Highlights
- •There are few therapeutic alternatives for the treatment of bacillus Calmette-Guérin (BCG)–unresponsive non–muscle-invasive bladder cancer. Although cystectomy is potentially curative, many patients are ineligible for or unwilling to undergo cystectomy. Nadofaragene firadenovec and pembrolizumab are new bladder-sparing treatments.
- •This study evaluated the lifetime cost-effectiveness of nadofaragene firadenovec and pembrolizumab from the US health sector perspective.
- •Using a placeholder price, nadofaragene firadenovec was cost-effective in the non–carcinoma in situ (CIS) population but not in the CIS population. Pembrolizumab, at its current price, was not cost-effective in the CIS population. Cost per quality-adjusted life-year gained and threshold price estimates can aid drug pricing negotiations between payers and drug manufacturers.
Objectives
Nadofaragene firadenovec is a gene therapy for bacillus Calmette-Guérin (BCG)–unresponsive
non–muscle-invasive bladder cancer (NMIBC) undergoing Food and Drug Administration
review. Pembrolizumab is approved for treating patients with BCG-unresponsive NMIBC
with carcinoma in situ (CIS). We evaluated the cost-effectiveness of these treatments
compared with a hypothetical therapeutic alternative, at a willingness-to-pay threshold
of $150 000 per quality-adjusted life-year (QALY) gained, in CIS and non-CIS BCG-unresponsive
NMIBC populations.
Methods
We developed a Markov cohort simulation model with a 3-month cycle length and lifetime
horizon to estimate the total costs, QALYs, and cost per additional QALY from the
health sector perspective. Clinical inputs were informed by results of single-arm
clinical trials evaluating the treatments, and systematic literature reviews were
conducted to obtain other model inputs. Sensitivity analyses were conducted to assess
uncertainty in model results.
Results
Nadofaragene firadenovec, at a placeholder price 10% higher than the price of pembrolizumab,
had an incremental cost-effectiveness ratio of $263 000 and $145 000 per QALY gained
in CIS and non-CIS populations, respectively. Pembrolizumab had an incremental cost-effectiveness
ratio of $168 000 per QALY gained for CIS. A 5.4% reduction in pembrolizumab’s price
would make it cost-effective. The model was sensitive to many inputs, especially to
the probabilities of disease progression, initial treatment response and durability,
and drug price.
Conclusions
The cost-effectiveness of nadofaragene firadenovec will depend upon its price. Pembrolizumab,
although not cost-effective in our base-case analysis, is an important alternative
in this population with an unmet medical need. Comparative trials of these treatments
are warranted to better estimate cost-effectiveness.
Keywords
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Article info
Publication history
Published online: December 15, 2022
Accepted:
December 3,
2022
Publication stage
In Press Corrected ProofIdentification
Copyright
© 2022 International Society for Pharmacoeconomics and Outcomes Research, Inc. Published by Elsevier Inc.
