Twenty years ago, when I started my industry career, I was fully focused on one development program. It was great that I could immerse myself into one disease area, one drug candidate, one clinical trial program, seeking to turn promising science into hope for patients. I had sight of all the opportunities and challenges, every decision that needed to be made, and what was happening at each of the clinical trial sites.
Two decades later, I oversee one of the largest and strongest pipelines in the industry — 70 new molecular entities (NMEs), 165 clinical development projects, with the potential to treat over 50 diseases. Clearly, it’s not possible for any one person to manage all of the complexities of our pipeline. We have 20,000 scientists, clinicians and business professionals focused on R&D, who represent extensive and diverse knowledge and capabilities. My role is to ensure we prioritize and advance the right programs across the pipeline that will deliver the highest impact for patients. Having an impact on many patients’ lives is the reason I, and I know many of you as well, went into medicine, and my purpose is fueled by the regular interactions I have with patients and patient organizations, seeing this impact first-hand. We have the opportunity to transform millions of lives with our medicines and we need to focus our efforts and resources towards that goal—this is our most important determinant of success.
The weight of these decisions can be immense and incredibly humbling. I often get asked about how we manage such a large and strong pipeline, so here’s an inside look into four strategic considerations we keep front and center.
1. We follow the science, going fast and strong if we see something that may work
In drug development, it is typical that we pursue one indication for a new drug to prove its mechanism of action prior to advancing further studies. At Novartis, we’re now increasingly focused on exploring ‘a pipeline in a single medicine’ based on the understanding of disease pathways and have multiple candidates where we are running several phase-3 trials with the same therapeutic in numerous indications simultaneously. One example is an investigational medicine where we are currently studying eight distinct diseases across a range of rare renal and hematological conditions. In such cases where we see a positive signal in the early data, we move forward fast and strong to explore all the potential applications of a new mechanism of action, which we’re able to do given the necessary resources, expertise and infrastructure an organization of our size has.
2. We double-down where we have differentiated knowledge and capabilities
Novartis is a leader in advanced technology platforms, like targeted protein degradation (TPD), cell therapy, gene therapy, radioligand therapy (RLT) and xRNA therapies. This gives us a unique opportunity to apply technologies across our pipeline and disease areas, and combine platforms to even further expand our capabilities. Take RLT for example, a modality that is used to deliver targeted radiation to tumor cells throughout the body and therefore has the potential to become a foundational pillar of cancer therapy. In addition to the success of our first RLT in an orphan disease, we are conducting research in more areas like advanced prostate cancer and multiple other solid tumors while working with external partners to find further potential combinations and applications.
3. We have the courage to go into the areas that have long been intractable
We are deliberately going after diseases where medical progress has been slow and there haven’t been significant new advances in recent years. Our goal is to change the standard of care where patients and healthcare systems need it most. Take pancreatic cancer for example, which has the lowest survival rate among all major cancers. We are studying a novel mechanism of action that targets the cancer in a different way. With bold bets like this we are also taking on a higher risk of failing, but regardless of the outcome of our trials in areas that have long been intractable like pancreatic cancer, we will gather critical new insights that will add to the scientific understanding of a devastating disease.
4. We don’t shy away from diseases with the highest evidence hurdles
There is no one way to study a disease or a new medicine and clinical trial recruitment is always challenging, whether you are exploring treatments for rare diseases with small patient populations or more common diseases with large patient populations. One of humanity’s biggest health challenges, cardiovascular disease (CVD), affects millions of people around the world, so conducting a study program takes enormous scale, resource and time. Novartis has a growing pipeline of potentially first-in-class molecules addressing CVD, with one of the long-term programs expanding to about 75’000 patients across more than 50 countries. Where others have backed away because of the hurdles that come with studying CVD, we are doubling down because we’re not afraid to take on the world’s biggest killer.
Approximately 85% of our confirmatory pipeline stands to be first-in-class or first-in-indication therapies. We start with where the highest medical need is. The four strategic considerations outlined above go hand in hand with what patients tell us, and we are incorporating their voices and perspectives into these important decision points along the drug development life cycle. To truly reimagine medicine for patients, we need to take bold risks, make tough choices and push the boundaries of science. The bar to changing the standard of care is high, and it can’t be reached by playing it safe, but the unmet patient need is higher, so it’s our responsibility to be curious and courageous to bring hope to patients with our breakthrough science.
You can find out more about R&D at Novartis here.