Objectives
Nadofaragene firadenovec is a gene therapy for BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) undergoing FDA review. Pembrolizumab is approved for treating BCG-unresponsive NMIBC patients with carcinoma in-situ (CIS). We evaluated the cost-effectiveness of these treatments compared with a hypothetical therapeutic alternative, at a willingness-to-pay (WTP) 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 three-month cycle length and lifetime horizon to estimate 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 (ICER) of $263,000 and $145,000 per QALY gained in CIS and non-CIS populations, respectively. Pembrolizumab had an ICER 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 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, though 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.
