EMA Revises Accelerated Approval Guidelines

From DCAT Value Chain Insights (VCI)

By Regulatory News posted 07-29-2015 06:46


The European Medicines Agency (EMA) has revised its guidelines on the implementation of accelerated assessment and conditional marketing authorization, two key tools in the European legislation to accelerate access to medicines that address unmet medical needs. Accelerated assessment and conditional marketing authorization 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. The public consultations on the revised guidelines are open until September 30, 2015. Comments should be sent using the forms provided.

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

The 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 fullfilment of major public health interest, which is the basis for a request for an accelerated assessment; optimization of the assessment timetable by better balancing evaluation phases to reach a Committee for Medicinal Products for Human Use (CHMP) opinion within the 150 days after the start of a marketing authorization application procedure (compared to 210 days in non-accelerated procedures); and emphasis on the importance of early dialogue with EMA so that accelerated assessment can be planned well ahead of the submission. The 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.

The guidelines also specify information on conditional marketing authorization. Conditional marketing authorization 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 authorization within a time frame 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 authorization, according to the EMA. The revised guideline emphasize the importance for medicine developers of planning a conditional marketing authorization prospectively and engaging in early dialogue with the EMA and other stakeholders, for example through parallel scientific advice with health technology assessment bodies.

In addition, the revisions include: clarification on fulfillment 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 authorization and the data that can be provided after authorization; 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 provided here.

Source: European Medicines Agency


Thank you for reading DCAT Value Chain Insights (VCI), an information resource from the
Drug, Chemical & Associated Technologies Association (DCAT). 

Learn more and sign up to receive a complimentary subscription