and integrating health economics expertise early in product development.