Case Study: Global Pricing & Market Access Strategy for a Rare Disease Gene Therapy

Project Objective

  • A biopharmaceutical company was developing the first gene therapy for a rare disease. It was expected to be a one-time therapy that would replace the current chronic standard of care

  • As gene therapies are typically very expensive, the client was aware that payers may have significant affordability concerns, particularly if a single up-front payment was required

  • Therefore, the client asked Windrose to evaluate the potential price and market access opportunities for the gene therapy, and to explore whether payer willingness-to-pay would differ if alternative payment arrangements such as annuity models were considered

Windrose Approach

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Gene Therapy Value-Based Price Construct

Gene Therapy Value-Based Price Construct

Price Testing Methodology

Price Testing Methodology

  • First, we determined all of the potential value drivers for the gene therapy, based on the economic, clinical and humanistic benefits relative to standard of care; this allows us to conduct  a calculation of the cost savings and quality of life gains to directionally determine the “value-based price” of the gene therapy

  • Next, we conducted primary payer and key opinion leader research across the US and eight ex-US markets to assess price, access and reimbursement potential for the gene therapy, as well as likely product positioning

  • As part of this, we used a robust pricing framework to evaluate affordability of the gene therapy, including considering how alternative payment models or access strategies would affect price potential

Impact

  • We established both the value-based price and payer willingness-to-pay for the gene therapy across markets

  • We provided recommendations to the client to optimize price and access for the gene therapy, including how payment models (up-front vs. annuity, vs. performance-based) would affect price potential

  • The final recommendations were well received by the client and helped them form a strategy to support product launch across markets

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Case Study: Framework and Policy Development

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Case Study: Global Pricing Strategy for a New Vaccine