OBJECTIVES: HITs are a new application of precision medicine, yet in terms of cost-effectiveness they face challenges when incorporating DTx costs in economic evaluations. We illustrate how uncertainty in alteration prevalence and DTx provision impact economic outcomes; and the role of DTx implementation and managed entry agreements (MEAs) in reducing access barriers for HITs.

METHODS: Using data from a review of HITs in neurotrophic tyrosine receptor kinase (NTRK) fusions, we estimated how alteration prevalence and associated uncertainty impacted the cost-effectiveness and budget impact results from the perspective of the United Kingdom; and explored examples from the literature on DTx implementation strategies and MEAs for NTRK HITs.

RESULTS: When incorporating DTx costs and assuming identical effectiveness across histologies, incremental cost-effectiveness ratio (ICER) exceeded willingness-to-pay (WTP) for histologies with NTRK prevalence below 1.05%. Differences in DTx costs to identify one eligible patient across histologies and variability around alteration prevalence provided additional uncertainty for both the ICER and budget impact results. To manage uncertainty, we identified examples that used phased approaches to DTx implementation based on alteration prevalence; and MEAs on treatment coverage with evidence development (CED), whereby a share of DTx costs were absorbed by the manufacturer.

CONCLUSIONS: Regardless of therapeutic efficacy, the high cost of DTx in histologies with low alteration prevalence may result in HITs that are not cost-effective for first-to-market innovators. This may impact on reimbursement potential of HITs or greater reliance on CED, with the burden of DTx costs disproportionately borne by the manufacturer. A phased DTx implementation strategy based on both alteration prevalence and therapeutic benefit, and the use of CED for DTx costs, present an opportunity to alleviate uncertainty and barriers to reimbursement. Innovative solutions are needed to ensure patient access to HITs that do not penalize innovators in countries with limited testing provision.