A patient’s perspective on a patient-centered conference: Highlights from #PCORI2015 (Part 2)

Check out my summary of Day 1 of PCORI's Annual Meeting.

Patients are changing from research subjects to research partners. -Dr. Harlan Krumholz, PCORI board of governors and faculty member at Yale School of Medicine

Day 2

Dr. Joe Selby, executive director of the Patient-Centered Outcomes Research Institute, opened day 2 the PCORI Annual Meeting by giving an overview of the type of research PCORI funds: patient-centered comparative effectiveness research. CER, he said, compares two or more options about things that matter to patients. According to Dr. Selby, this research should be conducted in "real world" settings and should ideally pay attention to differences in effectiveness for different sub-groups of people (e.g. does a particular treatment work better for women than men).

When PCORI began awarding research contracts just a few years ago, most of the funding they offered fell into four broad categories: (1) assessing prevention, diagnosis, and treatment options; (2) addressing disparities; (3) improving health care systems; and (4) communication and dissemination research. Now, PCORI funding mechanisms are becoming increasingly targeted, addressing specific diseases or other topics that have been identified as high priority. These include programs that focus on asthma, MS, and reducing long-term opioid use, among others. PCORI has also become increasingly interested in funding large studies that better represent the entire population.

The morning's keynote presentation was given by Dr. Victor Montori, an endocrinologist and health services researcher at the Mayo Clinic. Dr. Montori gave a compelling presentation on the importance of involving patients in decision making. What's best for a person, he said, depends on what that person values. Dr. Montori also lamented the curent research funding process, which leaves about 85% of scientific proposals unfunded. To do the type of large studies PCORI aims to fund, he argued, research institutions need to end competition and encourage collaboration instead.

During the following panel, which included a number of stakeholders from various health care organizations, Nancy Brown of the American Heart Association said that if patients really understood how the research process worked, they would be horrified. She also said that people don't want to waste their time thinking about their health--it's only when they become sick that health care becomes the center of everything. In the same panel, former U.S. congressman Tony Coelho spoke about the role patient advocacy organizations play in bringing the patient experience to our legislature. He also noted that we all need to advocate for PCORI so that it doesn't disappear in 2018.

After a series of several plenary presentations, I was eager to break out into smaller group sessions. The two breakout sessions I attended on Day 2 highlighted examples of successful awardees from the Communication and Dissemination Research (CDR) program and the Eugene Washington Engagement Awards. In the CDR panel, shared decision-making tools featured prominently as a means of improving communication between patients and their doctors. Some awardees used focus groups to develop and refine their decision-making tools. Research teams found it was crucial to solicit feedback from providers like doctors and nurses in addition to patients so that these tools could realistically be used in the clinic. In the second breakout session, Engagement Award recipients shared examples of "pre-research" activities they had conducted. Some projects used Engagement funds to conduct a needs assessment in their target population. Other projects used their award to build a curriculum to train people to better understand and conduct patient-centered comparative effectiveness research.

During the final breakout session of day 2, I presented as part of a panel discussion on the Pipeline to Proposals. The Pipeline program provides funding to individuals or groups who would not ordinarily receive research funding from PCORI, and trains us step-by-step, so that--theoretically--in three years we will have built the foundation for a successful, full-scale PCORI award application. (Read more about our project.) I enjoyed participating in this session because it gave me a much-needed opportunity to connect with follow Pipeline awardees, but also because I believe this program is truly emblematic of the type of patient-driven research collaborations PCORI was designed to support.

A recent Time magazine article (that I found in the airport on my way home from the PCORI conference) addresses a patient-centered research question about breast cancer treatment.

Day 3

Day 3 of the meeting featured a keynote presentation by NIH director Dr. Francis Collins. Even though PCORI is technically a nonprofit entity, not a federal agency like the National Institutes of Health, it was impactful to have the head of AHRQ and the head of NIH lend their time to this conference. I think it helps legitimize PCORI as a credible, scientifically rigorous organization, even though the work they fund is quite a bit different.

On day 3, I attended two very different breakout sessions: one on comparative effectiveness research methods and one on how to use your patient story to make a difference in research.

Over the past few years, PCORI has developed comprehensive methodology standards that govern all aspects of the proposal, from how to involve patients in the research process to how to analyze the data collected during a study. The purpose of following these standards is to ensure research findings are valid and can be replicated. Scientific methods are evaluated based on data quality, study design (e.g. randomized controlled trial or observational study), and how the data is interpreted. The PCORI staff member who lead the session emphasized that, while there is no such thing as the perfect study--there will always be trade-offs--it's important to be cognizant of where weaknesses might be so that you can consider how those weaknesses might impact your results.

The last session I participated in was by far the most hands-on. PCORI's engagement staff spoke about storytelling as a "method" of sparking engagement and collaboration. They also shared a tool they created to walk patients through the process of teasing out bits of their patient story that might be relevant to a research question. While the tool was still in draft form, I loved the concept. I also loved to have something tangible I could take home with me after the conference given that so many of the other sessions were very philosophical and not immediately applicable to my daily work.

Finally took the time to do some sightseeing while visiting Washington D.C.

Major themes and takeaways

• Communicating research results back to the community is essential for building and maintaining trust (bonus points for involving patients and other stakeholders in developing a plan for sharing research results).
• PCORI funding announcements are becoming increasingly more targeted and aimed at creating large, pragmatic clinical trials that more broadly reflect the "real world."
• Rigorous research methods and meaningful patient engagement are equally important to PCORI, even though they can sometimes feel at odds with one another. Many applications they reject are dinged, though, because of their lack of the latter.
• There's no one-size-fits-all model for engagement, but here are some activities that have been successfully used by PCORI awardees to create meaningful engagement: 
• (1) Including a patient partner as a co-investigator on the project and compensating them financially as such,
• (2) Include patients, caregivers, and other stakeholders like physicians on project advisory committees, and
• (3) Address research questions of interest to patients as identified by online surveys, focus groups, or other meetings.

As I mentioned in my first recap post, overall it was a great event. I'm glad I was given the opportunity to attend. I also look forward to seeing further progress towards socializing and truly implementing patient-centered principles in research and clinical practice.

To learn more or view archived videos from the 2015 PCORI Annual Meeting, visit www.pcori.org.