Germany Announces RWE Data Collection for Novartis Zolgensma Gene Therapy

February 5, 2021

Germany’s Federal Joint Committee (GBA, Gemeinsamer Bundesausschuss) has announced a new registry study related to spinal muscular atrophy (SMA) drugs. Zolgensma (onasemnogene abeparvovec) gene therapy from Novartis will be compared to Spinraza (nusinersen) from Biogen. Nearly 500 children with SMA (with or without symptoms) will be included in the study.

According to Josef Hecken, “Drugs are being approved for use earlier and earlier with weaker and weaker evidence. We have to try—especially with orphan drugs—to keep a balance between the need for urgently required treatment alternatives and good evidence on a drug’s long-term additional benefit.” Learn more here.

(Source: Neil Grubert, LinkedIn, 2/5/2021)


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