Global impact: How regulatory affairs is shaping development of cutting-edge pharma innovation

Views & Analysis
Global impact: How regulatory affairs is shaping development of cutting-edge pharma innovation

When it comes to developing cutting-edge innovations, Astellas aims to be at the forefront. The pharma organisation prides itself on having an open, collaborative, “science first” approach to help ensure breakthrough discoveries have the very best chance of long-term success in bringing new treatment options to patients worldwide.

“Our mission is to turn innovative science into value for patients and we are working in a lot of different areas with high unmet need,” says Stef Schutte, Astellas’ head of regulatory affairs. “We have a pipeline of novel therapeutics and technological approaches, advancing a diverse array of candidates, including small molecules, biologics and cell and gene therapies, so a lot of the time we are working where there's little, or no precedent.”

As these innovations – which have the potential to be transformative for patients – enter uncharted regulatory territory, the pathway is not always clear or well-defined, which is why Astellas adopts a proactive approach to early communication with health authorities.

“We work to build positive and effective relationships by having open and early engagement and dialogue. This enables us to provide the rationale for a novel approach and anticipate trends so if we run into unexpected issues, we can act quickly and safely.”

Collaboration accelerates progress for patients

Collaboration when innovating in areas of high unmet medical need is critical and at Astellas, it means experts with highly specialised knowledge of these new discoveries often work in close partnership with regulators.

“We connect these subject matter experts with the regulatory authorities to facilitate information exchange. It’s through this collaboration that we help shape the future.”

“We work to build positive and effective relationships by having open and early engagement and dialogue. This enables us to provide the rationale for a novel approach and anticipate trends so if we run into unexpected issues, we can act quickly and safely.”

In the very early phase of development, Astellas also participates in public-private partnerships with academia, regulators, experts and beyond. Bringing them together ensures key topics are brought to the forefront of conversations, enabling us to navigate and co-define the path forward, explains Schutte.

Global innovation initiatives also help Astellas identify appropriate pathways for accelerated development and review of new therapies across markets. In the US, for example, the FDA now provides the opportunity for initial target engagements for regulatory advice, known as ‘INTERACT meetings’. The consultation meetings discuss innovative, early-stage investigational products and are an opportunity for innovators to get informal advice from the FDA and the Center for Biologics Evaluation and Research (CBER) on specific technical aspects. Similar processes are available around the world, with PRIME in the EU and SAKIGAKE designation in Japan, promoting early practical application for innovative pharmaceutical products, medical devices and regenerative medicines.

The impact of COVID-19 and implications for the future

With COVID-19 setting new precedents for accelerating the development, submission and approval of novel therapies, Schutte expects some changes are here to stay. Specifically, the digitalisation of regulatory processes which is being widely adopted.

“When the pandemic struck, we pivoted basically overnight to integrate many remote and digital processes – things like preparing submission dossiers remotely, but also decentralised clinical trials which are more patient-centric as the trial revolves around the patient and not the test center. These are certainly things that are there to stay as they can really help accelerate progress,” reflects Schutte.

Regulatory authorities also made significant adaptations, for example, conducting remote Good Manufacturing Practice and Good Clinical Practice inspections, and also the acceptance of digital signatures. However, not all of these processes may be sustainable for the future.

“If you prioritise one disease area and accelerate approvals it can unintentionally slow the development of important treatments for other diseases, which might have an equally high unmet medical need, but maybe for a smaller population,” says Schutte.

“I think we must certainly strike a balance; prioritising patient need and focusing on therapies that have the greatest potential for delivering value, while continuing to reassess our processes to accelerate development for patients in need. It's no longer about just focusing on the high unmet medical need for everyone, but the high unmet medical need for different patient groups. I think many of these things will be accelerated compared to the pre-COVID-19 situation.”

A strategic partner

Regulatory affairs as a function has undergone a significant shift in its strategic role and impact over the past 10 years – delivering value from early-development through to the management of approved therapies.

“I think what you see is regulatory affairs professionals bringing together various aspects, not only in the development of therapies, but also in manufacturing and supply chain management,” explains Schutte. “By doing that, regulatory affairs professionals help guide the development and lifecycle of a therapy.”

Astellas’ regulatory affairs function plays a key role in helping to define the final target indication of the therapy, or broadening the target for the profile, or target product label. “In this way, we guide and inform clinical development plans, demonstrate how decisions are being made and define which decisions could have an impact on the final target indication of the therapy.”

The organisation focuses on building relationships with internal and external stakeholders, as well as health economics and patient outcome research groups. “We work together to define an optimal label which will help maximize the value of our therapy for patients.”

Looking to the future

As innovation continues to accelerate, Schutte says he expects to see more globalisation of solutions. “We will see a growing presence in Asia and specifically China as they undergo rapid regulatory reform that can help accelerate development and approval of innovative healthcare solutions in these markets.”

In China, for example, a category-one submission exists, where submission is only possible if the product is not already approved elsewhere in the world. “That means that the timing of submissions, and timing of development becomes more and more critical, in addition to meeting the regulatory requirements.”

There may also be a rise in work-sharing initiatives such as the US FDA’s Project Orbis, which enables concurrent submissions and assessment for all countries participating in the scheme, according to Schutte.

Another key element is likely to be an emphasis on integrated and more personalised healthcare solutions. “In principle, this goes beyond medicines. We anticipate a revolution in digital solutions, such as artificial intelligence, bioinformatics and companion diagnostics.”

Overall, regulatory affairs will continue to be a key player in how these approaches are communicated and are approved by the authorities. “We will always work together to define the regulatory pathways of these novel approaches,” says Schutte.

Astellas’ creation of the ‘Rx+ program’ aimed to leverage the organisation’s existing expertise in pioneering technology and knowledge from different fields. “Our aim is to build cutting-edge healthcare solutions, and services that go beyond medicines and contribute positively to the entire patient journey, encompassing prevention, diagnosis, treatment and post-treatment care and management,” adds Schutte.

Regardless of regulatory reforms, patient centricity has and always will be at the heart of Astellas' focus. ''There is an ever-increasing patient-insight driven approach to development and whatever we do, patients needs must be heard or understood,'' concludes Schutte. ''I am excited to build regulatory affairs' capabilities and the ability to help prioritise the therapies that offer the greatest potential to deliver value for patients.''

About the interviewee

Stef SchutteStef Schutte is executive vice president and head of the Regulatory Affairs function worldwide and is based in Leiden in the Netherlands. In this role Stef is a member of the Medical & Development Leadership Team as well as heading the Regulatory Affairs Leadership Team. Since May 2017, Stef has been a Board member and statutory director for Astellas Pharma Europe BV and its parent company, Astellas BV. Stef is a standing member of the European Federation of Pharmaceutical Industries Association’s Innovation Board Sponsored Committee, as well as a member of the International Federation of Pharmaceutical Manufacturers & Associations Regulatory Strategy Committee. Stef is also a member of the permanent Advisory Board of the George Washington University Regulatory Affairs program.

About Astellas

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Astellas Pharma Inc. is a pharmaceutical company conducting business in more than 70 countries around the world. We are promoting the Focus Area Approach that is designed to identify opportunities for the continuous creation of new drugs to address diseases with high unmet medical needs by focusing on Biology and Modality. Furthermore, we are also looking beyond our foundational Rx focus to create Rx+® healthcare solutions that combine our expertise and knowledge with cutting-edge technology in different fields of external partners. Through these efforts, Astellas stands on the forefront of healthcare change to turn innovative science into value for patients. For more information, please visit our website at

15 April, 2021