The CJEU on "Significant Benefit" vs. "Clinical Superiority"

In its October 2024 ruling, the Court of Justice of the European Union (CJEU) clarified the criteria for when a similar medicinal product can demonstrate "clinical superiority" over an orphan drug, thus qualifying for a market exclusivity exception under EU Regulation.

• Background on Orphan Medicinal Products (OMPs):
  

OMPs are developed for rare diseases with limited commercial potential. The EU provides incentives, including 10-year market exclusivity, to encourage OMP development. However, this exclusivity can be waived if another product is deemed safer, more effective, or "clinically superior".

• Key Concepts Defined:
• Significant Benefit: A prerequisite for OMP designation, which requires a product to show greater safety, efficacy, or care improvement compared to authorized treatments for the entire patient population.
• Clinical Superiority: A criterion for waiving exclusivity, applicable when a similar product proves greater efficacy or safety in a significant portion of the target population.

• The Case at Hand: The dispute involved two cystic fibrosis treatments: "Tobi Podhaler" (an inhalation powder with OMP status) and "Tobramycin VVB" (a nebulizer generic version). The European Commission (EU)granted Tobramycin VVB a derogation based on clinical superiority, as it reportedly caused less coughing in cystic fibrosis patients.
  
  Mylan, holder of the Tobi Podhaler MA, challenged this decision, arguing that "significant benefit" should protect its product's exclusivity against generics. The CJEU clarified that significant benefit and clinical superiority serve different purposes: significant benefit justifies OMP designation, while clinical superiority allows similar products to enter the market if they provide advantages for patient subgroups.

The CJEU’s decision reinforces that exclusivity for orphan drugs isn’t absolute, and patients' access to superior treatments remains a priority. This ruling may guide future regulatory interpretations concerning the availability and classification of OMPs in the EU.