Patient-centered outcomes research (PCOR) is designed to generate high-quality evidence important to patients and families, clinicians, and policymakers about treatments, services, and other health care interventions. PCOR studies have traditionally focused on understanding causal relationships among the use of health care treatments and services, treatment effects, and clinical outcomes. That focus has broadened to include a more holistic understanding of health and well-being, including the significant economic impacts of health care use on individuals, families, and their communities, such as out-of-pocket spending and informal caregiving needs. In many cases, data to study the economic impacts of health care from the perspective of individuals, families, and communities are unmeasured, not routinely collected, or unavailable for research. The growing recognition that economic factors often impact health outcomes, decision-making, and equity in health care is the impetus for the articles in this special issue of Medical Care.

The Affordable Care Act established the Patient-Centered Outcomes Research Trust Fund (PCORTF) in 2010 and structured it for use in strengthening the evidence base for decision-making.1 The trust fund provides resources to build data capacity, conduct research, disseminate research findings, train investigators on PCOR methods, and engage people with lived experience in carrying out these activities. Funding is provided by the PCORTF to the Secretary of Health and Human Services (HHS), the Agency for Healthcare Research and Quality, and the Patient-Centered Outcomes Research Institute, with each entity having a distinct yet related set of responsibilities for evidence generation, dissemination, and implementation.

The Assistant Secretary for Planning and Evaluation (ASPE) has the unique responsibility of coordinating across relevant federal health programs to build data capacity for PCOR. ASPE works in partnership with all HHS agencies on a portfolio of projects that support the collection, linkage, and analysis of data for PCOR studies. This body of work is collectively referred to as the Office of the Secretary Patient-Centered Outcomes Research Trust Fund portfolio (https://aspe.hhs.gov/collaborations-committees-advisory-groups/os-pcortf/explore-portfolio) and has led to the production of a variety of data products, including standards, algorithms, and linked datasets that would not have been possible without the PCORTF. These products support evidence generation in health and health care by HHS agencies and for departmental priorities such as reducing maternal mortality and substance use, increasing emergency preparedness, and improving the equitable delivery of health care.

The 2019 reauthorization of the PCORTF underscored the importance of assessing a full range of outcomes, explicitly expanding the scope of patient outcomes that should be considered in PCOR studies to include the potential burdens and economic impacts of the utilization of medical treatments, items, and services on different stakeholders and decision-makers respectively. These potential burdens and economic impacts include medical out-of-pocket costs, including health plan benefit and formulary design, nonmedical costs to the patient and family, including caregiving, effects on future costs of care, workplace productivity and absenteeism, and healthcare utilization.2 Following the reauthorization of the PCORTF, and in response to this new priority, HHS developed a new strategic plan for the Office of the Secretary Patient-Centered Outcomes Research Trust Fund3 based on input from a National Academies of Sciences, Engineering, and Medicine study committee4 and representatives across HHS.5 The fourth objective of the OS-PCORTF Strategic Plan focuses on addressing data capacity limitations to support a more comprehensive view of health outcomes, including improving the availability, quality, and relevance of data on economic outcomes.3

As part of its work to implement and achieve this objective, ASPE sponsored a symposium and this special issue of Medical Care to bring together multiple perspectives on building data capacity for economic outcomes in PCOR.6 The symposium was designed not only to review and discuss current efforts and challenges related to data capacity and infrastructure, as set forth in a set of invited manuscripts, but also to identify areas of importance not addressed in this work, interrogate underlying assumptions, and develop a foundation for initiating and sustaining efforts to advance data capacity for economic outcomes in PCOR studies.

The work featured in this special issue draws on the themes that emerged from the symposium. These articles highlight specific and important challenges in building PCOR data capacity on economic outcomes. However, as noted by symposium attendees, particularly patient stakeholders, these articles (and the discussions around them) often lack an explicitly patient-centered focus. That is, what are the information needs of patients related to economic impacts, and how can we ensure that efforts to build data capacity on economic outcomes align with those needs?

First, the measurement and collection of data on economic outcomes should reflect the types of questions and decisions patients, caregivers, clinicians, and policymakers face. Unfortunately, the evidence to inform these decisions and improve patient outcomes—and the data and data infrastructure needed to generate it—are generally lacking in most data sources. For example, as articles published in this issue of Medical Care have described, data are needed to understand household economic impacts7 and family decisions and trade-offs.8 The data needs for specific populations should also be considered. Palatucci and colleagues9 provide an overview of appropriate data collection on economic outcomes for individuals with intellectual disabilities, while efforts to build data capacity for economic outcomes among cancer patients and embed data collection within oncology practices are discussed by Halpern et al10 and Williams et al,11 respectively.

