Summary

The Office of the Assistant Secretary for Planning and Evaluation (ASPE), in partnership with other agencies and divisions of the U.S. Department of Health and Human Services (HHS), coordinates a portfolio of projects that build data capacity for conducting patient-centered outcomes research (PCOR). PCOR focuses on producing scientific evidence on the effectiveness of prevention and treatment options to inform the health care decisions of patients, families, and health care providers, taking into consideration the preferences, values, and questions patients face when making health care choices. The data infrastructure includes data sources and functionalities that support the research. Major building blocks are the services, standards, policies, and governance that enable the use of the data.

ASPE asked the National Academies of Sciences, Engineering, and Medicine to appoint a consensus study committee to identify issues critical to the continued development of the data infrastructure for PCOR. The committee’s work will contribute to ASPE’s development of a strategic plan that will guide their work related to PCOR data capacity over the next decade.

As part of its information-gathering activities, the committee organized three workshops to collect input from stakeholders on the PCOR data infrastructure, which includes a variety of types of data, such as clinical data, research data, administrative data from payer records, and patient-provided data. This report, the second in a series of three interim reports, summarizes the discussion and committee conclusions from the second workshop, which focused on data standards, methods, and policies that could make the PCOR data infrastructure more useful in the years ahead.

Participants in the workshop included researchers and policy experts working in these areas. The first report in the series centered on emerging data needs.¹ The third report will discuss research and data collaborations.

The conclusions included in this interim report are based primarily on the input collected as part of the workshop, background documentation received from ASPE and other public sources, and the committee members’ synthesis and expert judgment regarding the input received. As an interim report based on one in a series of information-gathering activities, the scope of this report is narrowly focused on a subset of key topics relevant to the committee’s charge. The conclusions reached by the committee are, at this stage, fairly high level. After completing all of its information-gathering activities, which include but are not limited to the three workshops, the committee will also issue a final report, containing the study’s overall findings and conclusions.

DATA STANDARDS

Part of the workshop discussed in this report focused on standards for PCOR. Standards are increasingly widely used for a variety of purposes, including collecting, storing, analyzing, and exchanging data. One theme that emerged from the workshop was that these standards are most useful when they are focused on addressing a specific problem or are driven by the specific value they can contribute. The needs and norms evolve over time, and because of this, standards need to evolve too. The workshop also identified some key areas where ASPE’s role is particularly important.

• continuing to promote the development of a data infrastructure and an implementation strategy that facilitates the use of standards and access to the data;
• convening stakeholder meetings to enhance communication and work toward developing a common language for standards;

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¹https://www.nap.edu/catalog/26297/building-data-capacity-for-patient-centered-outcomes-research-interim-report.

• facilitating accessibility to the data and collaborations with existing organizations working in this area; and
• leading efforts to catalogue and exemplify data standards and analytic standards.

Learning from the work on standards happening across the globe would further advance PCOR.

METHODS

A promising area of research focuses on better understanding the longitudinal, holistic experiences of people across time and different settings, which requires matching records across databases. To balance these opportunities and concerns, it would be useful to develop a carefully thought-out strategy for linking data from a variety of sources, and to focus on strengthening the data infrastructure in additional ways that would enable longitudinal research that provides a comprehensive understanding of people’s experiences over their life course.

• simplify integration of data across the research data ecosystem;
• address challenges posed by the limitations associated with health identifiers;
• incorporate person-generated data into health data systems; and
• leverage real-world data to expand the timeline view of a person’s health-related experiences.

There is also a need for transparency and the continued refinement of best practices related to how data and methods are used. This is especially

important for emerging data sources and methods that capture more lifestyle and behavioral information than traditional clinical measurements, as well as the application of artificial intelligence methods, which could have biases confounded by patterns associated with new technologies. These considerations are also important for the use of observational data.

The workshop highlighted the importance of interpreting best practices in the dissemination of research broadly, to include not only sharing results but also making available other resources and components associated with the research process, such as the software developed for the analyses.

DATA POLICY AND OTHER DATA INFRASTRUCTURE CONSIDERATIONS

A theme that emerged from the workshop was the need to involve the people and communities whose data are being used in decisions about the data collection and data use throughout the entire research lifecycle. This is essential for building trust, which increases willingness to participate and, in turn, the likelihood that the data that are obtained will be complete, reliable, representative, and relevant to diverse stakeholders. This is particularly important for data on social determinants of health.

The existing laws and regulations that govern the use of data for research, including the Health Insurance Portability and Accountability Act of

1996, are outdated and would benefit from a critical review and updating to facilitate PCOR while preventing misuses of the data.

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