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.
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.
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.
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.