Beyond improving the measurement and capture of data on economic outcomes, efforts are needed to build more comprehensive data resources through data linkage and improved data sharing and access. Brown et al12 report on a review of federally funded administrative and survey data sources linked or linkable to Medicare fee-for-service claims that can be used to increase the range of outcomes included in PCOR studies. Jones and colleagues13 describe a novel effort to link Medicare and Medicaid data in North Carolina and discuss how to support the development and use of patient-centered utilization measures, the development of integrated Medicare and Medicaid programs, and the evaluation of health equity impacts. Zhang and Meltzer14 discuss their work in developing an integrated dataset of Medicare beneficiaries to better understand cost-related medication nonadherence. Moving beyond specific linked datasets to integrated data infrastructure, Bradley et al15 and Waitman et al16 provide overviews of efforts to link a state-level All-Payer Claims Database in Colorado with a cancer registry and strengthen PCORnet, the National Patient-Centered Clinical Research Network, respectively, to support the inclusion of economic outcomes in PCOR studies. The final article in this collection responds to this imperative, synthesizing the robust discussions among symposium attendees to arrive at a set of cross-cutting considerations to guide efforts to build data capacity and identify initial opportunities to expand the availability and use of relevant, high-quality economic outcomes data in PCOR.17

Although the articles in this issue highlight the potential benefits of improved data capacity for economic outcomes in PCOR, they also make it clear that much work needs to be done—particularly in supporting the paradigm shift within health economics research to include the perspectives of patients and families. The significance of this shift mirrors the initial sea change in efforts to engage patients and other stakeholders in all aspects of clinical comparative effectiveness research following the establishment of the PCORTF in 2010. With the expanded scope of outcomes in the 2019 reauthorization, engaging patients and families in identifying questions, data, and research on economic impacts will be crucial to expanding the evidence about the outcomes and effectiveness of health care and providing equitable health care. More broadly, continued collaboration within the PCOR community around these and other opportunities to advance the collection, linkage, and analysis of economic outcomes data for PCOR will be needed to realize the gains from the nation’s investment in the PCORTF and support decision-makers in their efforts not only to improve health and well-being but also to limit the economic burdens of health care.

1. Patient Protection and Affordable Care Act, Publ. L. No. 111-148, 124 Stat. 119 (2010). Accessed July 26, 2023. https://www.govinfo.gov/content/pkg/PLAW-111publ148/html/PLAW-111publ148.html 2. Further Consolidated Appropriations Act, 2020, Pub. L. No. 116-94 2019. Accessed July 26, 2023. https://www.congress.gov/116/plaws/publ94/PLAW-116publ94.pdf 3. Building Data Capacity for Patient-Centered Outcomes Research. Office of the Secretary Patient-Centered Outcomes Research Trust Fund Strategic Plan: 2020-2029. Office of the Assistant Secretary for Planning and Evaluation, U.S. Department of Health and Human Services. 2022. Accessed July 26, 2023. https://aspe.hhs.gov/sites/default/files/documents/b363671a6256c6b7f26dec4990c2506a/aspe-os-pcortf-2020-2029-strategic-plan.pdf 4. National Academies of Sciences, Engineering, and Medicine. Building data capacity for patient-centered outcomes research: Consensus Committee Report – Priorities for the Next Decade. The National Academies Press 2022. 5. Research Data Networks and Patient-Centered Outcomes Research Trends and Opportunities: Scan and Interviews with Key Informants. Accessed July 26, 2023. https://aspe.hhs.gov/sites/default/files/2021-07/PCOR%20Research%20Network%20Landscape%20Report_Final_07_14_2021_508.pdf 6. RAND Corporation. Symposium on Building Data Capacity to Study Economic Outcomes for Patient-Centered Outcomes Research. Accessed July 26, 2023. https://www.rand.org/health-care/projects/pcor/hhs-symposium.html 7. Nicholas LH. Using address data to track household financial burden of disease. Med Care. 2023;61(Supp 2):S92–S94. 8. Galbraith AA, Faugno E, Cripps LA, et al. “You have to rob Peter to pay Paul so your kid can breathe”: using qualitative methods to characterize trade-offs and economic impact of asthma care costs. Med Care. 2023;61(Supp 2):S95–S103. 9. Palatucci JS, Chakravarty S, Kratchman AL, et al. Determining economic factors that matter to people with intellectual and developmental disabilities and their caregivers: a process framework. Med Care. 2023;61(Supp 2):S104–S108. 10. Halpern MT, Ekwueme DU, Yabroff KR. Enhancing cancer economic data resources: the interagency consortium to promote health economics research on cancer (HEROiC). Med Care. 2023;61(Supp 2):S109–S115. 11. Williams CP, Liang MI, Rocque GB. Cancer-related financial hardship screening as part of practice transformation. Med Care. 2023;61(Supp 2):S116–S121. 12. Brown DS, Srinivasan M, Zott C, et al. Medicare data linkages for conducting patient-centered outcomes research on economic outcomes. Med Care. 2023;61(Supp 2):S122–S130. 13. Jones KA, Clark AG, Greiner MA, et al. Linking Medicare Medicaid claims for patient-centered outcomes research among dual-eligible beneficiaries. Med Care. 2023;61(Supp 2):S131–S138. 14. Zhang JX, Meltzer DO. Developing an integrated longitudinal dataset for patient-centered outcome measures in cost-related medication nonadherence. Med Care. 2023;61(Supp 2):S139–S146. 15. Bradley CJ, Liang R, Lindrooth RC, et al. Building data infrastructure for disease-focused health economics research. Med Care. 2023;61(Supp 2):S147–S152. 16. Waitman LR, Bailey LC, Becich MJ, et al. Avenues for strengthening PCORnet’s capacity to advance patient-centered economic outcomes in patient-centered outcomes research (PCOR). Med Care. 2023;61(Supp 2):S153–S160. 17. Timbie JW, Reynolds KA, Evans EL, et al. Advancing data capacity for economic outcomes in patient-centered outcomes research: challenges and opportunities. Med Care. 2023;61(Supp 2):S161–S165.