Patient-centricity: What's the Return on Engagement?
Patients are Ready
Laura Sol, chief of staff to the CEO at the American Heart Association (AHA), described a Patient-Centered Outcomes Research Institute- (PCORI) supported effort to engage patients, providers, researchers, and others to use crowdsourcing to collect novel research ideas. Eight thousand visitors to the project web site resulted in several hundred ideas and new insights about what topics matter most to patients. "One thing was made clear – you can't do research by yourself. All the perspectives are important," she declared. They learned that quality of life is the number one driver for patients, and those with multiple conditions, struggling with multiple treatments, need guidance about how to manage their health. AHA heard the one-condition-at-a-time approach common in research didn't address patients' needs, and they're making changes to address it.
Tania Simoncelli, executive director of Count Me In, echoed the readiness of patients to participate. "In the first 20 months of our metastatic breast cancer project, we enrolled 3,600 participants, all by word-of-mouth and social media. The patient-reported data participants have identified new study questions and important subpopulations we never would have found otherwise."
Michael Goettler, global president, Rare Disease of Pfizer, related its experience working with the sickle cell disease community. "It's not enough to understand the disease, you have to understand the whole patient and their experience." He described the often poor treatment that sickle cell disease patients receive in the emergency room, shaking his head to think that at a time of medical crisis, study investigators were trying to obtain consent for a clinical trial. "We wanted to do better," he said. "Now we work with families to pre-consent for our studies in a calmer moment. Enrollment is twice the historical rate."
Theresa Mullin, director, Office of Strategic Programs, Center for Drug Evaluation and Research at the U.S. Food and Drug Administration (FDA), shared a few lessons from the patient-focused drug development meetings. "First and foremost, we learned that patients have all the cognitive characteristics of experts. They are articulate, reliable experts about what it's like to live with a condition. That was a powerful takeaway for us." She described current efforts to convert patients' compelling anecdotes into evidence that can be used in regulatory decisions and included in the labeling of approved products.
Joe Selby, executive director of PCORI, followed this with examples arising from some of the completed studies it has funded. Engaging stroke survivors in designing a study of blood thinners led to use of "home time" as a primary outcome measure for comparing the effectiveness of various therapy approaches. "The Duke study team showed that use of statins reduced days hospitalized by up to seven weeks in some subgroups, the outcome that mattered most to patients. This made the results more meaningful to patients. I think we're just seeing the tip of the iceberg of what we stand to learn."
Ensuring it's Not a Passing Fad
While the panel agreed that we are still pushing the patient-centricity rock up a hill, rather than chasing it down the other side, there were some cautions about its popularity masking genuine effort. Goettler has seen this across the industry. "Inviting patients in to share their stories becomes very fashionable. Everybody is reaching out to them, but that's a burden on the patients, too. Stay focused on doing meaningful things, not just counting up activities," he advocated. Mullin added that FDA may soon be asking sponsors, "Have you done what you need to understand what people with the disease think?" when reviewing study protocols or applications. She suggested they'd do well to be able to answer that question affirmatively. Simoncelli suggested that measures of engagement could be used as a carrot, rather than a stick. Once people see its value and that it produces positive change, they would be more apt to try it. Selby agreed, especially when it comes to encouraging physicians to engage patients in treatment and care decisions. Sol advocated we all do more storytelling about what we set out to do in engaging patients, what we learned in the process, and how it mattered to patients. Simoncelli added a plea for sharing what didn't work along with what did.
Mullin and Selby focused on the need for measurement tools for outcomes that matter to patients – such as pain and fatigue – that are fit for purpose and cost effective to use widely. "It is all about obtaining better evidence. It doesn't have to be perfect, but it has to be good enough," Mullin said. Selby suggested that patients may be the key to helping propel research that cuts across conditions, through systems like PCORI's PCORnet, which joins clinic-based data collection with patient-powered research networks. Simoncelli pointed to the need to address problems rooted in medical records. "Most of the records we receive come to us by fax, and there are a lot of errors in them. We've simply got to work together on a policy solution." Lagging interest in patient engagement by payers and institutional review boards was another problem that surfaced during the session.
McCleary wrapped up discussion by recognizing the contributions each of the panelists had made to advances over the past five years. While there was much work still ahead to bridge gaps, build an adequate evidence base, and make practices sustainable across the board, the experiences shared by the panel offered much optimism about the promise ahead.
Global President, Rare Disease, Pfizer
Director, Office of Strategic Programs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration
Executive Director, Patient-Centered Outcomes Research Institute
Executive Director, Count Me In, Broad Institute of MIT and Harvard
Chief of Staff to the CEO, American Heart Association