OBJECTIVES: With value-based decision making growing as a key concept in pricing and access, this research explores the construction of a comparable quantitative representation of payer value perceptions in the context of orphan products. A second step looks at how this “value score” relates to pricing potential.

METHODS: Borrowing from multi-criteria decision analysis (MCDA) methodologies, we established a value scoring framework for the context of European HTA assessments (HAS France, NICE HST) and a selection of high-value non-oncology orphan products. Framework domains and related weights were created based on key clinical assessment parameters as described by HTA methodology guidelines, published literature and internal HTA expertise. The domain scoring was defined as a limited numerical range to allow to fit a diverse set of products and indications. We then used a linear regression model to investigate the relationship between value score and price, and further tested the approach through sensitivity analyses. HTA data of orphan products were collected from published HTA documents to provide scoring information. Treatment costs were calculated based on public list prices.

RESULTS: Depending on the healthcare system we found moderate to strong relationships between value and price, the best model fit with R² = 0.7368. However, in line with the small number of observations, models were highly sensitive to outliers, product addition or omission. Sensitivity analysis showed that certain domains alone “explained” the value-price relationship best (e.g. QALY score for NICE) and that there are additional external factors that may not be captured (e.g. disease prevalence).

CONCLUSIONS: The exploratory analysis provides insights into the link between value perception and price and provides an additional methodology for the price finding process. Going forward, the framework needs expert validation and further exploration in additional markets as well as a larger product n-size.