Primary Risk Drivers
Below is a snapshot of domains that materially influence the MARA Rating.
Clinical effectiveness
The evidence indicates that somapacitan works comparably to somatropin, which suggests non-inferiority. However, there is no mention of Phase 3 trial data or substantial superiority over existing treatments, which limits the rating to B++. The document states that clinical trial evidence shows somapacitan works as well as somatropin, but does not provide compelling evidence of superior outcomes.
Cost effectiveness
The cost comparison suggests that the cost of somapacitan is similar to or lower than that of somatropin, indicating a clear cost-effective profile under common thresholds. The document states that there is enough evidence to show that somapacitan provides benefits and value for money, supporting a rating of A+.
Quality of life
The document does not provide specific data on HRQoL improvements associated with somapacitan. While it mentions that somapacitan can be used routinely across the Healthcare, it lacks detailed evidence on quality-of-life outcomes, leading to a rating of B+.
Supporting Domains
Safety and Adverse Effects
The document does not highlight any significant safety concerns or adverse effects associated with somapacitan, indicating an acceptable safety profile. However, without detailed safety data, the rating is limited to A.
Comparator Selection
Somapacitan was evaluated against somatropin and somatrogon, which are appropriate comparators for the intended population. The document indicates that somapacitan is offered to the same population as these treatments, supporting a rating of A+.
Patient Population and Subgroups
The patient population for somapacitan is well-defined, focusing on children aged 3 to 17 years with growth hormone deficiency. However, there is limited information on subgroup analyses, leading to a rating of A.
Care Pathway Integration
The document indicates that somapacitan can be integrated into existing treatment pathways without significant disruption, as it is used similarly to existing treatments. This supports a rating of A+.
Resource Use and Cost Implications
The document suggests that somapacitan is a cost-effective option compared to existing treatments, with manageable budget implications. However, it does not provide extensive details on broader resource use, leading to a rating of A.
Evidence Quality and Robustness
While the evidence base is supportive, it lacks multiple rigorous Phase III RCTs and relies on cost comparisons rather than robust clinical trial data. This leads to a rating of B++.
Uncertainty, Sensitivity, and Broader Impacts
The document indicates that somapacitan addresses a specific unmet need in the pediatric population, which mitigates some uncertainty. However, there are still some unknowns regarding long-term outcomes, leading to a rating of A.
