In connection with the NIH Pragmatic Trials Collaboratory’s annual Steering Committee meeting and 10th anniversary celebration, we interviewed Dr. Christy Zigler and Dr. Emily O’Brien, asking them to reflect on the role of the Patient-Centered Outcomes Core in helping the NIH Collaboratory Trial teams plan and implement their trials, and to discuss their focus for the Core’s future contributions to pragmatic clinical trials.

Based on your experience working with the NIH Collaboratory Trials, what are some of the common challenges of patient-centered outcomes?

Based on our experience, the challenges that NIH Collaboratory Trials face with patient-centered outcomes can occur across several project periods. In the planning phase, we see a number of basic questions about patient-reported outcomes (PROs), including “What outcomes should be measured?” and “What measures should be used to capture these outcomes?”

Once appropriate measures have been identified, there are often challenges related to implementation of these measures and resources. For example, project teams are usually interested in understanding the availability of existing PROs within the electronic health records that study sites could leverage, as well as which measures have appropriate translations or cross-cultural validation versions that are already established.

We also have come across some interesting Core-crossing challenges, including ethical considerations for collecting PROs (Who is responsible for monitoring patient responses?) and data collection methods (Should PROs be integrated within the EHR systems or in a separate repository?). Finally, sites are sensitive to patient and system burden of PRO collection, how to reduce the likelihood of missing data, and how patient-centered outcomes fit into the overall statistical analysis plan.

What strategies have NIH Collaboratory Trials used to overcome these barriers?

So far, the NIH Collaboratory Trials have been able to share resources and test out some direct strategies to overcome these challenges. For example, some projects have created outcome-specific data silos and also tested different administration methods. The Cores have also been able to provide insight into best practices for making these decisions, such as when to use particular formats for PRO administration (eg, in clinic, via telephone, via text, via electronic capture).

How are the NIH Collaboratory Trials’ experiences with patient-centered outcomes helping the field of pragmatic research?

We’ve really be able to pull together the lessons learned around patient-centered outcomes in pragmatic research to help inform the field. In particular, even for NIH Collaboratory Trials that have not conferred directly with our Core, we have been able to interview them about their work. For example, we highlighted critical advice from the Guiding Good Choices for Health (GGC4H) NIH Collaboratory Trial on the Living Textbook. Their real-world experience with determining and utilizing patient-centered outcomes provide a road map for other investigators.

What do you think the Core can contribute over the next decade?

Our wish list for future contributions includes:

• More cross-core collaboration—specifically, conferring with the Biostatistics and Study Design Core on quantitative measurement questions around topics like meaningful change and longitudinal responsiveness within the context of pragmatic trials.
• More focus on lessons learned from NIH Collaboratory Trial teams who have worked through their enrollment periods and have successful response rates. This real-world knowledge is very valuable to the field.
• More focus on how to create equity within patient-centered outcomes in pragmatic trials.