Transforming Research by Partnering With Patients - A View from the Field

To mark our fifth anniversary, we asked patients, caregivers, clinicians, and researchers to describe their experiences with engagement of healthcare stakeholders in research and share their thoughts about building a patient-centered clinical effectiveness research community. The views expressed are those of the author and not necessarily those of PCORI.

How did you get involved in clinical research?

Since birth, I’ve had a heart condition, called patent foramen ovale, or PFO, that can result in severe migraines. When doctors couldn’t help me, I volunteered for a study investigating the problem. I was randomly assigned to receive an implanted heart device that fortunately stopped my migraines, but unfortunately it led to nearly every possible complication. The study had several undesirable aspects and was ultimately stopped for low enrollment. I thought that if the researchers had engaged patients in the design phase, they would have had a better trial and more patients would have chosen to participate.

After that study experience, I founded the nonprofit PFO Research Foundation. I later became director of stakeholder engagement for the National Institute of Health’s Clinical Trials Transformation Initiative, an FDA public-private partnership that engages stakeholders in the clinical trials enterprise to identify and promote practices to increase the quality and efficiency of clinical trials. Now, I am also director of patient engagement for the Clinical and Translational Sciences Awards program and have two associations with PCORI. I am a patient partner on a PCORI-funded study, and a member of the executive leadership committee for PCORnet, the National Patient-Centered Clinical Research Network.

How does your work with PCORnet advance your efforts to bring patients’ voices more meaningfully into the research process?

Patient advocacy leaders and many others are concerned about the skyrocketing costs and slow pace of research and therapy development, as well as the challenges of engaging patients in research.  PCORnet is developing a research infrastructure—built by patients, researchers, health systems, medical product developers, and government, including regulatory agencies—aimed to reduce the costs of comparative clinical effectiveness research as it engages patients in all aspects of research.

We have taken our cues from patients and engage diverse populations. We offer many opportunities for engagement, including research prioritization, study participation, or serving as a member of a study team. PCORnet makes a point of reaching out to ethnic and racial minorities and underserved patients, giving them a voice in shaping research. Within the PCORnet community, patients and caregivers work hand-in-hand with researchers to ensure that studies are relevant and timely. Engagement of the patient community in clinical in research design and conduct helps ensure that the results will be applicable to real-world populations.

We at PCORnet maintain high standards for patient privacy and public trust by making patients and public equal partners with researchers in creating policies that meet national standards and practices that govern the research enterprise. We are creating a common data model, which can be deployed behind the firewalls of health systems and research networks. We are also developing innovative tools that allow patients to express their individual preferences related to consent for participation in research. For example, patients can choose to participate in the network by simply donating de-identified data or by taking an active role in prioritizing or developing research studies.

What do you see as some of PCORnet’s major accomplishments? And what are the most important next steps?

We have made great progress building a multi-stakeholder community of equal partners serving diverse populations with multi-faceted opportunities for engagement in research. Thirty-four research networks are currently participating. These include Clinical Data Research Networks (CDRNs), which are based in healthcare systems such as hospitals or health plans and collect information during the course of routine patient care, and Patient-Powered Research Networks (PPRNs), operated by groups of patients and their partners.

Now, it’s time to begin generating useful evidence for the healthcare community.

I am excited about the large studies we have recently begun to test-drive our network. The first is a three-year, $14 million clinical trial comparing the benefits and risks of two commonly used doses of aspirin for preventing heart attacks and strokes in people with heart disease. Involving researchers, clinicians, and patients in seven PCORnet partner networks, it will enroll and follow as many as 20,000 participants.

We also have two studies on prevention and treatment of obesity. One study will examine three main methods of weight-loss surgery to provide patients and those who care for them with information on the comparative benefits and potential risks of each procedure. The other study will look at how different types of antibiotics given to infants and young children may affect their weight in later childhood.

We look forward to performing additional research that answers questions of importance to patients and researchers, and we ask other parties in the research enterprise to join us. Collaboration will lead more rapidly to new therapies and answers for patients’ questions.

You’ve been working for a long time to improve the way clinical research is conducted and see that patient experiences are at the center of that process. How much progress have we made? How much further do we have to go?

We are at a tipping point. The research system is changing. I want to be sure that patients have a voice in building the research system we want. We need timely access to data and understand that there’s a lot of complexity to that. Sometimes change seems incremental, but in the end we can achieve transformation. Only when we abandon inefficient practices and recognize all stakeholders as equal partners will we achieve a truly innovative system of rapid research.