EMA publishes new draft guidance on fast track routes to marketing authorisation

The European Medicines Agency (EMA) has announced the launch of two-month public consultations on revised guidelines on accelerated assessment and conditional marketing authorisation, which are fast track routes for medicines that address unmet medical needs. The public consultations on the revised guidelines are open until 30 September 2015. Details on how to send comments are provided by EMA on its website.
Accelerated assessment and conditional marketing authorisation are intended for innovative medicines that target a disease for which no treatment is available, or that provide patients with a major therapeutic advantage over existing treatments.

Based on the experience gained in implementing accelerated assessment and conditional marketing authorisation in recent years and taking into account discussions on the optimisation of the use of these tools at the European Commission Expert Group on Safe and Timely Access to Medicines for Patients (STAMP), EMA has revised its guidelines to improve these existing frameworks. The updated guidelines are expected to optimise the use of these tools by medicine developers and consequently allow more medicines that address unmet medical needs to reach patients earlier.

Accelerated assessment

EMA’s accelerated assessment procedure allows for a faster assessment of eligible medicines by EMA’s scientific committees. The main changes included in the proposed revision of the guideline are:

  • more detailed guidance on how to justify fulfilment of major public health interest, which is the basis for a request for an accelerated assessment
  • optimisation of the assessment timetable by better balancing evaluation phases to reach a CHMP opinion within the 150 days after the start of a marketing authorisation application procedure (compared to 210 days in non-accelerated procedures)
  • emphasis on the importance of early dialogue with EMA so that accelerated assessment can be planned well ahead of the submission.

EMA highlights that the eligibility criteria laid down in the accelerated assessment guideline are also being considered for a new scheme, currently under development, that is designed to facilitate the development and accelerated assessment of innovative medicines of major public health interest, in particular from the viewpoint of therapeutic innovation.

Conditional marketing authorisation

Conditional marketing authorisation allows for the early approval of a medicine on the basis of less complete clinical data than normally required, if the medicine addresses an unmet medical need and targets a seriously debilitating or life-threatening disease, a rare disease or is intended for use in emergency situations in response to a public health threat.
While less complete, the available data must still demonstrate that the medicine’s benefits outweigh its risks and the applicant should be in a position to provide the comprehensive clinical data after authorisation within a timeframe agreed with the CHMP. In addition, the benefit to public health must outweigh the risk due to the limited availability of clinical data at the time of marketing authorisation. The revised guideline emphasises the importance for medicine developers of planning a conditional marketing authorisation prospectively and engaging in early dialogue with EMA and other stakeholders, for example through parallel scientific advice with health technology assessment bodies. This is expected to help translate conditional marketing authorisations into early access to medicines for patients.
In addition, the revisions include:

  • clarification on fulfilment of unmet medical needs, i.e. medicines providing major improvements in patient care over existing therapies can be eligible in certain cases
  • clarification of how a positive benefit-risk balance is to be substantiated where there are less complete data, with further guidance on the level of evidence that must be provided at the time of authorisation and the data that can be provided after authorisation
  • updated guidance on the extent and type of data required to be included in annual renewal submissions.

An overview of the proposed changes to the two guidelines is available on the EMA website.

Note that accelerated assessment and conditional marketing authorisation are tools used in the EMA’s Adaptive Pathways scheme, which is currently in development.

Author: Anthony Lodge

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