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 its 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 third in a series of three interim reports, summarizes the discussion and committee conclusions from the third workshop, which focused on ways of enhancing collaborations, data linkages,
and the interoperability of electronic databases to 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 centered on emerging data needs1 and the second report on data standards, methods, and policy.2
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.
FEDERAL PARTNERSHIPS
The workshop discussed in this report included several stakeholder groups that are involved in collaborations focused on PCOR. While it was not possible to discuss many important collaborative projects, the first session, which was held with federal agency representatives, highlighted several key areas where additional collaborative work could continue to build and strengthen the PCOR data infrastructure.
CONCLUSION 2-1: Collaboration among federal agencies and between federal agencies and other partners (such as states, patient groups, and others) is essential for continuing to build the patient-centered outcomes research data infrastructure. The areas where additional collaboration would be particularly useful include the following:
- Increasing consistency in the use of standards for data interoperability and element definitions;
- Addressing barriers that hinder data linkages, such as the limitations associated with health identifiers and mitigating potential selection biases resulting from linkage error;
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1https://www.nap.edu/catalog/26297/building-data-capacity-for-patient-centered-outcomes-research-interim-report.
2https://www.nap.edu/catalog/26298/building-data-capacity-for-patient-centered-outcomes-research-interim-report.
- Balancing the burden of the data collections and disclosure risks with the value of the datasets;
- Communicating the usefulness of the data collections to those who are asked to provide data about themselves and those who collect the data;
- Promoting discussion and education about fitness for use of the data; and
- Working with stakeholders and patients to promote sharing of data.
While there is frequent collaboration among HHS partners on PCOR data infrastructure work, and ASPE’s public website contains a comprehensive list of past and current projects funded from the Patient-Centered Outcomes Research Trust Fund, additional dissemination efforts focused on external stakeholders could further increase the usefulness of these investments.
CONCLUSION 2-2: There is a need to increase awareness among all stakeholders about new data infrastructure developments funded by the Patient-Centered Outcomes Research Trust Fund. Increased awareness will enhance the efficiency and effectiveness of research, which will increase the impact of the investments made in infrastructure development.
STATE-LEVEL DATA AND COLLABORATIONS
Many states have robust data collection systems and can produce information that is useful to state and local policy makers. State-generated data are also valuable at the national level, including for answering broader questions about issues that may be influenced by local policy, such as health care access and disparities.
CONCLUSION 3-1: There are opportunities to learn from what states have accomplished in building data capacity.
The data collected, their quality, and ease of access to the data vary by state. Challenges associated with access make it particularly difficult to use state-generated data for research at the national level.
CONCLUSION 3-2: The usefulness of data available for Patient-Centered Outcomes Research could be increased by the sharing and adoption of best practices among the states for the data collected, their quality, and ease of access.
CLINICAL TRIAL NETWORKS AND COLLABORATIONS
The session on clinical trial networks and collaborations illustrated the need for better integration between clinical care and research in ways that align differing interests and are mutually beneficial. Better integration can improve the data available for patient care as well as the data available for research.
CONCLUSION 4-1: Infrastructure investments could enhance the utility of data routinely generated in the course of care for clinical trials.
PUBLIC-PRIVATE PARTNERSHIPS
While the benefits of data sharing are clear, the workshop also underscored the risks involved for the organizations providing the data.
CONCLUSION 5-1: Successful partnerships across health care systems require participant trust, clear evidence of mutual benefit, and the ability to control risk.
COLLABORATIONS WITH PATIENT GROUPS
Collaborations with patient organizations can help to address patient concerns about participating in research studies and to build patient engagement, which is important for achieving a patient-centered approach. Disease registries directed by patient groups can be a particularly useful additional source of data, one that provides information that would not be available to researchers otherwise.
CONCLUSION 6-1: Patient groups can be helpful partners in all aspects of Patient-Centered Outcomes Research, including engaging patients in order to improve research participation and the impact of results.
CONCLUSION 6-2: 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.
