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We identified optimal cost-efficient routes to market for an autologous GT for an ultra-rare indication ​

Client Challenge

Goal was to find an optimal ​route-to-market for Europe for an GT targeting an ultra-rare condition​

Gene therapy launch for a biotech ​client planned for 2021 in Europe

The autologous GT targeted a small ​patient population with a prevalence ​< 100 patients in Europe

Client was financially and personnel constrained, but were mandated to commercialise in-house by investors​

P4A Solution

P4A reviewed the data and ​recommended the biotech ​implemented the following: ​

  • Pathway Mapping​
  • Payer & KOL research to ​identify pathway barriers​
  • NPV modelling of ​different launch options​
  • Follow-up recommendations ​ ​

The Successful Outcome

  • Identified paid for named patient access routes, including import pathways or cross-border pathways in all EU markets ​
  • Defined treatment site selection strategy and shortlist of potential EU centres​
  • Formulated the materials and services that support physicians in making a request to access treatment for their named patient ​
P4A found us a solution that enables EU ​ patients to access our gene therapy ​ without breaking the bank ​
Chief​ Commercial​ Officer​
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We are award winning global experts in access for orphan drugs and Cell and Gene Therapies.

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