1. Special procedures - recap

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There are situations where the early, if not immediate, availability of a specific medicine outside the established (“normal”) regulatory procedures or even without a marketing authorisation (MA) is warranted. This is when the notion of ‘timely access’ might be expanded to include ‘early access’. Several regulatory mechanisms exist in the European Union (EU) pharmaceutical legislation to support patients' early access to (mostly) new and promising medicines, particularly those that target an unmet medical need or are of major public health interest.

They consist of provisions allowing shortening of the timeframe for obtaining marketing authorisations via special authorisation and support procedures, such as:

  • accelerated assessment procedure reducing the timeframe for review of a marketing authorisation application (MAA) from a maximum of 210 days to 150 days,
  • conditional marketing authorisation (MA) on the basis of less complete data than is normally the case, and subject to specific obligations for certain categories of medicines,
  • marketing authorisation under exceptional circumstances where the applicant is unable to provide comprehensive data on the efficacy and safety under normal conditions of use, because the condition to be treated is rare or because collection of full information is not possible or is unethical. This type of marketing authorisation is subject to specific procedures, in particular concerning the safety of the medicine,
  • the PRIME scheme to support development of medicinal products of major public health interest through early and enhanced scientific and regulatory dialogue, including scientific advice/protocol assistance

These special procedures have been dealt with in in lesson 2 (please refer to Lesson 2 for further information).