Patients are helping researchers look beyond mortality and morbidity measures to ask how tests and treatments affect quality of life.
Several years ago, Anne C. Beal, MD, MPH, learned first-hand about the scarcity of useful information available to patients and caregivers faced with difficult decisions. Toward the end of his life, Beal’s father suffered from multiple health conditions. “As his daughter, I often found myself in situations where we needed to make choices about his care. But we often did not have the information we needed or the information that reflected what was important to him.”
Recognizing an opportunity to correct this deficiency, Beal jumped at the chance to join the Patient-Centered Outcomes Research Institute (PCORI) as deputy executive director and chief engagement officer. PCORI is the not-for-profit authorized by the Affordable Care Act to fund and disseminate comparative effectiveness research—with the goal of helping patients and providers make informed decisions.
“We are working to arm patients and their families with the information they need when they are feeling sick and vulnerable to make the choices that matter most to them,” said Beal, who recently left PCORI.
As an example, Beal points to a PCORI-funded epilepsy study. “Epilepsy medications are pretty much equally effective at controlling seizures,” Beal said. “However, some of these medications make children feel sleepy or foggy. So parents naturally question, ‘What is the impact of this medication on my child’s cognition and on his or her school functioning.’”
To help parents find out, PCORI is funding an Emory University study involving 300 children with location-related epilepsy. The children are being randomly assigned to take one of three anti-seizure drugs. The researchers plan to test attention, memory, and other cognitive functions to determine the drugs’ effect on school performance.
The epilepsy study is just one of more than 200 research projects that PCORI has funded to date.
A Seat at the Table
“PCORI is blessed with having the word “patient-centered” as part of our title,” Beal said. “This requires us to make sure patients and those who care for them have a seat at the table and are really engaged as partners in the research.”
The Institute takes a number of steps to involve patients. For one, patients make up at least one-third of the seats on various PCORI advisory panels that help set research priorities. In addition, to receive PCORI funding, research studies must actively involve patients in planning and implementing the research. For example, one of the partners on the epilepsy study is a patient advocate who was diagnosed with epilepsy at age 15.
Beal believes that involving patients in research—as well as in their own care—is a vital step in improving health care. “Patients often express a feeling of being subjects for their care rather than being partners in their care. You often hear about what a hospital or doctor did to them. It will be a sea change when patients truly feel they are participating in their health care, rather than just being subjected to the whims and opinions of others.”
Populations at a Time
Beal’s zeal to improve health care is driven by an epiphany she had as a young pediatrician providing care to homeless residents in New York City. “There was a 13-year-old boy with sickle cell disease who had never seen a doctor. He had never been appropriately vaccinated and had never taken the antibiotic prophylaxis required to treat his disease. I was struck by the fact that someone could live in New York City and not have access to needed health care.”
In many ways, that was a turning point for Beal. “While I loved interacting with my patients, I felt like I was only helping one patient at a time. In contrast, through my work in healthcare research and policy, I can focus on improving care for large numbers of people at a time.”
Anne C. Beal, MD, MPH, is the former deputy executive director and chief officer for engagement, Patient-Centered Outcomes Research Institute (PCORI), Washington, D.C. She recently took a new position as senior vice president and chief patient officer, Sanofi.
Publication Date: Thursday, May 15, 2014