The company was seeking to understand the current clinical landscape and patient journey, identify key hurdles for uptake of a new gene therapy, and to identify the clinical value drivers that would define an innovative agent to treat Fabry disease that would drive product utilization. To achieve this objective, Certara conducted an assessment of a gene therapy for Fabry disease in the United States, United Kingdom, France and Germany. In addition, they assessed a gene therapy for phenylketonuria in the US and EU3 which identified

  • the current clinical and market access landscapes for PKU
  • unmet needs and value drivers for a new gene therapy from the perspective of KOLs and payers
  • the ability to test price using multiple methodologies, identifying clinical, patient reported outcomes (PROs), and economic data that will enhance the perceived value of the product
  • recommendations on the clinical development and value and access strategies in each market
Certara Case Study evolving pricing and reimbursement payer landscape 1

Certara conducted an assessment of a gene therapy for Fabry disease in the United States, United Kingdom, France and Germany.

According to NORD (National Organization for Rare Disorders), Fabry disease is a rare inherited disorder of glycosphingolipid (fat) metabolism resulting from the absent or markedly deficient activity of the lysosomal enzyme, α-galactosidase A (α-Gal A). This disorder belongs to a group of diseases known as lysosomal storage disorders.

Certara Case Study evolving pricing and reimbursement payer landscape 2

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