Questions Pile up for Novartis as Senators Call on FDA to Take Action

The importance of having a robust investigation process in place supported by a clear SOP and structured guidance documents on how to conduct an investigation

Why wait three months to tell the US Food and Drug Administration (FDA) about manipulated data? Why wait two months between documenting an initial issue and opening a nonconformance report (NCR)? And how harshly will FDA act? These questions and more are piling up for Novartis, following the announcement on August 6, 2019 that FDA is investigating the company for manipulating data linked to its $2.1 million gene therapy Zolgensma (onasemnogene abeparvovec-xioi).

The arising issue with data manipulation submitted in the biologics license application (BLA) for Novartis’ gene therapy Zolgensma highlights for any sponsor the importance of having a robust investigation process in place. Conducting such an investigation should be supported by a clear SOP and structured guidance documents. Determining the root causes is the key step. Stating that “human error” or "lack of training" are the cause of the error is not considered as an adequate root cause by a regulator like FDA or MHRA. Based on the identified root causes, one then needs to define and describe the actions, including target completion dates and a responsible person assigned to oversee the successful closure of the CAPAs. When all breaches/issues related investigations are completed, i.e., the root cause of the breach/issue was established, CAPAs were defined and endorsed by Quality Assurance and notified to Competent Authorities. It is essential to confirm closure of the breach/issue. As a last step, the Subject Matter Expert responsible for the oversight on the investigation and CAPA processes ought to finalize the Investigation Report, ensure all signatures are present and documentation is archived properly. With this well-defined process one may avoid such inadequate conduct of an investigation.

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