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EMA’s Adaptive Licensing Pilot Project Signals an Exciting Step for Medicines Development

Magda_Chlebus_EFPIA

The EMA’s announcement last week of an adaptive pathway pilot project with real medicines in development is a bold step in improving the way innovative and needed new therapies reach patients, and signals a new exciting direction for Europe. It stands in stark contrast to the US and UK which have been focused on accelerating the existing process of approving medicines via “breakthrough designations” which aim to dedicate greater focus and attention to areas of unmet medical need.

According Tufts CSDD Research Director Christopher-Paul Milne, “a key success factor for [FDA’s breakthrough] will be whether it serves … new development tools and methodologies, such as targeted diagnostics and adaptive clinical trial designs.[1]”  EMA has been the first to step-up with an actual adaptive pathway to test this approach in situ, placing Europe, EFPIA, and IMI2 at the centre of a potential global leap forward.

Medicine’s Adaptive Pathways to Patients (MAPPs) is an approach building on the advances in medical science, genomics, and personalised medicine to facilitate an approval process that adapts quickly to a given patient’s response to therapies. It will launch with a clearly defined patient population with unmet medical needs, followed by continued gathering of evidence in support of expanding the pool of recipients of the new therapy as the knowledge base of MAPPs grows.

Ultimately, MAPPs is about bringing better, needed new therapies to patients who need them. To quote Hans-Georg Eichler, the European Medicine Agency’s Senior Medical Officer, “The approach seeks to maximize the positive impact of new medicines on public health by balancing timely access for patients with the need to provide adequate evolving information on their benefits and risks.”[2]

While the EMA’s announcement is extremely exciting, there are several key challenges to be addressed before a new therapy can go live in this pilot.

In order for a MAPP to be implemented, all stakeholders must be aligned and agree on the evidence package for early approval and re-assessments at the design phase. This is a particular challenge in Europe, as it requires ‘buy-in’ from the member states and multiple Health Technology Assessment (HTA) authorities.

As well, reimbursement discussions are already taking many years in some countries, and simply moving more new medicines faster to a bottleneck at the member state level will ultimately not help patients. These however, are both problems that the EMA pilot can help solve.

EMA’s announcement is extremely timely and will enhance the ongoing international collaborations between EFPIA, EMA and MIT’s NewDigs research platform to test theoretical design models for MAPPs trials. The outcomes of this collaboration, when combined with the evidence generated by the EMA pilot, will contribute vital insights and findings that will inform the research proposals to be incorporated into IMI2 under Horizon 2020.

EPFIA will also be organising an international conference on new clinical trial models that will take place at the Royal College of Physicians in London on October 21, 2014. All of these activities will solidify Europe’s position as the global thought leader in adaptive pathways.

EMA has responded to the increasing speed of regulatory innovation and international competition by creating a unique opportunity for Europe to build on its success with programmes such as IMI with an adaptive pathway pilot. EFPIA will work closely with its members and stakeholders to harness this opportunity for the benefit of patients in need of improved therapies. It is, indeed, a very exciting challenge that industry and researchers will ultimately address and meet.

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Magda_Chlebus_EFPIA

EFPIA Science Policy Director

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