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, 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.
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 its work related to PCOR data capacity over the next decade. This report summarizes the committee’s findings and conclusions in the areas that could benefit from being prioritized as part of ASPE’s work over the next decade. The report also offers input on strengthening the overall framework for building the data infrastructure over the coming years.
The committee’s primary information-gathering mechanisms for this study were three workshops, which focused on obtaining broad inputs from data users and other stakeholders on topics that the committee and ASPE had jointly determined to be particularly relevant to the strategic planning. The topics included (1) data user needs; (2) data standards, methods, and policies; and (3) collaborations, data linkages, and interoperability
of electronic databases. The committee has issued three interim reports, which contain conclusions based on the three workshops. The full text of these reports is included in Appendixes B through D and can be consulted for additional details on the discussions that took place at the workshops.
This report is the final product of the committee’s work and represents a synthesis of all of the committee’s information-gathering activities, its review of additional documentation about the data infrastructure, its deliberations, and its integrated judgment of the input received. The report’s conclusions reflect the conclusions of the interim reports, but they are further integrated and combined here as part of a more holistic and streamlined discussion.
Below we summarize the insights and overall conclusions reached by the committee at the end of the study. Due to the complex nature and many important elements of the data infrastructure, the conclusions cover a lot of ground. However, the committee would like to highlight two overarching conclusions, which provide additional context for the more detailed conclusions. First, the committee concluded that the PCOR data infrastructure in the next decade would benefit from a broader focus on people and communities (Conclusion 3-1). Using this lens will support further development of the infrastructure by highlighting data gaps (e.g., about a person’s context beyond a clinical setting, or new types of data), taking a longitudinal perspective, linking fragmented datasets (e.g., different types of data from a variety of sources and over time), identifying the questions that matter most to people, and advancing research methods that enable this broader focus.
Second, the committee concluded that greater attention to how the uses of the PCOR data infrastructure are shaping knowledge, practice, and policy can meaningfully inform future investments in the infrastructure (Conclusion 3-27). This requires a more dynamic perspective, expanding the voices that provide guidance, finding the right balance between protecting privacy and advancing knowledge, and proactively identifying areas of investment to ensure that the right work gets done.
FOCUS ON THE PERSON AS A WHOLE
The committee noted the importance of taking a holistic view of an individual’s health rather than limiting the perspective, and the data acquired, to that captured about an individual in a clinical setting. Accordingly, the committee believes that broadening the focus of the PCOR data infrastructure from patient-centered to person-centered would enhance the goals and data requirements needed to conduct meaningful outcomes research. Everything that has been learned about the contributions of social determinants of health indicates that ignoring the health impacts of where individuals live, work, and play also impedes meaningful understanding of
important intervention targets to improve health. In other words, expanding the focus from populations to include information about the characteristics of their communities would be of further benefit. A person-centered approach incorporates into the data critical influences beyond the health care processes when people are patients.
The distinction between a person-centered approach and a patient-centered one is not intended to imply that the roles of patient and person are necessarily separate but rather to underscore the broader context that might be missed when the focus is exclusively on the person as a patient. This shift in perspective could be transformative for both research and health care.
CONCLUSION 3-1: Broadening the focus from the patient to the person more generally and from populations to communities would enable a more comprehensive approach to the data infrastructure and a better understanding of the outcomes that matter to people.
INCLUDING HIGH-PRIORITY TYPES OF DATA IN THE DATA INFRASTRUCTURE
The committee identified several emerging data needs and stakeholder priorities that are not well met by the current data infrastructure. A theme that emerged from the workshops was the magnitude of the gaps in the data that are available to better understand and address health disparities. The cost of care and its impact on the quality and length of life of people in the United States emerged as another topic area suffering from substantial gaps in the available information. The workshops also highlighted the need for and the underutilized potential in performing linkages to existing mortality data. This might be an area where enhancements to linkages in the data infrastructure could have a high impact and be particularly feasible.
CONCLUSION 3-2: A variety of data types were identified that are less likely to be available or easily accessible in the PCOR data infrastructure, including data on mortality, cost of care, social determinants of health, and disability status, as well as other characteristics of people associated with disparities in health outcomes. Increased attention to filling gaps in the availability of these data will enhance the utility of the infrastructure for answering questions that matter to people and will enable research on potential intervention targets.
