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We supported a mid-size company to assess the opportunity for a gene therapy platform they were planning on in-licensing​

Client Challenge

Understand the pricing, access ​and uptake potential of a potential gene therapy platform that the client wished to in-license ​

The pharma company had identified a autologous gene therapy platform ​ being developed in multiple orphan ​ indications as a target for in-licensing

​Although all indications were rare, each had different landscapes and with different patient pathways. While the pivotal clinical trials were completed for 1 indication, they were still ongoing for the other planned indications

P4A Solution

P4A used a combination of secondary research, primary research validation and supported the revenue forecasting exercise to inform the go-/no-go decision​

P4A’s approach required inputs from various internal and external stakeholders:​
  • Secondary research to develop disease landscapes​
  • Primary research with payers and KOLs to ​understand the pricing, access and uptake potential​
  • P4A developed an opportunity optimisation plan and worked with the client to inform the forecast model​
  • Workshop to align on go-/no-go decision ​

The Successful Outcome

  • Understood the P&R, patient access and competitive landscape for the product in each indication​
  • Provided list & net pricing potential, levels of access restrictions, peak patient share and the shape of the uptake curve that fed in to the forecasting model​
  • Identified impact of label expansion and highlighted the need for changes in the launch sequence to optimise revenue​
  • Identified evidence and market shaping mitigation measures to further optimise the P&R / commercial potential of the product​
P4A not only told us what the opportunity is today, but also what we should do between now and launch in 2025 to improve revenues​ ​
Franchise Lead for Regenerative Medicine unit​
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We are award winning global experts in access for orphan drugs and Cell and Gene Therapies.

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