Range anxiety is the worry that an electric vehicle won’t have enough charge to reach your destination. Now imagine you’re not anxious about a car battery, but about the engine of your heart.
That’s life for Scott Popjes. “When I travel, I have to think about where the nearest good heart hospital is, in case I have any issues,” he says. “So I always map out my journey.”
Popjes, 59, of Los Angeles, lives with hypertrophic cardiomyopathy (HCM), a genetic disorder that affects one in 350 individuals worldwide. HCM thickens and stiffens the heart muscle, making it hard to pump blood. The increased strain on the heart can cause an irregular heartbeat (arrhythmia) and often leads to heart failure. As a consequence, intense activities such as basketball and baseball, which Popjes loves playing, are no longeran option. He has also had surgery to remove some heart tissue, and his implanted defibrillator has twice shocked him to stop life-threatening arrhythmias.
Today, Popjes, works with Cytokinetics to represent HCM patients on an advisory council.
The advisory council is part of a larger company-wide effort to keep the patient’s voice front and center as Cytokinetics makes key decisions in areas such as clinical-trial design. Such programs are part of a larger industry trend toward listening to patient perspectives, says Ann Moravick, founder and CEO of Rx4Good, a patient advocacy and engagement consultancy.
People with different diseases “bring diverse perspectives to the healthcare system. They are often caring for families, facing economic constraints and don’t fully understand the language that healthcare professionals speak,” Moravick says. “Patient centricity is looking at an individual as a person, listening to them and acting on their perspective.”
Progress toward patient-centricity has been slow enough in the industry that the contract research organization Parexel called it a “broken promise.” But there are ways for such efforts to succeed, which includes demonstrating the measurable impact of patient perspectives on industry reputation and business success, Moravick says.
And a few pharma firms are taking big steps along that path.
An eye-opening simulation
Shirley Ritter, a therapeutic medical scientist at Cytokinetics, doesn’t have HCM or any other form of heart disease—but she has experienced its impact.
Along with 45 other Cytokinetics employees, she participated in an immersive simulation that the company co-created in partnership with a U.K. company, A Life in a Day, with input from patient advisors. Over two days, staff engaged in realistic scenarios through role-play and events to gain insight into the challenges faced by HCM patients, from physical to psychological.
During the simulation, regular alerts from an app prompted participants to experience symptoms such as shortness of breath. This involved breathing through a straw, which feels more like a bad panic attack than strenuous exercise, explains Ritter, a nurse. That experience hit especially hard for her because she once lost her own child to Noonan syndrome with severe HCM.
The simulation also prompted participants to consider the wider effect of a disease, which limits daily habits and can impact your career. In a role-playing scenario for an internal job interview, an actor pretending to be from human resources uses your private medical history to suggest that you wouldn’t be up to the task of the new role because of your condition.
Employees at Cytokinetics found the experience valuable. A follow-up survey by A Life in a Day found that 92 percent said the simulation increased empathy for people with HCM, many described it as eye-opening, and everyone reported that it motivated them to increase their focus on patients.
Walking the walk
The key to true patient-centricity is to institutionalize it within a company’s culture and ways of working, Moravick says. That means all employees, from front desk to finance, appreciate the needs of patients, and new structures are established that foster dialogue. “It’s making sure that from the very top of the company through the fabric of the organization, there’s an approach to integrating patients into decision-making,” she says.
Cytokinetics is taking big steps along that path, says Mary Pomerantz, senior director of patient advocacy and engagement at the company. “What I feel is special about us is an authentic effort to really walk the walk as it relates to patient-centricity,” she says. “That starts with our CEO, who speaks to a deep commitment at the leadership level, and we recruit individuals who feel connected to the mission.”
That mission—putting patients first—includes designing endpoints for whether a drug is effective in phase 2 clinical trials. “We talk to experts, advocacy organization leaders, actual patients and caregivers,” Pomerantz says. “Whatever the business question, we find an approach that honors patient preferences. What’s best for patients is ultimately our guiding light.”
During phase 3 trials for efficacy that precede approval, Cytokinetics involves a patient advocate in monthly meetings of a steering committee that tracks a trial’s conduct and progress. That advocate becomes an author of the published data and often contributes to a plain-language summary of the results to keep the science accessible to a lay audience.
If a product does reach the commercial stage, Cytokinetics uses patient input to determine how best to communicate information. This can lead to changes in response to patient concerns. For example, the company revised some educational materials for its approved prescription medicine by simplifying access to the materials and website.
Listening to patients can mean incorporating small suggestions that improve quality of life, too, such as altering the shape and color of pills so a person can better distinguish a drug and dose from the other medications they may have to take daily.
Scott Popjes not only suggested that pills should be purple, but he also helped mold the simulation of living with HCM. “Shaping that, seeing it come to fruition and then seeing the folks at Cytokinetics participate in that experience was fantastic,” he says. “Suddenly they're like, ‘Oh, I get it now—you deal with that all the time.”
Explore Cytokinetics’ patient-centricity efforts in this report. Learn more about some of its pioneering medicines for heart-muscle conditions here.
