How Patient Voices are Changing Medicine

How well are you able to carry out your usual social activities? How does your sleep issue affect your ability to do things with your family? Do you feel like you’re getting better?
 
These questions may seem unusual, but the answers are critical in understanding a patient’s disease burden, specifically, the health-related quality of life impact of a treatment, according to one of AbbVie’s leaders in patient-centered outcomes research.
 
This leader may be new to AbbVie, but her focus in patient experience isn’t. Katy Benjamin, Ph.D., director, health economics and outcomes research, leverages her expertise in developing tools to measure treatment outcomes from the patients’ perspective. She digs into what patients think and feel, combining this valuable information with more traditional clinical data to understand the burden of disease and how our molecules and medicines may reduce that burden – ultimately, to help create better treatments for patients. 
 
We sat down with Katy to understand the role of the patient-centered research (PCR) group within AbbVie and how some critical insights are discovered outside of the lab.

Katy Benjamin, Ph.D.: I’ve always been interested in patient behavior – what motivates someone to go to the doctor, to stick with a treatment. To answer the question: What are the factors that make people believe they’re getting better?
 
My background is in psychiatric social work, with a master’s in that area, and a master’s from Harvard in public policy. Before earning my doctorate at Johns Hopkins, I worked as a social scientist for the Agency for Healthcare Research & Quality, a U.S. public health agency, leading early efforts to understand how to measure the patients’ perspective when evaluating the effectiveness of medical care. I then worked in consulting for many years to develop and validate questionnaires to measure how patients viewed their health and their medical treatments, mainly for use in clinical trials. Over the course of my career, I’ve had the opportunity to talk to hundreds of patients with a wide variety of medical conditions.
 
I had to understand the “what” and “why” for patient outcomes. And as a social worker, I was trained to talk to people. My research training gave me the tools to turn these insights into questionnaires to measure what’s important to patients.

Benjamin: The role of the PCR team is to help bring patient centricity – the voice of the patient – to AbbVie. That can be applied in a broader sense, like how we approach unmet need in medical care based on what patients can tell us. It’s also applied to how we build our clinical program and specific clinical trials, ensuring we put measures in place to help us understand patient perspective in terms of evaluating the efficacy of our treatments. 
 
Patient reported outcomes (PROs), or data that comes directly from the patient, are an important way to measure their experience.  They can be assessed in clinical trials, and analyzed relative to clinical data. They can also be assessed in real world studies. Real world evidence (RWE) is becoming increasingly important for us to truly understand the complexity of diseases and the burdens patients face. We need to tie our programs to the real world, and that’s why AbbVie recently created an evidence generation team within HEOR to strengthen our approach. We in PCR work closely with our RWE colleagues to understand the whole picture of people’s medical care experiences – what’s happening and why.

Benjamin: This hits on one of my personal passions, and that’s really listening to what the patient has to say. If we look back at the history of drug development, the voices of patients typically weren’t considered important. Instead, we focused on the voice of the medical community and clinical outcomes – e.g., whether the tumor shrinks or if a laboratory value changes.
 
But, these types of clinical outcomes may not be something patients are noticing themselves. Instead, they may be having other experiences that we don’t know about unless we ask. A great way to do patient-centric research is by working with patient organizations. For example, we may learn of a symptom people are experiencing, which isn’t being picked up by clinicians who are instead focusing on a more prominent symptom. We can also understand more comprehensively which symptoms are most important to measure because they impact the aspects of patients’ lives that are most important to them. We gain this initially through qualitative research studies and in turn get a broader understanding beyond specific groups in clinical trials. Regulators and Health Technology Assessment (HTA) authorities are increasingly requiring that we understand and demonstrate to them the impact our novel medicines are having on patients.  

Benjamin: We’re seeing a growing awareness for how important the patient point of view is to understand the quality and value of medical treatments across the world, reflected in government initiatives that include patient perspective when assessing the value of both new and existing medical treatments.
 
In the U.S., building on previous initiatives like workshops with patient advocacy groups, the FDA is developing guidance documents on how to appropriately collect, analyze and report data on patient experience. These insights on how patients view their condition, treatment and participation in clinical studies are important to us in industry as well as to regulators. These guidances illustrate that regulators recognize the value of patient-reported outcomes and offer practical information on how this information can impact product labeling, including the uses and effectiveness of treatments.
 
The European Medicines Agency (EMA), which evaluates new drug applications across the European Union, takes patient-centered outcomes evidence into account in its evaluation of treatment efficacy and approval, as do most country regulatory agencies. The EMA and FDA are collaborating on strategies to include the patient voice in their regulator decision-making.