1. Pink Sheet

Patient Advocacy With FDA Review Staff Will Be Tougher Post-Sarepta

 
• By Cole Werble

Efforts by CDER management to encourage more interaction between patients and FDA reviewers may be part of the collateral damage from the difficult FDA review of Sarepta’s Exondys 51. The hopes of parents of boys with DMD pushed the regulatory flexibility by CDER management on this application – but may end up limiting the willingness of FDA reviewers to engage with patients in the future.

FDA entrance sign 2016

When FDA embarked on a more aggressive reaching out to patient advocacy groups in 2012, one of the frequently stated long-term objectives was to find a way to bring patients and the researchers involved with them into more direct contact with FDA drug reviewers.

The broad patient-focused scheduled meetings on diseases selected by FDA review divisions were intended as just one part of what was seen as an eventual effort to provide the resources of patients and

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