1. Non-standard marketing authorisations

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The development of new approaches to foster access to novel treatments for patients has continued since its earliest introduction and continues today. This should happen at the earliest appropriate time in the product life-span in a sustainable fashion. Regulators have been actively pursuing various approaches, recognising that patients and providers are willing to tolerate greater risks, especially risks of the unknown about medicines, when the morbidity of the disease is significant or when the disease is potentially life-threatening.

In the EU, mechanisms providing faster patient access to new medicines include ‘approval under exceptional circumstances’ (1993) and ‘conditional marketing authorisations’ (2005) for obtaining authorisations subject to certain annually reviewable conditions. Further, in order to meet, in particular, the legitimate expectations of patients and to take account of the increasingly rapid progress of science and therapies, ‘accelerated assessment procedures’ have been set up, reserved for medicines of major therapeutic interest.

A short recap: For an approved standard 'full' MA, all required quality, non-clinical and clinical data have been provided, and the benefit/risk profile is considered positive by the regulatory authorities, i.e., that quality, safety and efficacy of the product are established. Irrespective of that, the MA for the medicine may be granted subject to specific conditions (e.g., to conduct post-authorisation safety and/or efficacy studies[1], or the existence of an adequate pharmacovigilance system). A full MA is valid for five years and may then be renewed on the basis of a re-evaluation of the benefit/risk balance and shall then be valid for an unlimited period (with exceptions on grounds related to pharmacovigilance)[2].

Legal basis: Marketing authorisation subject to conditions Article 9.4 b, c, ca, cb, cc. of Regulation (EC) No 726/2004 and Article 21a of Directive 2001/83/EC

In the following the mentioned procedures for earlier access will be described in more detail.

[1] A post-authorisation efficacy study (PAES), imposed in accordance with Regulation (EC) No 357/2014, or a post-authorisation safety study (PASS) in accordance with Article 1(15) of Directive 2001/83/EC.

[2] Article 24 (1-3) of Directive 2001/83/EC and Article 14 (1-3) of Regulation (EC) No 726/2004