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EMA on Regulatory Science to 2025

Cooperation, Integration, Evidence, Access

Thomas Kühler
Head, Regulatory Science & Policy, EU/AMESA
Sanofi R&D

n January 2019, the European Medicines Agency (EMA) published the Regulatory Science to 2025: strategic reflection paper for public consultation. It comprises sections on both human and veterinary medicines and discusses the importance of international regulatory science cooperation. In this reflection paper, EMA stresses the need to foster closer integration of science and technology in medicines development and to establish improved evidence generation paradigms to support different stakeholders involved in the assessment and decision-making of new medicines. It also underscores measures to secure patient access to medicines and debates the readiness of the European regulatory system to adequately respond to emerging health threats.

The reflection paper will serve as an important cornerstone to keep the European regulatory system competitive and flexible to change. However, there are couple of areas where, I believe, it could have been more explicit or progressive.

Firstly, the digital revolution in the pharmaceutical sector has begun and will profoundly change the drug development value chain. Evidence generated from sources other than randomized control trials will (and must) become routinely accepted in decision making–not only by the regulators but also by downstream players. The skill set required by the profession to demonstrate the value of a new treatment will change and thus new competencies will be required. Affordability and budget constraints will continue to shift power from regulatory towards HTA and payers as the debate on pricing and affordability will continue.

Furthermore, the rapid adoption of new technologies could accelerate the regulatory licensing process and facilitate the regulatory surveillance of marketed medicinal products. Regulatory documentation– data that are updated in real time as opposed to a static dossier–could reside in a single cloud-based location and be accessed and evaluated by regulators simultaneously around the globe. Outcomes, issues, and decisions could be debated jointly and precious time could be saved for the benefit of all stakeholders involved whether it is society as a whole, individual patients, regulators, payers, or industry.

Second, research in Advanced Therapeutic Medicinal Products, or ATMPs, which comprises gene therapy, gene-modified cell therapy, cell therapy, and tissue engineering, is surging. At the end of 2018, there were more than 1,000 trials ongoing with a total targeted enrollment of close to 60,000 patients worldwide. Current regulatory procedures in the EU were put in place long before ATMPs were conceived and available procedures are not necessarily optimized for where science has taken us today. Thus, there is an imperative to address this issue by adapting fast and efficient regulatory pathways.

In the end, it is not industry nor regulators who lose out if flexible, agile, and swift procedures capable of assessing these and other novel types of products are not available; rather, it is society and patients who are unable to access safe and efficacious products on time. European regulatory procedures are sturdy and robust but may lack some of the speed and flexibility required to become the gold standard.

A hard Brexit was recently avoided as the UK and the EU agreed to extend the timelines for Article 50. Through this Article, any EU Member State has the right to unilaterally leave the Union–a right which the UK government exercised in 2016–and comes with a two-year period during which the exit deal (or the Withdrawal Agreement) is to be negotiated. The new date for the UK to leave the Union is now set to 31 October 2019 and therefore provides the UK government additional time to vote the Withdrawal Agreement through Parliament. The current Withdrawal Agreement is not up for renegotiation and talks on what the future relationship with the Union may look like cannot take place during this period.

The only thing we know for sure is that the final word on Brexit has not yet been said. The pharmaceutical community must therefore continue to come together in order for us to collectively prepare for whatever the future may hold.

A Personal Note

When I graduated as a chemical engineer many years ago, little did I know that I would venture into organic chemistry, then medicinal chemistry, to eventually become involved in regulatory affairs. Throughout this career meandering, I have met many inspirational people across various disciplines, including Alberto Grignolo at PAREXEL International, current Editor-in-Chief of DIA Global Forum.

Alberto and I live on different continents, but made the most out of the recent DIA Europe 2019 meeting in Vienna, which we both attended, and met over a pleasant dinner to catch up on various aspects of our profession. Towards the end of the dinner, we came to talk about DIA and Global Forum in particular. He mentioned that there was a vacancy on its Editorial Board and that he was looking for someone to cover the European region, particularly one that could put into perspective recent changes and present a future outlook.

I decided in a split second this was definitely an opportunity that I could not pass up. Indeed, it is with great honor that I join the Editorial Board of DIA Global Forum and assume the role of Co-Editor for Europe. I am thankful for this responsibility and feel both humbled and privileged for the opportunity to give something back to the profession that has given so much to me.

To be clear, the views expressed here are mine and not necessarily those of my employer.

Thanks for reading.