Primary Risk Drivers
Below is a snapshot of domains that materially influence the MARA Rating.
Clinical effectiveness
Nintedanib shows moderate benefit in slowing the decline of lung function compared to placebo, with a statistically significant difference in the annual rate of decline in FVC. However, uncertainties remain regarding its long-term treatment effect and whether it improves overall survival, which prevents a higher rating.
Cost effectiveness
The cost-effectiveness estimates for nintedanib are likely within acceptable thresholds for Healthcare resources, with an ICER estimated between £20,000 to £30,000 per QALY gained, indicating a strong economic value.
Quality of life
The evidence for HRQoL improvements is mixed, with no statistically significant difference in K-BILD scores between nintedanib and placebo. While some improvements were noted, they were not robust enough to warrant a higher rating.
Supporting Domains
Safety and Adverse Effects
Nintedanib has an acceptable safety profile, with adverse events primarily being gastrointestinal in nature. The frequency of serious adverse events was similar to placebo, indicating good tolerability.
Comparator Selection
The INBUILD trial used placebo as a comparator, which is appropriate given nintedanib’s role as an add-on treatment. This aligns with Healthcare clinical practice, supporting the validity of the evidence.
Patient Population and Subgroups
The INBUILD trial population is broadly representative of the Healthcare patient population with PF-ILD, with baseline characteristics reflecting those seen in clinical practice, although some limitations exist regarding concurrent treatments.
Care Pathway Integration
Nintedanib can be integrated into existing care pathways with minor adjustments, as it is positioned as an add-on treatment to standard care for PF-ILD.
Resource Use and Cost Implications
The budget impact of nintedanib is manageable and aligns with planning, although there are concerns about the long-term costs associated with treatment discontinuation.
Evidence Quality and Robustness
The evidence base is supported by a Phase 3 RCT (INBUILD) with a robust design, although there are some uncertainties regarding long-term outcomes and the extrapolation of survival data.
Uncertainty, Sensitivity, and Broader Impacts
There are significant uncertainties in the economic model and assumptions regarding treatment effects and mortality, which could impact the overall assessment of nintedanib’s value.
