OBJECTIVES:

Amidst global concern over escalating healthcare costs, pharmaceutical costs are being further scrutinized. Notably, expansion of orphan drug approvals have intensified this discussion. Different markets have adopted distinct strategies to manage and regulate these pharmaceutical expenses. This analysis endeavors to assess the divergence in launch prices of orphan drugs between the United States and EU4 (Germany, France, Italy, Spain) markets to understand the impact of each market’s approach to managing pharmaceutical costs.

METHODS:

Historical wholesale acquisition cost (WAC) pricing at launch of non-oncology orphan drugs in US and EU4 markets from 2015-2022 was evaluated utilizing POLI, a pricing database. The analysis compared WAC prices per pack across markets, employing consistent dosing assumptions. To ensure a meaningful evaluation of comparable costs, adjustments were made for variations in pack size, currency exchange rates, and German free pricing period. Products launched in a single market were excluded from the dataset. Data was then evaluated to discern trends in launch pricing.

RESULTS:

47 orphan launches met the outlined criteria. From 2015-2022, orphan drug launches in EU4 markets were introduced at 36.2% of the launch price observed in US. Notably, in Germany, France, and Italy, orphan drug launches exhibit a high degree of comparability, ranging from 36.1% to 37.1% of corresponding US launch price, whereas in Spain, orphan product launches occurred at lower 24.4% of US launch price. Over time, there was no discernible trends in launch prices.

CONCLUSIONS:

Differences in orphan drug launch prices from US and EU4 markets prompts discussion on factors shaping the orphan pricing landscape. Consistent pricing in Germany, France, and Italy may reflect common regulatory frameworks, healthcare policies, or market competition dynamics that foster an aligned market response compared to US.