To provide a theoretical basis for different drug decision-making scenarios by conducting a comprehensive clinical evaluation of nusinersen.

Based on the method of multi-criteria decision analysis, a comprehensive clinical evaluation index system of nusinersen was established. This system, including core criterion model and contextualized criterion model, covered multiple evaluation dimensions such as safety, effectiveness, economy, and social attributes. The evidence of nusinersen in various criteria was summarized and integrated through systematic reviews. Then, different stakeholders were assigned weights and scores in various criteria of the core criterion model. Finally, a holistic value of nusinersen was estimatedthrough standardizing and combining the results in contextualized criterion model.

The criteria, type of therapeutic benefit disease severity (0.08±0.02), unmet needs (0.08±0.01), quality of evidence (0.08±0.01), effectiveness (0.08±0.01) and safety (0.08±0.02) received the greatest weights after standardization. The higher mean scores for nusinersen versus placebo for the treatment of spinal muscular atrophy were disease severity (4.8±0.4), innovation of the drug (4.7±0.6), expert consensus or clinical practice guidelines (4.5±0.6), effectiveness (4.0±1.0) and quality of evidence (4.0±0.8).The holistic value of nusinersen was 0.41. The overall impact of the contextualized model on the evaluation of the drug was 0.26, indicating that it may overestimate the comprehensive value of nusinersen.

The method of multi-criteria decision analysis proves the positive role that nusinersen plays in spinal muscular atrophy treatment.