As we take stock of the scientific progress made by the biopharmaceutical industry, we begin to see its impact on the traditional pharmaceutical business model—one that has historically been influenced by blockbuster-drug development, physician preferences, and cumulative innovation. And beyond the need for continued innovation, greater improvement of patient outcomes and personalized medicine, the rising costs of R&D and healthcare are challenging all pharma leaders to think and act differently.
The time is here for measurable change—change that improves our ability to translate molecular insights into successful clinical studies and understand true patient needs above and beyond traditional study endpoints. There is no longer a place in healthcare for small and incremental steps in our mission to improve the lives of our patients. To succeed and deliver in a meaningful way, leaders in the biopharma industry must complement deep scientific insights and significant investments into clinical and translational research with a new patient paradigm. The combination of those will help us humanize diseases, especially for patients with rare, complex and advanced cancers.
Why the shift now? According to a recent Deloitte survey, personalized care from providers — including clear communication and sensitivity—is the number one priority for healthcare consumers. For patients battling rare and complex diseases, a holistic approach is warranted — one that considers their full lives, not just their disease. Biopharma is being called upon daily to apply this understanding to the way we interact with people with cancer—treating them as individuals rather than objects of their disease.
We need to create measurable momentum by innovating with a new approach to patient-centricity. This approach has five key elements:
- Redefining “benefit”
The biopharma industry frequently considers therapeutic success in cancer as a remission rate or freedom from disease progression. While these study endpoints are undoubtedly important from a regulatory standpoint, the biopharma industry must seek expansion of current definitions of success/benefit that focus on the patient’s quality of life—freedom from side effects and impact on their daily living. For example, it’s often a positive outcome for patients if a particular therapy is preventing a tumor from growing and keeping it under control, provided that the treatment is not associated with toxicity. As a result of their ongoing therapy, patients may be experiencing debilitating side effects that prevent them from maintaining any sense of normalcy in their lives let alone doing the things they love—reading, biking, or playing with their children. Often what a pure clinical trial context deems to a patient’s outcome may, in reality, compromise their ability to do things that matter most. As a consequence, the development of a cancer drug and improved mechanisms to support patients beyond treatment, including improved side effect management strategies, cannot stop at the time of drug approval.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
- Seeing patients as drug developers, not end users
The biopharma industry can take a big step forward by no longer viewing the patient as primarily an end user of the drug. Instead, patients should be seen as a co-developer of the drug. Biopharma companies need to start conversations with patients as early as in Phase I during the drug development process and, most importantly, maintain a feedback loop with patients throughout the whole development process. While patients may not have a full scientific understanding of indication and mode of action, they do have unique perspectives that can augment the process by providing input to endpoints, QoL measurement, and side effect management strategies.
In other words, patients can provide insights that translational and clinical researchers often can’t offer.
- Maximizing touchpoint opportunities in treatment
Consistent touchpoints with patients—not only at the beginning and end of a clinical trial—will yield more meaningful and actionable insights that inform where we can make the greatest impact. This means including patients in the entire treatment process and understanding the impact of drug scheduling, side effect management strategies, and longitudinal quality of life (QoL) measurements. It also requires going beyond the patient—talking to physicians, nurses, caregivers, friends, and family—who all need to be part of patient care. The patient has a context around them—age, gender, culture, support systems, work, and hobbies. A deeper understanding of this context and engagement with the “stakeholders” in the person’s life can be revealing, insightful, and deeply compelling. Creating a steering committee comprised of nurses can be instrumental in drawing the connection between our work and the realities of what is occurring at the frontline with patients.
We have also leveraged advancements in technology to create unique touchpoint opportunities through a Digital and Data Solutions team. In this function, companies can define three distinct roles that work together experience design, digital innovation, and enterprise-wide data science. Our approach to solving these problems is to deeply understand root and proximate causes by frequently engaging with the appropriate users /stakeholders.
- Humanizing communication
One of the most important things we have learned is to have the willingness to expose ourselves directly and frequently to the patient experience. Focus groups and surveys will not get drug developers the same quality of information achieved by visiting patients face-to-face in their home. We will never get a true picture of what the drug is truly doing for patients on all parameters if we are not exposing ourselves directly and broadly to their day-to-day lives. Contextual, in-person, and human conversations—such as immersive, hours-long interviews at a patient’s home—are how to gain the best insights about patients and achieve a meaningful approach to patient-centricity. Going the extra mile to connect with patients and their inner circle to identify details–such as which activities and routines are most important to their daily lives– could mean the difference between a positive or negative outcome.
The development of patient portals and online communities have also proven to be effective in engaging and supporting patients as they live with their disease. And the need for connection with others who can relate to their experiences has become increasingly important, especially for patients with rare and complex diseases. This need led us to create a patient community platform to help humanize cancer, be the catalyst for self-advocacy, and shift the focus from disease to life with disease.
- Learning from failure
While not a novel concept, we must continue to instill an openness and ambition to learn from treatment failures, honestly communicate and learn from those failures. We must not only engage patients who are seeing positive results but equally engage patients who are not finding success in their treatment as we often learn—scientifically and clinically—more from failures than successes. A 50 percent clinical benefit rate in late-stage cancer is highly respectable, but still means that 50 percent of patients are not benefitting from the treatment—that’s not good enough. We must be obsessed with what discriminates success from failure, communicate honestly, and learn how we can do better for that 50 percent. If we don’t, we will fail at our mission to provide patient-centric care.
As an industry, we must be humble enough to realize that we can’t successfully meet the needs of every single patient. But if we refuse to settle for “good enough,” focus on the day-to-day living of patients, and dig into the data behind the clinical successes and failures, we can and will foster better outcomes.
Photo: Getty Images
