During a side conversation at our recent workshop on building a patient-centered research community, one patient advocate summed up her take on what the Patient-Centered Outcomes Research Institute represents. “PCORI is opportunity,” she said. She was describing her hopes for a change in culture and practice that repositions patients, caregivers and the broader healthcare community as valuable partners in the research process, and bases research priorities on outcomes important to patients.
As we were speaking with potential attendees in planning the workshop, a researcher picked up on the same theme. “Now, as a researcher, I realize the most important players need to be on the team: patients and their families,” he said. “I dream of a day when clinicians, researchers, policymakers, funders and patients and families all look back on today as the ‘dark ages’ of research, when we naively neglected to include the most important voice in efforts to improve healthcare delivery, patient outcomes, and health care policy.”
Different participants, from different worlds, with different perspectives. But a shared vision of what we at PCORI see as the theme that ties our work together: “research done differently.”
The workshop was not the kind of gathering any of us could recall being held previously, and we marveled at the mix of participants. There were well-known “professional patients” as well as passionate individuals new to patient-centered research, like a young man with epilepsy who ended the weekend deciding he wanted to work for us. There were caregivers – including a man from a rural area caring for his wife with breast cancer and others caring for parents with dementia. Other stakeholders came from research universities and hospitals, professionals dedicated to studying and treating rare diseases, and leaders in community-based participatory research in vulnerable urban populations. Some 150 patients, researchers and other members of the healthcare community gathered in Washington October 26-28 for our workshop, “Transforming Patient-Centered Research: Building Partnerships and Promising Models.” They came to build community, generate the “what ifs,” reimagine patient-centered research, and help us build a culture of patient-centeredness, foundational principles and a sustainable architecture for patient engagement and rigorous research.
The workshop began with real-world examples of patient/researcher partnerships that showed how research can, indeed, be done differently, including:
- A small group of mothers of children with kernicterus who partnered with researchers, and then healthcare
professionals and systems around the country, to improve outcomes for newborns. - Two brothers who created a model for unifying patients with similar conditions “to share real-world health experiences in order to help yourself, other patients like you and organizations that focus on your conditions.”
- A rancher and his wife, an educator, who were sought out by a university to unite rural communities and researchers to study health issues important to that community, including colon cancer and hypertension.
- A minority community experiencing disparate mortality outcomes that sought out a major research center to improve outcomes in heart disease, stroke and cancer.
Workshop participants rotated among five engagement sessions, each focused on a key area where PCORI is seeking ongoing stakeholder input:
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
- Identifying and Selecting Research Questions
- Reviewing Research Proposals for Funding
- Matching Patients and Stakeholders with Researchers
- Disseminating Research to the Community
- Evaluating PCORI’s Patient and Stakeholder Engagement Programs
We heard a range of suggestions that we will explore further, including how to best capture research ideas from patients and other stakeholders, the concept of “micro-grants” for building partnerships between patients and researchers, enhanced patient participation in funded proposals, tools for matching researchers with patient partners, and even the creation of a PCORI Ambassador initiative. And we were repeatedly challenged to “listen,” be fully transparent, responsive and inclusive.
We’ll compile the conversations from each session into a detailed report that we’ll release in about a month. We’ll also post pictures, videos and other materials that paint a detailed picture of the workshop and our next steps. Be sure to watch our website to see these reports and sign up for our email alerts to stay current on everything we do. For information on how to work with us, send me a note at [email protected].
The challenge and opportunity of shifting the research culture to one that is truly patient-centered, that designs research with and welcomes patients, caregivers and their organizations as partners, belongs to all of us.
We look forward to hosting future workshops that continue to connect patients, clinicians, researchers, industry and all healthcare community members in new ways. While each group, and each individual, brings a different perspective, we all hope to improve research with the same goal: to improve patient outcomes.
I want to thank our workshop planning committee, which helped plan the event. This group of dedicated patients, patient advocacy organizations, and members of the PCORI Board and Methodology Committee was instrumental in ensuring that our event was focused, productive and above all else patient-centered. Information about all of our working group members is provided in the Workshop Leaders document posted on the event page.
This workshop was just the beginning. I am confident that, as a community, we can address this challenge – and seize this singular “opportunity.”
