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As regulatory reform continues to evolve in the US and Europe, the role of regulatory affairs is poised to help shape the more personalized treatment landscape on the horizon.
Across the globe, the healthcare industry is working hard to transform care and accelerate improved outcomes for patients. From the growing importance of real-world evidence (RWE) and emerging technologies for data capture and analysis, to the discovery of new modalities and disease states, this rapid transformation is touching every aspect of our work. As contributors to this period of change, all of us working in biopharma have an opportunity to leverage innovation to enhance the patient experience and evolve the paradigm of care.
The pandemic has also been a profound catalyst for change—the adoption of virtual care solutions, accelerated drug development processes, and heightened levels of collaboration, particularly between regulatory agencies and industry, laid the foundations for new models of engagement.
As the expectation for better and more personalized care grows every day, regulatory affairs (RA) is set to play an increasingly important part in ensuring patients can access the potentially life-changing innovations of tomorrow.
Historically, clinical development often has been based on known pharmacological mechanisms, with small, stepwise innovations. Today, innovation is increasingly more radical, focused on next-generation therapies for complex or rare diseases, using platforms sometimes for the very first time. It is truly an exciting period of discovery, but also unchartered territory with increased uncertainty of the outcome—for industry and regulators alike.
Regulators, including the FDA and EMA, are already calling for new approaches as we adapt to these novel areas. Harmonization of approach among agencies is underway in some regions, but there is a long way to go before alignment is a global reality. More agile and responsive regulatory systems are needed—not only for realizing the potential of these new therapies for patients but for encouraging future innovation.
For RA, this is an opportunity to demonstrate our strategic impact in enabling organizations to adapt to the complexities of innovative science and the changing regulatory landscape, while continuing to fully explore the optimal use of therapies already available. We have a unique opportunity to inform development strategies, guide decision-making, and enhance our understanding to assess how we can deliver even greater value for patients, today and tomorrow.
As our efforts combine to transform and accelerate care for patients by realizing the potential of novel therapies, RA has learned to pivot quickly. New engagement models have been established, and our response to these evolving approaches is more important than ever.
Early, frequent interactions with a broad group of stakeholders and experts are vital to define and agree on the path forward. Ensuring open, transparent dialogue enables us to facilitate information exchange and share progress, learnings, and insights to ensure the key unmet needs of patients and prescribers are met.
RA has an increasingly central and strategic role as knowledge experts, acting as the link between industry and regulatory agencies. Being aware of and interpreting regulatory trends ensures we understand how new modalities may be approached by regulators so we can contribute to shaping pathways to expedite their approval for patients in need.
Internally, we share these insights and expertise to guide teams from the earliest stages of discovery through the post-approval setting with strategic, as well as operational, support across the entire value chain.
Externally, proactivity and partnership matter. We foster relationships with regulators and industry associations, as well as together in public-private partnerships, such as the Innovative Medicines Initiative and Innovative Health Initiative. By partnering and pooling resources, we ensure that research results are turned into useful innovations for patients, setting the precedents which will define the industry-wide pathways needed for tomorrow’s medicines.
RA professionals continually embrace new possibilities to drive the industry forward. We never stop investigating how to elevate the safety, efficacy, quality, and performance of our innovations, as well as focusing on optimizing the treatments already available, to continually seek new ways to improve patient outcomes.
Together with health authorities, we have a key role in advancing regulatory science—the science and research intended to inform decision-making in a regulatory framework. Our goal is to implement the principles, processes, and tools needed to ensure we are equipped for the future.
This is evident in how we are leveraging enhanced data access and analytics, with the validity of RWE being increasingly recognized by regulators as a tool that can enhance our exploration of alternative data sets with patient-centered outcomes.
A key example is the 2021 FDA approval of a new use for PROGRAF® (tacrolimus) in lung transplantation—considered a significant milestone by regulators. First approved nearly 30 years ago in the US for the prevention of organ rejection in patients receiving liver transplants, a retrospective observational study of more than 20,000 people supported the expanded approval of PROGRAF® for the prevention of organ rejection in adult and pediatric lung transplant patients.1
This marked the first approval of an immunosuppressant therapy for this use, highlighting the potential of a noninterventional study relying on fit-for-purpose, real-world data to meet regulatory requirements. This demonstrates the impact RA can have by truly listening to and understanding the needs of patients and prescribers, adopting novel data-driven approaches, and working in partnership with regulatory agencies to maximize the value we can deliver to patients.
Addressing unmet needs and bringing new treatment options to patients is never easy, but rapid advances in many areas are helping us to navigate complexity and unlock the potential of health innovation.
RA already makes an important contribution to this—from the earliest stages of drug discovery and development through patients in the real world. However, we know more can be done.
Regulatory reform is ongoing worldwide and, with a more personalized treatment landscape on the horizon, this reform will support industry, regulators, and society alike in realizing the value of scientific and technological discovery. A joined-up, patient-centric approach will not only deliver the novel therapies of today, but inspire and galvanize the innovators of tomorrow. With new perspectives already shaping the future, the full potential of RA is yet to be delivered in transforming value for patients.
Stef Schutte, PharmD, MSc, EVP, head of regulatory affairs, Astellas Pharma Inc.