Commentary highlights insurance barriers to pediatric gene therapy access

A commentary in Pediatric Research examines insurance coverage as a barrier to gene therapy access for children with rare monogenic diseases. Discussing advances in gene editing and cell and vector engineering, the authors argue that access to existing treatments—and future development—depends on U.S. insurers’ coverage and reimbursement decisions. The article reports no new research, patient cohort, or clinical endpoints.

The commentary cites a $4.25 million list price for atidarsagene autotemcel (Lenmeldy), a one-time treatment for metachromatic leukodystrophy, and approximately $2.1 million for onasemnogene abeparvovec (Zolgensma), used for spinal muscular atrophy. It also identifies high prices for the sickle cell disease therapies exagamglogene autotemcel (Casgevy) and lovotibeglogene autotemcel (Lyfgenia). The authors state that independent economic assessments have judged gene therapies cost-effective when their potential lifetime benefits are compared with prolonged standard care.

For clinicians, the commentary underscores the distinction between regulatory approval and practical treatment access. Its economic arguments are not new evidence of treatment effectiveness or durability. Only a subscription preview was available in the supplied text, limiting assessment of the full policy proposals. The authors declare no competing interests.