A fundamental reason for the data limitations that make it difficult to answer questions important for PCOR is that most of the data available for research are collected for payment or treatment purposes rather than
availability of these data for research is crucial, opportunities also exist to incorporate additional and newer data sources into the data platforms used for research.
CONCLUSION 3-3: An area with opportunities for additional expansion is the collection of patient- and person-generated data and the routine integration of these data into data platforms that can be used both for research and for other purposes, including regulatory decision making and to inform shared decision making.
CONCLUSION 3-4: Patient-directed disease registries can be a source of in-depth, longitudinal, prospective clinical and patient-reported data that are not available from other data sources.
The usefulness of the data and the data infrastructure could be further enhanced by adopting a longitudinal perspective on a person’s journey through the health care system and through life events that have a relevance to health more broadly.
CONCLUSION 3-5: Assembling a comprehensive longitudinal record of individuals’ health journeys, which also includes the social context of their lives to the extent possible, would facilitate more far-reaching outcomes research.
ADDRESSING FRAGMENTATION
The data that exist for PCOR are collected and curated in a variety of databases across a fragmented health system. These data silos are a major barrier to research as well as to increasing the usefulness of the information available for decision making more broadly. Several areas of promising work aimed at overcoming these barriers could result in a better integrated data infrastructure.
CONCLUSION 3-6: The data available for PCOR are fragmented across a variety of databases. Expanding data linkages could greatly increase the usefulness of these data for research.
CONCLUSION 3-7: Collaboration among federal agencies and between federal agencies and other partners to address barriers that hinder data linkages, such as the limitations associated with the lack of unique health identifiers and patient or person matching, will improve the PCOR data infrastructure. The usefulness of data available for
PCOR could further be increased by sharing and adopting best practices among the states concerning the collection of data, data quality, and ease of access.
DATA NOT DESIGNED TO ADVANCE KNOWLEDGE
Given that the PCOR data infrastructure relies mostly on data that were not collected primarily for research purposes, developing standards for clinical data and enhancing the interoperability of data systems would facilitate the use of data for research. ASPE has an important role to play in this area.
CONCLUSION 3-8: Standards are most useful when their development is driven by their potential uses and a clear concept of the value they can contribute.
CONCLUSION 3-9: Taking an international perspective is important for the development of a PCOR data infrastructure; in particular, the infrastructure focused on standards would benefit from building on work that happens internationally.
CONCLUSION 3-10: ASPE, in collaboration with other partners and stakeholders, could add significant value in the area of standards for PCOR by:
- continuing to promote the development of a data infrastructure and an implementation strategy that facilitate the use of standards and access to the data;
- convening stakeholder meetings to enhance communication and work toward developing a common language for standards;
- facilitating access to the data and collaborations with existing organizations working in this area;
- leading efforts to catalogue and exemplify data standards and analytic standards for a holistic view of individuals’ health; and
- increasing consistency in the use of standards for data interoperability and element definitions.
CONCLUSION 3-11: Prioritizing projects that address fidelity or use of standards may convey greater value for the PCOR infrastructure than developing new standards.
GOVERNING DATA ACCESS
Data access and privacy considerations were a recurring theme during the committee’s information-gathering activities. At the same time, it appears that relatively few projects have been funded in this area.
CONCLUSION 3-12: This is an opportune time to revisit and update the legislation and rules governing data privacy and the sharing of data for research.
CONCLUSION 3-13: Governance challenges that create barriers to developing the PCOR infrastructure can be found at all levels of the system. Data availability could be increased by exploring challenges at the local level, including variable interpretations of federal laws and regulations, and by identifying approaches to address those challenges.
DATA ACCESS OPTIONS
While a variety of mechanisms exist for accessing PCOR-relevant data, these processes could all benefit from additional streamlining to facilitate data use. One theme that emerged from stakeholder input was the importance of being transparent about how the data will be used, building trust, and working closely with patient groups and communities. Stakeholder input also highlighted reasons why some organizations might be reluctant to share data, pointing toward considerations necessary for successful data sharing agreements.
CONCLUSION 3-14: Investments in identifying mechanisms for facilitating the ability of researchers, patients, and other people to access data will contribute to increased use of the PCOR infrastructure.
