Value-based price forecasting: an accurate method for orphan drugs?

AUTHOR(S) Anstee K , Jao R



HTA decision-making for orphan drugs is a challenge. Small population sizes, limited understanding of natural history of disease and lack of comparator within market may increase evidence uncertainty for novel orphan drugs.

Therefore, HTA agencies have adopted orphan-specific pathways or considerations.

  • In France, early access authorisation allows data collection for orphan drugs prior to EMA approval.
  • In the UK, the highly specialised technology appraisal route for ultra-rare conditions and the recent addition of the disease severity QALY modifier for standard technology appraisal account for the innovation of orphan drugs within the cost-effectiveness analysis.
  • In Germany, the G-BA automatically assume additional benefit of orphan drugs, requiring an abbreviated assessment and accounting for limited data certainty.

Traditional price forecasting methods incorporate price corridors benchmarked against comparators. However, as usually there is no appropriate comparator within rare diseases, forecasting price and payer-perceived value of novel orphan products is a challenge.

Therefore, for orphan drugs, an alternative forecasting method incorporating nuances of each payer within value-based pricing may be more appropriate.


This research aimed to determine accuracy of using a value-price model to forecast price of an orphan product across different payer archetypes.

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