Comprehensive Clinical Evaluation of Nusinersen Based on Multi-criteria Decision Analysis Method

Objective This study aims to provide a theoretical basis for different drug decision-making scenarios by conducting a comprehensive clinical evaluation of nusinersen. Methods Based on the method of multi-criteria decision analysis, a comprehensive clinical evaluation index system of nusinersen was established, including core criterion model and contextualized criterion model, which 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 assigned weights and scores in various criteria of the core criterion model, an overall value of nusinersen was estimated after standardized and combined with the results in contextualized criterion model. Results 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 standardized. The most 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 (4.8±0.6), expert consensus or clinical practice guidelines (4.5 ±0.6), effectiveness (4.8±1.0) and quality of evidence (4.8±0.8). The holistic value of nusinersen was 0.41. Conclusion Nusinersen for spinal muscular atrophy treatment was considered as a treatment which adds value based on the method of multi-criteria decision analysis.