OBJECTIVES: During the decision-making process on the reimbursement of health technologies, describing the limitations of health economic analyses in a standardized procedural framework can bring added value to the critical assessment of reimbursement dossiers of health technologies. The aim of this research is to describe the usability of a procedural framework to identify, quantify and interpret such limitations, using the submissions of cell- and gene therapies as an example.

METHODS: In the procedural framework designed for the critical assessment of cost-effectivenes analyses, the quantifiability of an indentified limitation is assessed through the input parameters of the originially submitted model, which is followed by the interpretation of the limitation’s impact on estimates of costs and outcomes compared to the base case cost-effectiveness conclusion.

RESULTS: Based on our experiences with recent cost-effectivenes analyses of cell- and gene therapies, a non-quantifiable, but major limitation influencing the cost-effectiveness conclusion is the effectiveness estimates of the comparator, which is related to the frequent single-arm study designs in the evidence base, and therefore the widespread use of secondary data sources. According to our results, the discount rate applied in the analyses have a quantifiable and major impact on cost-effectiveness conclusions; an also quantifiable, yet minor impact was identified in the case of the methodological approach used to estimate relative effectiveness.

CONCLUSIONS: The procedural framework, demonstrated with an example, was sufficiently flexible and usable to document and structure the limitations of cost-effectiveness analyses during the critical assessment process. The use of the framework described hereby would be desirable when making decisions on the reimbursement of health technologies with the overarching goal of to standardizing the terminology.