OBJECTIVES :

Duchenne muscular dystrophy (DMD) causes progressive muscle damage from birth resulting in loss of ambulation, pulmonary and cardiac decline, and premature death typically before age 30. Until recently, an urgent unmet need remained for disease modifying treatments; however, in 2016 eteplirsen was approved via the US Food and Drug Administration (FDA) Accelerated Approval (AA) pathway for patients with DMD with a mutation amenable to exon 51 skipping. AA programs allow faster approval of drugs that treat serious conditions and fill an unmet medical need based on a surrogate endpoint. The availability of only immature clinical data at approval, however, can cause reluctance to cover by payers and result in restricted access. The objective of this study was to examine the published evidence for eteplirsen pre- and post-approval, identify key barriers to access, and evaluate current payer restrictions.

METHODS

Data for eteplirsen pre-approval were obtained from the Prescribing Information, with post-approval data from a recently conducted systematic literature review. Payer restrictions were identified via database searches.

RESULTS

Eteplirsen was approved based on dystrophin expression data; post-approval data (including data in non-ambulatory patients) demonstrate considerable differences in ambulation and pulmonary function compared with natural history controls. Coverage restrictions ranged from not covered, to ambulatory patients only with initial authorisation for 8 weeks to 6 months. Key reasons for coverage restrictions were a lack of high-quality evidence and scepticism around the available evidence.

CONCLUSIONS :

AA allowed faster regulatory approval for eteplirsen; however, limited data at approval due to the nature of the AA process have resulted in coverage restrictions, which indicate that post-approval data may not always have been considered. Evidence generation for eteplirsen must continue and expand post-approval to provide the information required by payers to make appropriate and informed reimbursement decisions to address the urgent unmet need in DMD.