Primary Risk Drivers
Below is a snapshot of domains that materially influence the MARA Rating.
Clinical effectiveness
Ranibizumab demonstrated moderate clinical effectiveness in improving best corrected visual acuity (BCVA) compared to verteporfin photodynamic therapy (vPDT) in the RADIANCE trial, with statistically significant gains in BCVA at 3 months. However, the uncertainty regarding long-term efficacy beyond this period limits the rating to A.
Cost effectiveness
Quality of life
The RADIANCE trial reported statistically significant improvements in the NEI VFQ-25 composite score and EQ-5D scores for ranibizumab compared to vPDT, indicating moderate gains in health-related quality of life. However, the lack of clarity on the statistical significance of some measures prevents a higher rating.
Supporting Domains
Safety and Adverse Effects
Ranibizumab was found to have a manageable safety profile with mostly mild ocular adverse events. The evidence suggests that serious adverse events are rare, supporting a good safety rating.
Comparator Selection
The manufacturer did not include bevacizumab as a comparator, despite it being listed in the appraisal scope. The primary comparison was made with vPDT, which is less commonly used, leading to a suboptimal comparator selection.
Patient Population and Subgroups
The trials included a diverse patient population with relevant characteristics for the intended use of ranibizumab. However, there were some limitations in subgroup analyses, but overall, the population is considered representative.
Care Pathway Integration
Ranibizumab can be integrated into existing care pathways with minor adjustments, such as monitoring requirements. The treatment aligns well with current clinical practices for managing visual impairment due to choroidal neovascularisation.
Resource Use and Cost Implications
The cost implications of ranibizumab are manageable, especially with the patient access scheme in place. The overall resource use is justified by the clinical benefits observed, supporting a positive rating.
Evidence Quality and Robustness
The evidence base is primarily derived from a Phase 3 trial (RADIANCE) and supported by additional studies, although some methodological concerns exist regarding the exclusion of certain data. Overall, the evidence is considered robust.
Uncertainty, Sensitivity, and Broader Impacts
There are notable uncertainties regarding the long-term efficacy of ranibizumab and the assumptions made in the economic model. While the treatment addresses an unmet need, the uncertainties limit the overall confidence in the findings.
