In my role as chief medical officer at Astellas, I lead the global Medical and Development organization and am dedicated to the development of transformative therapies which will deliver meaningful outcomes that matter most to patients. As a physician by training, I am committed to putting patients front and center.
We are living through a defining decade for the future of medical science. A potent combination of advances in our understanding of disease pathology and the availability of novel modalities and technologies is fueling an unprecedented acceleration in innovation. Right now, being at the forefront of drug development is an exciting place to be, but it’s also a challenging one. Not only are the stakes high — patients are waiting — but there are also often no precedents and no well-trodden paths to rely on.
In this incredibly fast-paced environment, I’m learning every day and here are five lessons I can share from my experience of navigating development at the new frontier of healthcare:
Embrace patient-centric approaches.
More than ever before, patients and caregivers are knowledgeable, organized, and eager to engage in discussions around unmet medical needs and clinical trial designs. A key learning from our work in novel modalities in rare diseases is that the more deeply — and more swiftly — we embrace patient input, the more effective we are in advancing the innovations that matter most. It demands us to understand the patient journey in a way that is only possible with early, meaningful, and open input from patients and their caregivers. We need to know their experiences, the key issues impacting their quality of life and their insights on risk as well as benefit. Collaboration must involve many voices and perspectives and it is just as important that we partner with those best placed to shape and define the frameworks that will one day enable access to those most in need. By opening ourselves to collaborate, across industry, patient communities and regulators, we can accelerate the discovery, development, and delivery of the patient-led medicines of tomorrow.
Foster an agile and experimental mindset.
In the past, we worked on small molecules or monoclonal antibodies in precedented areas. Today, we are often working with new technologies in unprecedented indications. Often rare diseases are insufficiently characterized, for example the disease natural history may need to be studied and clinical endpoints qualified before even starting an interventional trial. Therefore, early partnership is essential in addressing these issues, with regulatory authorities to align on disease definition and endpoints that will support clinical investigation, as well as with physician experts and patient groups to better understand these novel disease indications. But even with this pre-study work, much will be learned throughout a clinical program, requiring new levels of flexibility to incorporate learnings in real-time. To be successful in this fast-moving environment, teams must have an agile and experimental mindset, ready to adapt our clinical programs to incorporate new learnings or insights on the disease or effect of the therapy.
Have a sense of urgency.
The pandemic taught us that we can deliver our trials differently, offering greater patient-centricity and flexibility, notably through decentralized approaches. As a matter of fact, those new approaches enabled the Covid-19 vaccine trials to complete in record time. We are also working in a highly competitive environment and standards of care can change rapidly, making trial results irrelevant if not delivered quickly. Meanwhile, patients are waiting for new innovations and better outcomes, which should motivate all our efforts, to act with urgency and focus in solving the unmet needs of patients.
Know your value as a partner.
Today, around 25% of approvals for new molecular entities (NMEs) represent biotech companies securing their first approval.1 This means a significant proportion of biotechs are rejecting the established view that they need a big pharma partnership to get their innovations through late-stage development and approval. For pharma companies with an appetite for biotech partnership, it’s a reality check and time to rethink what we offer our partners. It demands that we bring more to the table than just our global reach — more agility in how we plan and conduct clinical trials, more flexibility in how we explore the potential of an NME and, importantly, applying fresh thinking to how we approach access.
Be prepared for the unexpected.
Working in the unknown, in potential breakthrough technologies such as gene and cell therapies, comes with risk. We must always strive to ensure the safety of the courageous patients who enroll in clinical trials, acknowledging the ambition needed to prevent a serious disease or even achieving a cure. Serious safety events may occur and necessitate a pause in clinical dosing and the implementation of new risk mitigation measures. Dramatic efficacy results can also trigger consideration of earlier regulatory submissions, engaging with regulators to identify whether a smaller number of treated patients could be used to support potential approval. Collectively, we are all learning as we go — as organizations, from each other, and from the patients we serve. In our pursuit of developing what could become the first effective therapy to treat a previously untreatable disease, we must be bold and agile, ready to learn from the unexpected, and never forget the patients who are waiting.
Learn more here about how Astellas is turning innovative science into value for patients.
1New Drug Approvals for 2021, U.S. Food and Drug Administration, https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/novel-drug-approvals-2021