Orphan Drugs – How Will the New Proposed European Union (EU) Commission Pharmaceutical Regulation Impact Therapies for Rare Diseases?

OBJECTIVES: We aim to analyse the implications of the new proposed legislation and its impact on orphan drugs (OD) and to compare these changes to the existing OD landscape.

METHODS: We are undertaking a comprehensive assessment of the pharmaceutical legislation within EU. Using Darzalex® as an analogue, we compared the new legislation vs. the current orphan environment, in particular, the impact of the shortened exclusivity period on OD access in the EU.

RESULTS: The exclusivity period for Darzalex would be shorter vs. current legislation, that in addition to the initial 9-10-year exclusivity period allows up to two 1-year extensions instead of full exclusivity for new indications. There is the possibility of achieving an additional 1 year of exclusivity for a product being released and continuously supplied within 2 years of marketing authorisation. However, based on our case study, it is not feasible to guarantee pricing and reimbursement within the 2-year timeframe in all Member States, unless the establishment of a Named Patient Programme (NPP) or compassionate use programme is deemed sufficient to meet this requirement.

CONCLUSIONS: Orphan designation cannot exceed 13 years from the initial marketing authorisation as stipulated within the new legislation. Our analogue of Darzalex® demonstrates that the orphan designation favours generics/biosimilars where extensions are only for 1 year vs. 7 years currently. The new legislation may encourage manufacturers to think twice before launching in Europe.