OBJECTIVE To actively promote the clinical access of nusinersen sodium injection and risdiplam powder for oral solution for the treatment of spinal muscular atrophy(SMA), a rare disease, and to promote the rational use of these two drugs in clinical practice.

METHODS By applying the "Guidelines for Drug Evaluation and Rapid Selection in Chinese Medical Institutions(Second Edition)", the effectiveness, pharmacological properties, safety, economy, and other attributes of nusinersen sodium injection and risdiplam powder for oral solution were quantitatively evaluated in 5 major components.

RESULTS After the evaluation of the optimized evaluation system, the scores of pharmacological properties of nusinersen sodium injection and risdiplam powder for oral solution were 24 and 22.8 points respectively; the scores of effectiveness were 25 and 23 points respectively; the scores of safety were 13 and 14.9 points respectively, the scores of economy were 13 and 12.67 points respectively; the scores of other attributes were 5.3 and 5 points respectively; and the total scores were 80.3, 78.37 points respectively.

CONCLUSION Risdiplam powder for oral solution is administered orally in powder form, and the dosage needs to be calculated strictly according to the body weight for daily administration. The economy is not as good as that of nusinersen sodium injection in terms of long-term medication. Risdiplam is marketed at a later stage, and the consensus of the recommended guideline is less than that of nusinersen sodium, and the level and strength of the evidence are lower than that of nusinersen sodium. The results of the evaluation provide valuable references for the access to healthcare organizations and the rational use of both drugs in clinical practice.