CONCLUSION 3-15: Building and maintaining trust among the people and communities whose data are being sought for research is essential for producing high-quality data, and patient groups can be helpful partners in these efforts. Including representatives of patients and other people in the research process to understand how to measure health impacts that matter to individuals is an important component in building trust. Providing value back to data donors, such as through the sharing of research results, could help underscore the importance and benefits of the information to stakeholders, including individuals, families, clinicians, and communities, in addition to enabling them to use the information in ways they find relevant. These uses could play a
particularly important role in reducing health disparities, complementing research efforts in this area.
CONCLUSION 3-16: Successful data sharing partnerships across health care systems and government agencies require participant trust, clear evidence of mutual benefit, and the ability to control risk.
ADVANCING RESEARCH PRACTICES AND ANALYTIC METHODS
Advances in the methods used for PCOR have led to renewed interest in aspects of how the research is carried out. The committee identified some areas that could benefit from additional attention in the coming years.
CONCLUSION 3-17: PCOR products would be enhanced by investing in methods that are essential for the conduct of PCOR, such as including persons throughout the research continuum, addressing problems of missing data, improving study designs, ensuring appropriate inference from methods utilizing observational data, and addressing structural bias in data systems and studies.
CONCLUSION 3-18: Applying best practices to the analytic methods used in PCOR is important to facilitate the reliability and reproducibility of study results.
CONCLUSION 3-19: The results of PCOR are only replicable and most useful when the underlying data and comprehensive research documentation (such as analytic code) are made available for use by others.
PROJECT SELECTION TO SUPPORT THE DATA INFRASTRUCTURE FRAMEWORK
In addition to the conclusions about specific aspects of the PCOR data infrastructure that could benefit from more work in the years ahead, the committee also offers several big-picture observations on project selection. Among the types of projects most likely to enable the data infrastructure to make progress are two general categories of projects that have a high expected return on investment.
CONCLUSION 3-20: The development of the data infrastructure might be enhanced and critical gaps could be filled by proactively identifying necessary projects in areas that examine the overall framework for the
PCOR data infrastructure, particularly in the context of broader issues such as the balance between privacy and increased data use.
CONCLUSION 3-21: Investments in areas unlikely to be funded or developed by other entities may have a particularly high value.
CONCLUSION 3-22: Investments in projects that have potential use and application beyond the condition or disease for which they are proposed will accelerate the use of the infrastructure.
DISSEMINATION OF RESULTS AND USE OF THE DATA INFRASTRUCTURE
Over the years, increasing efforts have been made to disseminate information about the PCOR data infrastructure and PCOR studies. The discussions with stakeholders, however, revealed that awareness of the work could be further increased by additional attention to dissemination.
CONCLUSION 3-23: There is a need to increase awareness among all stakeholders about new data infrastructure developments funded by the Office of the Secretary PCOR Trust Fund. Increased awareness will enhance the efficiency and effectiveness of research, which in turn will increase the impact of the investments made in infrastructure development.
CONCLUSION 3-24: Investments in implementing and disseminating infrastructure tools and products will accelerate the achievement of overall PCOR infrastructure goals.
CONCLUSION 3-25: Dissemination and translation of the research findings could be greatly enhanced by using forms of communication that are relevant to those outside the research community.
UPDATING THE DATA INFRASTRUCTURE FRAMEWORK
While it is clear that the projects funded through the Office of the Secretary PCOR Trust Fund (OS-PCORTF) are well targeted toward further developing and enhancing key aspects of the PCOR data infrastructure, that infrastructure has not reached its full potential. It is not yet able to provide data that can answer the questions that matter to people and enable them to make informed decisions. While ASPE regularly obtains additional input from others, such as researchers and patient advocates, the lack of their representation on the OS-PCORTF Leadership Council likely limits input
on data infrastructure needs. The committee’s work identified several ways in which the overall approach to thinking about the data infrastructure could be strengthened.
CONCLUSION 3-26: Explicitly focusing on improved health as the goal of the PCOR infrastructure may be a useful way to prioritize projects and target infrastructure investments.
Finally, the committee’s work underscored the need for additional feedback and metrics that would enable ASPE to assess the impact of the investments in the data infrastructure.
CONCLUSION 3-27: A tighter feedback loop with the external end users and developers of evidence would enhance the value of data infrastructure investments. Examining what evidence was generated due to ASPE interventions and identifying what impact it had on policy and knowledge would also help close the gaps observed in realizing the potential of the PCOR data infrastructure.
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