Bernie Zeiher, chief medical officer (CMO) of Astellas explains why the ‘patient-defined value’ of medicines is crucial to developing therapies that truly address patients’ unmet medical needs. Zeiher outlines some of Astellas’ work with patient advocacy groups to generate these insights, why a new era of real-world data (RWD) generation and access is good news for the development of more patient-centric therapies, and how the biopharma industry must continually challenge itself to determine where true value lies for the patient.
Embracing patient-defined value
As an industry, our primary purpose is to develop therapies that address the unmet medical needs of patients. To do this effectively, we must truly understand the patient’s perspective on risk as well as benefit, as key issues impacting their quality of life, and challenge ourselves to deliver the therapies that provide the most value. If we are to transform healthcare, it’s critical for all of us to embody a patient-centric perspective in the truest sense.
This concept is an evolution beyond the traditional view of physicians, regulators, and payers as the key assessors of value, to a more holistic view, where patients define their own needs and priorities. While this evolution has presented challenges, it has also delivered important opportunities – enabling us to reframe our collective purpose to ensure we explore all avenues in the pursuit of maximising the value of breakthrough science for patients.
A new framework for value creation
At Astellas, value for patients is deeply embedded in our strategic vision and approach to research and development. We define it as ‘outcomes that matter to patients and the cost to the healthcare systems of delivering those outcomes.’ Beyond aspiration, ‘value’ also provides a strategic framework for new, more patient-centric ways of working, designed to improve execution, productivity, quality, cost and ultimately, meaningful health outcomes.
In practical terms, delivering patient value is no small effort. It requires deep, insightful understanding of unmet needs and designing innovations that overcome long-held barriers to care. If, as an industry, we get this right, we know the potential for a healthier, more sustainable future is great.
Critical insights to define value
With a clear, shared end goal in sight, we need to first identify, unravel and understand the key insights to guide us there. This starts with bringing patient-centric, insight-driven strategies through from the earliest stages of our pipeline.
At Astellas, one of our research focus areas is mitochondrial-related diseases. This is a highly complex and novel area of research where patients have few, or no, treatment options. Understanding their experience and perception of value is absolutely critical. For this reason, we are collaborating closely with patient advocacy communities, such as the Parent Project Muscular Dystrophy and the United Mitochondrial Disease Foundation, to embed insights into the earliest stages of discovery and development.
Significantly improved access to real-world data (RWD) is also facilitating deeper insights and defining new benchmarks for patient value delivery, with more sophisticated data collection, rapid digitisation and analytic capabilities now possible. A good case in point is the FDA approval of the supplemental New Drug Application (sNDA) for PROGRAF® (tacrolimus) for the prevention of organ rejection in adult and paediatric lung transplant recipients in the US last summer. The decision was based on real-world evidence generated from the US Scientific Registry of Transplant Recipients database which tracked outcomes based on discharge immunosuppression treatment regimens in more than 20,000 people who received a primary lung transplant between 1999 and 2017.
Significantly improved access to real-world data (RWD) is also facilitating deeper insights and defining new benchmarks for patient value delivery, with more sophisticated data collection, rapid digitisation and analytic capabilities now possible
This retrospective observational study was initiated based on patient and community need for expanded use of PROGRAF®, first approved for liver transplant recipients nearly three decades ago, and an insight into its routine use following lung transplants in combination with other treatments to help prevent organ rejection.
Fuelled by cases such as the sNDA for PROGRAF®, the conversation on how RWD can meet regulatory requirements is gaining momentum. Importantly, the potential of RWD to deliver vital new insights to broaden our view of patient need and value is immense and a growing priority for healthcare companies around the world.
The value of incorporating patient input
Patient insights are powerful. Used effectively, they can bring tremendous value to the design, quality and speed of our clinical programs – enabling us to enrol and retain more of the right patients, reduce protocol amendments, a timely and costly exercise, and, importantly, capture outcome measures that matter to patients and improve their overall experience.
Nowhere was this more evident than over the last two years. The pandemic triggered an experiment of nature, pushing the industry to accelerate patient-centric approaches and quickly adopt new decentralised approaches across clinical programs, such as direct-to-patient shipments and virtual visits. These measures enabled patients to safely remain in clinical studies rather than needing to discontinue treatment, but also to become more involved in their own care than ever before. This positive shift is giving us deeper insights and a more holistic view of the benefits of patient engagement.
Supporting a ‘future-ready’ healthcare system
In these rapidly changing times, we often find ourselves in unchartered territory, where traditional development approaches are incompatible with the unique innovations on the horizon. We must also recognise that innovation is futile if it cannot be delivered to the patients who need it most.
This means early dialogue with regulators and policymakers to demonstrate the value of emerging novel therapeutics and to help redefine patient pathways. Patient value must be communicated in a tangible, robust way, cognisant that each of our stakeholders’ perspectives is inevitably influenced by their unique perception of value.
‘Value first’ to meet society’s needs
Ultimately, it is the patients we serve who determine the value of the treatment options available to them and how they will improve their lives. With our research and development endeavours, and ambition to accelerate novel treatments, we know that patients are more empowered and informed than ever.
From an organisational perspective, a ‘value first’ mindset challenges us to be bold and laser-focused. Embedding a patient-centric culture is crucial, united by a strong vision from leadership and with the resources and mechanisms to deliver patient-centric work in practice.
These are exciting times for science, with cutting-edge technologies, once thought to be the realm of science fiction, on the horizon. To keep moving forward, we must continually challenge ourselves to determine where true value lies for the patient and be confident that we are advancing innovation in medicine not just because we can, but because we should.
