Our Impact

Understanding the data case study

How an interactive gene therapy budget impact model produced greater internal understanding and facilitated communication with external stakeholders

The Challenge

Inbeeo’s client, a local affiliate of a major global pharmaceutical company, is developing several gene therapy (GTx) products.

In advance of their launch and P&R negotiations, the affiliate wanted to be able to ease payer concerns about the potential future GTx market size in order to strengthen their negotiating position.

The number of GTx products in clinical development is rapidly rising, with >1000 trials listed on However, with few having been approved to date, there is significant uncertainty in many areas, such as the success rate of clinical development, the level of uptake, and the future price landscape – a significant challenge to developing a robust forecast.

The Approach

From a starting point of GTx trials on, a list of distinct GTx products in development was created. Epidemiological data about the indications covered by these was found to estimate patient numbers. To create the budget impact model, three further calculations were made:

  • Probability of success based on developmental phase
  • Price estimates based on existing products and future expectations
  • Adoption rate estimates

The research was brought together in an interactive model that included various different scenarios aligned to our best assumptions.

The Impact

Inbeeo’s interactive GTx budget impact model was a key resource for the client to understand the evolution of the GTx P&R landscape, and helped them formulate a communication strategy for national/regional stakeholders about their upcoming GTx products.

Key findings were presented at ISPOR Vienna 2022.