News on market exclusivity for orphan medicinal products
On 4 October 2024, the Court of Justice of the European Union (CJEU) ruled in a case concerning whether the concept of 'clinical superiority' - a prerequisite for an orphan medicinal product to qualify for the derogation from market exclusivity granted to another orphan drug - should be interpreted as strictly as the concept of 'significant benefit', assessed to determine whether an orphan medicinal product meets the criteria for the 'orphan' designation.
The concept of 'significant benefit' is a criterion for designating a medicinal product as an orphan medicinal product, allowing the medicinal product to benefit from a 10-year market exclusivity period. The 'clinical superiority' criterion is relevant when assessing whether a similar orphan medicinal product can be granted a derogation from the market exclusivity of the orphan medicinal product. Consequently, the interpretation of the concept of 'clinical superiority' may entail significant implications for pharmaceutical companies.
The case pertains to VVB UAB's application for marketing authorisation for the medicinal product Tobramycin VVB. As Tobramycin VVB was considered similar to an orphan medicinal product for the same therapeutic indication, namely Tobi Podhaler, VVB sought a derogation from the market exclusivity granted to Tobi Podhaler, claiming that Tobramycin VVB was clinically superior to Tobi Podhaler, given its greater safety. Following the issuance of a scientific opinion from the European Medicines Agency (EMA) supporting that Tobramycin VVB was clinically superior to Tobi Podhaler, the Commission granted Tobramycin VVB the marketing authorisation, thereby derogating from the market exclusivity granted to Tobi Podhaler.
The holder of the marketing authorisation for the original orphan medicinal product, Mylan, argued that 'clinical superiority' should have been assessed in line with the strict interpretation applicable to the concept of 'significant benefit', thus claiming that the assessment of 'clinical superiority' of a similar medicinal product must include an overall assessment of all the criteria relating to 'clinical superiority'.
In this ruling, the CJEU established that the concepts of 'significant benefit' and 'clinical superiority' are based on the same (non-cumulative) assessment criteria, namely greater efficiency, greater safety, or a major contribution to patient care, however, having different purposes and scope.
Moreover, the CJEU clarified that the 'clinical superiority' of a medicinal product is not assessed by means of an overall assessment of all the criteria provided for, but rather that it is sufficient for one of the three established criteria to be fulfilled for a second medicinal product to be considered 'clinically superior' to an existing orphan medicinal product. Thus, when assessing 'clinical superiority', the criterion of greater safety must be assessed individually rather than by establishing an overall benefit/risk balance for the entire population, as is otherwise required for 'significant benefit'.
In conclusion, the CJEU confirmed the reasoning and interpretation of the General Court and dismissed Mylan's appeal.
This judgment holds significant importance for pharmaceutical companies, as it provides clarity on the interpretation of the criteria required for a medicinal product to qualify as an orphan medicinal product when seeking to benefit from a market exclusivity derogation. While the interpretation of the 'clinical superiority' criterion potentially broadens the scope for competition, it also raises questions about whether it weakens the market exclusivity granted to orphan medicinal products.