The Iterative Development of Medicines Through the European Medicine Agency's Adaptive Pathway Approach.

Giuseppe Nicotera,Gianluca Sferrazza,Pasquale Pierimarchi,Annalucia Serafino

doi:10.3389/fmed.2019.00148

Giuseppe Nicotera, Gianluca Sferrazza + Show 2 more

Open Access

https://doi.org/10.3389/fmed.2019.00148

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Abstract

The development of novel regulatory tools such as adaptive clinical trial design and utilization of real-world evidence are topics of high interest. Recently, the European Medicines Agency (EMA) introduced the Adaptive Pathways (AP) that represents an innovative tool in healthcare systems allowing the early dialogue with multiple stakeholders on promising and innovative medicinal products in areas with an high unmet medical need. The innovative aspect in the AP is the early involvement of several stakeholders such as pharmaceutical industry, the Academia, Health Technology Assessment (HTA) bodies, and patient representatives bringing their real experience with the disease and their expectations about the treatment. AP is not a new licensing tool but an opportunity for a very early discussions, before starting the phase II studies, among all stakeholders, including regulators, companies, HTA bodies, and patient representatives on a new potential medicine in areas of high unmet medical need. The aim of this paper is to describe the evolution of the AP approach from the beginning of the pilot project to date, highlighting major advances, and achievement at European level.

Highlights

The continuous progress in sciences and technologies has the potential to bring a wide range of beneficial therapies to patients over the coming years
The use of early access tools has proved to be inefficient in all those cases in which requests for access to these tools were made too late when the margin for using corrective measures was impossible or reduced to a minimum
Finding a balance between early access and the need to obtain certain data requires a greater effort on the part of the regulatory authorities or greater post-marketing surveillance and a continuous assessment of the risk-benefit ratio of the product during the various phases of experimentation clinic [34, 35]

Summary

Introduction

The continuous progress in sciences and technologies has the potential to bring a wide range of beneficial therapies to patients over the coming years. These include personalized and precision medicine and advanced therapies that will require new modalities of evaluation and new ways of managing them in clinical practice. Between the multiple roles of the EMA one of the most important is the coordination and the cooperation with the medicines regulatory authorities at national levels, in order to facilitate a dialogue and a wider exchange of information, strengthening the quality, safety, and efficacy of medicinal products globally. One of the main goals for all the Regulatory Medicine Agencies is to counterbalance the need to have timely access to promising medicines for all patients, without compromising patients’ safety.

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