OBJECTIVES: We investigated using secondary research of the price erosion of non-oncological orphan drugs that had been made available in the last 5 years (2017 – 2022) and we determined the pricing evolution over time. We identified the evidence packages that were presented at the initial HTA assessment and any subsequent data packages and any changes to the HTA assessment (if it differed from the original assessment).

METHODS: We looked at the non-oncological products which had been available between 2017 – 2022 and is available in the EU5 and US. The criterion for selection is that the diseases cannot exclusively be targeted at children and adolescents and a manufacturer has not cordoned off the market. We looked at the evidence that was initially submitted and any additional evidence (via re-evaluation or submission of real-world evidence) and their initial HTAs and any use of MEAs had been documented and the pricing changes (if any) over this 5-year period.

RESULTS: A total of nine products had been investigated in our research and can conclude that price stabilisation is possible within both the US and EU5. For the US, the cumulative price changes of 15% or less and is usually below inflation increases (no correlation). HTA reports are important for pricing potential and re-evaluations are key to pricing erosion, however pricing erosion was minimal even after HTA re-assessments.

CONCLUSIONS: There seems to be a barrier to entry for treatments where the price is over €100K for treatments in Spain and Italy. Real World evidence has had minimal to no impact in terms of over-turning initial assessments and are mainly used as confirmatory results. Price erosion in the EU5 is minimal and majority of EU prices have not significantly eroded at the manufacturers selling price level.