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Development and validation of an orphan drug’s payer value story for a form of dysmorphic short stature


A mid-sized biotech client sought to develop and validate a payer value proposition for a first-in-class drug indicated for a rare form of dysmorphic short stature.

RJW&partners developed a payer value proposition then tested the messages, evidence, and pricing potential with payers and KOLs in the EU5, Sweden and the Netherlands.

Payer feedback was analysed to inform refinements to the value proposition, define the drug’s price potential across different evidence scenarios, and inform the structure of the global value dossier.

“The Payer Value Story that you put together has really helped advance our thinking.”

Head of Market Access, EUMEA


Our client needed to develop a payer value proposition that was credible and meaningful to payers. There was a risk that payers perceived short stature as a cosmetic issue and could benchmark product pricing against low-cost growth hormone.

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RJW&partners helped develop a robust and credible payer value proposition for the drug.

Key aspects of the disease burden and drug’s benefits were captured in value messages supported by trial data and effectiveness modelling.

These messages were subsequently tested with payers and KOLs in a virtual payer advisory board that also evaluated the drug’s price potential across different evidence scenarios.

Following the advisory board, refinements were made to the value proposition and recommendations shared for the global value dossier structure.


RJW&partners delivered a compelling payer value proposition that clearly articulated the burden of disease and the drug’s key benefits.

Payer and KOL feedback from a virtual payer advisory board was captured and used to inform final refinements to the messages and supporting evidence.

We also provided recommendations on the structure of the global value dossier to ensure that evidence from trials, non-interventional studies, and modelling analyses were relevant and meaningful.

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