Accelerating Progress for Patients: Redefining R&D Approaches to Tackle Diseases with Serious Unmet Medical Needs

The speed of innovation in life sciences is having a seismic impact on our ability to deliver ground-breaking new therapies rapidly and effectively. As a physician, it is remarkable to witness how recent advances in our understanding of disease pathophysiology and the availability of novel technologies, including cell and gene therapies, have opened up new avenues of treatment for conditions that were previously viewed as untreatable.

I have generally hesitated to use the word “cure,” but the latest breakthrough therapies have been shown to prevent, dramatically alter the course of, or even cure diseases that previously had limited to no options. Furthermore, these therapies are being developed at a record pace. Consider the HIV epidemic, which has shifted from a diagnosis seemingly devoid of hope to a condition so well treated that patients can now expect to live a long and full life. This transition from fatal diagnosis to manageable condition has occurred through the course of my own medical career.

Contrast the time period over which HIV became more manageable to what we are witnessing with the COVID-19 pandemic. COVID-19 has mobilized an unprecedented global effort to develop an effective vaccine. The release of the genetic sequence of SARS-CoV-2 in January 2020 catalyzed the advancement of more than 60 vaccine candidates into clinical development. Within 11 months, two novel, highly effective mRNA vaccines received emergency use authorization from the FDA and in numerous countries around the world. These are not only the first mRNA vaccines approved for humans, but also the fastest vaccine development programs in history. Had the pandemic struck a few years ago, the outlook for a vaccine may not have included mRNA or other novel vectors and would have taken significantly longer to develop, with dramatic implications for public health and the global economy.

While the idea of preventing or even curing disease is now increasingly viewed as a realistic aspiration, it is also a tall order. As an industry, we have much work to do to realize this higher ambition because innovation alone is not enough. To move rapidly, but also safely in this new environment, we must reassess our ways of working, culture, resources and capabilities, while maintaining the highest standards of safety and quality.

The good news is that, under the right conditions, we’ve shown revolutions in how we innovate in healthcare are possible. Looking forward, the key question to ask now is: how can we apply the lessons from working at “pandemic pace” to the future?

My career in the pharmaceutical industry has taught me that developing innovative therapies requires tremendous interdisciplinary collaboration, especially as we adopt specialized and next-generation therapeutic modalities, such as cell and gene therapies. Several key factors must be considered as we work together to build the innovation-led organizations of the future.

First, we must build necessary capabilities and this starts with an honest assessment of the existing R&D organization. By clearly understanding strengths as well as skills gaps and inefficiencies, we can adapt our approach to be future-fit. In addition to novel science and therapeutic modalities, digital is transforming how we do our work. Real world data can help us understand the natural history of a rare condition and conduct better clinical trials. Historically, we have relied on controlled trials. However, in some rare conditions, it may not be feasible to conduct adequately powered, placebo-controlled clinical trials and the use of historical controls or delayed treatment may be necessary.

Second, if capabilities and skills are the engine, then a culture supporting innovation is the rocket fuel. We must create and support an environment that enables innovation to flourish. This involves establishing a culture of mutual respect and diversity, ensuring that teams share a clearly communicated vision and mission. Consider the inspiration and sense of urgency that must have been present on the teams developing the SARS-CoV-2 vaccines. They had to be incredibly focused and driven to deliver a vaccine that could not only help themselves and their families, but also help end this global pandemic.

That sort of inspiration and shared purpose can only come from a drive to address the needs of patients. This provides the impetus to design better clinical programs and bring candidates forward even faster. No matter what their role, I believe that everyone working at Astellas is motivated by the same truth: what they do matters on the most human level. People all around the world, who may have no understanding of what goes on in our laboratories and development centers, are having their lives saved, prolonged and changed for the better by what we do as an industry.

Be sure to join me next month for a new article, in which I will share my perspective on how the industry needs to evolve and embrace risk when developing “breakthrough therapies.”

Bernhardt G.Zeiher, M.D., F.C.C.P., F.A.C.P.
Chief Medical Officer, Astellas Pharma Inc.