OBJECTIVES: Traditional health technology assessments (HTAs) assess the cost-effectiveness of a product at the end of the development process, whereas early HTA informs decision-making at early stages of development. The advantage of early HTA is that go-no-go decisions are facilitated prior to investment of considerable resources. The objective of this research was to provide an overview of early HTAs conducted in the US.

METHODS: A comprehensive literature review was conducted to identify empirical studies that reported on the implementation of early HTA in the US. Searches were run in Embase and Medline in December 2023 to identify English-language literature published since 2017. Other sources included hand-searching reference lists of relevant literature reviews.

RESULTS: The literature searches identified 454 records, of which 98 were selected for full-text screening. Seven publications, reporting findings of early HTAs for 7 technologies, met the criteria for inclusion. Technologies assessed included 4 medical devices, 2 drugs, and 1 gene therapy. Markov state transition models were the most common modeling approach (n=4), followed by simulation models (n=2) and decision-analytic models (n=1). Although all early HTAs conducted extensive sensitivity and scenario analyses to identify influential parameters, only 1 conducted value-of-information analysis. All early HTAs reported economic evaluation results in favor of the technology under investigation; 4 early HTAs found the technologies to be potentially cost-effective at relatively high willingness-to-pay thresholds (>$100,000), while 3 early HTAs concluded that the uptake of those new technologies may lead to cost-savings. The lack of robust clinical evidence and uncertainties related to the cost of the technologies were noted as limitations.

CONCLUSIONS: Our findings are in agreement with the conclusions of other reviews published on the topic. Early economic modeling is the preferred tool for informing manufacturers in the initial stages of the development process; however, guidance on how to conduct early HTA is lacking.