Early Economic Modeling to Inform a Target Product Profile: A Case Study of a Novel Rapid Test for Clostridioides difficile Infection.

Abstract

Background. Target product profiles (TPPs) specify the essential properties tests must have to be able to address an unmet clinical need. Aim. To explore how early economic modeling can help to define TPP specifications based on cost-effectiveness considerations using the example of a new rapid diagnostic for Clostridioides difficile infection (CDI), a contagious health care-associated infection causing potentially fatal diarrhea. Methods. A resource-constrained simulation model was developed to compare a hypothetical test for CDI with current practice (i.e., test with glutamate dehydrogenase enzyme immunoassay first; if positive, test with polymerase chain reaction and cytotoxicity assay) for adult individuals with suspected CDI at the Leeds Teaching Hospital National Health System (NHS) Trust in the United Kingdom. Parameters are taken from UK-based observational data collected between 2018 and 2021, published literature, and expert opinion. A methodological framework was developed 1) to derive minimum diagnostic sensitivity and specificity and maximum price for different test turnaround-time values based on cost-effectiveness considerations from the health care perspective using the National Institute of Health Care Excellence willingness-to-pay threshold of £20,000 per quality-adjusted life-years and 2) to test their robustness using a series of sensitivity analyses. Results. A new rapid test for CDI with a 15-min turnaround time would require a minimum diagnostic sensitivity and specificity both equal to 96% and a maximum price of £44 to maintain cost-effectiveness compared with standard of care. Conclusions. This study provides a framework to inform the essential test properties based on cost-effectiveness considerations and to isolate the most influential model parameters and scenarios via a series of sensitivity analyses. These specifications, in turn, could be used to inform future TPPs for tests.

Highlights: Target product profiles (TPPs) for new medical tests provide test developers with performance benchmarks and technical requirements for new tests. Early economic evaluation has already been used to identify acceptable ranges for certain performance requirements for new tests. Currently, however, early economic evaluation methods are yet to be used in the context of TPP development, and there is no guidance as to how this could and should be done.A de novo approach was developed to identify the minimum performance requirements and maximum costs for new tests, based on cost-effectiveness considerations, while also isolating most influential parameters. The added value of this framework lies in structuring early economic evaluation methods as a means of informing transparent, evidence-based minimum TPP performance specifications while also accounting as much as possible for the (inevitable) uncertainty surrounding the minimum performance requirements.This study represents the first application of early economic modeling as a means of deriving the minimum performance specifications for a novel point-of-care test for Clostridioides difficile infection as set out in a future TPP.