Orphan drugs market access landscape has changed rapidly over recent years. This research aims to provide an update on OD pricing environment between US and Europe, and investigate differences, influencing factors and future trends.

METHODS:

Recent OD analogues were assessed covering different therapy areas. Market-specific pricing websites (EU5 and US) and reports from HTA bodies were analysed to understand their price level and reimbursement decisions. Payers (1-2 per market) were interviewed to validate the insights from secondary research.

RESULTS:

Price differences of ODs between US and Europe vary, depending on disease / product-related factors and market dynamics. Not all ODs have higher price in US compared to Europe. Some of the ODs (e.g. Vimizim) have similar price in Europe as US, while for other ODs, the price differences are 2-3 folds (e.g. Opsumit). Even within Europe, the price of ODs is not uniform. Several factors impact the price differences of ODs between US and Europe. For ODs with substantial benefits esp. in debilitating diseases with no/limited treatment options currently, their prices tend to be similar between US and Europe. But for ODs with no/small incremental benefits where other treatment options already exist, their prices tend to be lower in Europe vs. US. In the future, ODs will face increasing scrutiny from payers and discussions already started in Europe to re-examine the pathways for ODs. In the US, mixed messages have been given post presidential election, raising further uncertainties for OD pricing in the US.

CONCLUSIONS:

There will be increasing demand on ODs to justify their prices as healthcare systems are under increasing budget pressure. Flexible pricing with innovative contracting agreements / managed entry agreements will become increasingly important for ODs to address data uncertainties and support access in Europe and US.