OBJECTIVES: Nadofaragene firadenovec (awaiting regulatory approval) and pembrolizumab are bladder-sparing alternatives for treating Bacillus Calmette–Guérin (BCG)-unresponsive, high-risk non-muscle invasive bladder cancer (NMIBC). Cost-utility and value of information (VOI) analyses of these treatments, compared with a hypothetical treatment comparator based on approved and investigational therapies, were conducted from a United States health system perspective.

METHODS: Total costs, quality-adjusted life years (QALYs) and incremental cost-utility ratios (ICUR) were obtained using a cohort simulation Markov model. The ICUR for nadofaragene firadenovec was evaluated separately in two populations- those with carcinoma in-situ (CIS) and those without CIS (non-CIS). VOI analysis was conducted using the single loop Monte Carlo scheme. Individual and population expected value of perfect information (EVPI) for a range of willingness-to-pay (WTP) thresholds was obtained using net monetary benefit estimates from 10,000 Monte Carlo simulations. Expected value of partial perfect information (EVPPI) was estimated for the deterministically most influential model input.

RESULTS: At $150,000 WTP, nadofaragene firadenovec was not cost-effective in the CIS population ($263,233/QALY gained) but cost-effective in the non-CIS population ($145,394/QALY gained), using a placeholder price based on recent trends in anticancer drug prices. Pembrolizumab was not cost-effective in the CIS population ($167,677/QALY gained). The most influential model variable was the probability of NMIBC progression to MIBC. The probability of nadofaragene firadenovec being cost-effective was 17.4% (CIS); 55.6% (non-CIS); and of pembrolizumab, 48.8%. Individual EVPI and EVPPI, respectively, for nadofaragene firadenovec were $5,148 and $0 (CIS); $23,851 and $12,599 (non-CIS); and for pembrolizumab, $18,183 and $12,429. Assuming 22,730 incident cases annually, 10-year EVPI for nadofaragene firadenovec was $1.03 billion (CIS) and $5.56 billion (non-CIS); and for pembrolizumab, $3.6 billion.

CONCLUSIONS: Nadofaragene firadenovec and pembrolizumab may prove to be cost-effective therapeutic alternatives. Given the considerable uncertainty around currently available cost-effectiveness estimates, further research could be valuable to guide decision making.