This article is part of a series that covers the proposed reforms to EU pharmaceutical legislation. To view our first article and the whole series, please click here.

As part of the revision of the EU pharmaceutical legislative framework, a new regulation is proposed, which will replace, among others, Regulation (EU) 141/2000 on orphan medicinal products. According to the European Commission, the current legislation on orphan medicinal products no longer meets the needs of both patients and the pharmaceutical sector, with 95% of the 6000+ recognised rare diseases having no treatment option[1]. Below we set out the main changes in the proposed regulation.

Regarding the criteria for orphan designations, the prevalence criterion (not more than five affected persons per 10.000) is maintained as the appropriate threshold under the proposed directive. However, as this criterion is not appropriate for all rare diseases, the European Commission will be able to set up specific designation criteria for certain conditions with, for example, a short duration and high mortality rate. The additional “no satisfactory method” (or alternatively establishing “significant benefit” over existing methods) criterion also remains in place. The previous alternative criterion that medicinal products will likely not generate a sufficient return on investment in order to qualify for an orphan designation has been omitted from the proposed directive. Another change is that the current unlimited validity of the designation is proposed to be limited to 7 years (with the possibility of an extension).

The European Commission aims to adopt a new framework for market exclusivity of variable duration, replacing the (in principle) 10-year market exclusivity for orphan medicinal products. According to proposed article 71(2)(a), the market exclusivity for orphan medicinal products will last for nine years in principle. However, orphan medicinal products addressing a high unmet medical need will have market exclusivity for 10 years (article 71(2)(b)) and orphan medicinal products which have been authorised based on bibliographic data according to article 13 of the proposed new regulation will have a 5-year market exclusivity (article 71(2)c)). For new therapeutic indications of orphan medicinal products referred to in 71(2)(a) and (b), the market exclusivity can be prolonged by twelve months for each of the first two new indications in different orphan conditions; these new indications must be authorised at least 2 years before the end of market exclusivity. The market exclusivity can also be extended for a further year based on meeting certain supply obligations in all Member States. The criteria to derogate from market exclusivity remain the same as in the current legislation. Where a marketing authorisation holder holds more than one orphan marketing authorisation for the same active substance, these authorisations will not benefit from separate market exclusivity periods.

The European Commission’s aim is that the new market exclusivity framework will improve competitiveness and reduce prices, offering manufacturers of generics and biosimilars opportunities for faster market entry for their products. The proposed regulation attempts to achieve this with article 71(6), which ensures that the application for a marketing authorisation for similar medicinal products is not prevented by market exclusivity of orphan medicines, if the remainder of this market exclusivity is less than two years.

According to article 73 of the proposed regulation, the working arrangements referred to in the new fee regulation will set out total or partial reductions for EMA fees related to orphan medicinal products. Other support for orphan medicinal products development, by means of scientific and regulatory advice, will be further strengthened under the proposed regulation. According to article 60(1)(b), the EMA can offer enhanced scientific and regulatory support for orphan medicinal products which are likely to address a high unmet medical need.

[1] Impact assessment report accompanying the revision of the medicines for rare diseases and children legislation, SWD – IA – OP revision_RSBv 27102022 (europa.eu).