Expanding the Role of Patients in Setting Research Priorities | Greater Boston

Expanding the Role of Patients in Setting Research Priorities

By: Tiffany Parnell
Friday, July 1, 2016

Studies point to a frequent disconnect between the priorities of clinical investigators and patient and physician end users. Engaging patients and other stakeholders in research prioritization can enhance study relevance and may even shorten the time it takes to bring scientific discoveries from the laboratory to the bedside.

In the United States, roughly $70 billion in commercial funding and $40 billion in governmental and nonprofit funding is dedicated to biomedical and public health research each year, according to a 2014 study in The Lancet. However, the gap between the priorities of researchers and those of clinicians and patients may limit the impact of the research.

A patient-centric Approach

Clinical investigators’ objective of furthering the science in their respective disciplines doesn’t always align with the goals of patients, who want better treatment options, and physicians, who often lack evidence on the outcomes that matter most to their patients. The burgeoning field of translational science may play a role in ensuring research findings are pertinent and effectual.

The National Center for Advancing Translational Sciences (NCATS), part of the National Institutes of Health, defines translational science as “... the field of investigation focused on understanding the scientific and operational principles underlying each step of the translational process.” The goal of translational science is to turn new observations, discoveries and interventions into health benefits, according to Petra Kaufmann, MD, MSc, Director of the Office of Rare Diseases Research and Division of Clinical Innovation at NCATS.

By making patients the center of research endeavors, translational science may hold the key to streamlining patient recruitment, one of the most time-consuming elements of clinical research. If patients are included in discussions about protocols, for example, researchers can enhance participation by ensuring that joining a study is feasible for patients and won’t overly burden their day-to-day lives, according to Dr. Kaufmann.

Janine Farragher, a Vanier Scholar, occupational therapist and PhD candidate at the University of Toronto and a participant in Canada’s national kidney scientist training program, KRESCENT, has seen the value of translational research firsthand. In a 2015 review in the Canadian Journal of Kidney Health and Disease, she recounts how translational research in tissue typing has benefited patients undergoing kidney transplantation.

“Tissue typing uses diagnostic techniques to test the histocompatibility between prospective donor kidneys and potential recipients,” Farragher says. “An expanded pool of patients are eligible to receive kidney transplants because of advances in tissue typing, and their risk of graft rejection is also reduced. The collaboration that occurred between basic scientists and clinicians to develop, refine and implement tissue-typing techniques in clinical practice exemplifies how translational research can lead to improved outcomes.”

Jean Slutsky, Chief Engagement and Dissemination Officer at the Patient-Centered Outcomes Research Institute (PCORI) — a U.S. organization dedicated to engaging patients and providing stakeholders with real-world outcomes they can use to make informed decisions — also attests to the benefits of patient-centered approaches.

“One [2015 PCORI] research study examined the impact of a blood thinner on outcomes among stroke survivors after they left the hospital,” Slutsky says. “A traditional study might have focused only on whether patients had another stroke or side effects like bleeding — important questions, to be sure. But in this study, researchers had asked patients what outcome was most important to them and learned that they cared most about how long they could live at home without suffering complications requiring additional hospital or nursing home care. The study found that taking the blood thinner did result in more time at home.”

Potential Limitations

The discipline of translational science and the push to engage patients in research prioritization are relatively new endeavors. One potential limitation Dr. Kaufmann can foresee is the difficulty associated with finding a unified patient voice. Researchers could also abandon promising studies that may have subtle potential that isn’t obvious to patients. To prevent such complications, Dr. Kaufmann stresses collaboration and education.

“At the end of the day, it’s all about setting [projects] up so there is good communication and mutual understanding,” she says. “... There are ways to accommodate the potential research avenue that a researcher is excited about but may be too early to provide health benefits and, at the same time, meet the needs of patients and the doctors who treat them.”

Medical Diagnostics _350726315-full