AMCP dossier for formulary submission for a gene therapy

Our client required the drafting of an ‘unapproved’ product AMCP dossier to inform healthcare decision makers in the US, later to be updated as an ‘approved’ dossier once data was available. The dossier needed to communicate the clinical and economic benefit of the gene therapy for a rare genetic neurological condition.

An unapproved dossier was developed in accordance with the AMCP guidance for dossier development, including information from the client’s GVD and recent submission documents. Special attention was given to the structure, referencing, and accuracy of the statements and claims presented.

The production of an AMCP dossier enabled our client to have early stakeholder engagement, with the opportunity to gather valuable feedback on the evidence, positioning, and data requirements from healthcare decision makers. The dossier enabled payers to familiarise themselves with the product early for future formulary decisions and helped establish the product’s value and competitive positioning.