National Academies Press: OpenBook
« Previous: Summary
Suggested Citation:"1 Introduction." National Academies of Sciences, Engineering, and Medicine. 2021. Building Data Capacity for Patient-Centered Outcomes Research: Interim Report One - Looking Ahead at Data Needs. Washington, DC: The National Academies Press. doi: 10.17226/26297.
×
Page 5
Suggested Citation:"1 Introduction." National Academies of Sciences, Engineering, and Medicine. 2021. Building Data Capacity for Patient-Centered Outcomes Research: Interim Report One - Looking Ahead at Data Needs. Washington, DC: The National Academies Press. doi: 10.17226/26297.
×
Page 6
Suggested Citation:"1 Introduction." National Academies of Sciences, Engineering, and Medicine. 2021. Building Data Capacity for Patient-Centered Outcomes Research: Interim Report One - Looking Ahead at Data Needs. Washington, DC: The National Academies Press. doi: 10.17226/26297.
×
Page 7
Suggested Citation:"1 Introduction." National Academies of Sciences, Engineering, and Medicine. 2021. Building Data Capacity for Patient-Centered Outcomes Research: Interim Report One - Looking Ahead at Data Needs. Washington, DC: The National Academies Press. doi: 10.17226/26297.
×
Page 8
Suggested Citation:"1 Introduction." National Academies of Sciences, Engineering, and Medicine. 2021. Building Data Capacity for Patient-Centered Outcomes Research: Interim Report One - Looking Ahead at Data Needs. Washington, DC: The National Academies Press. doi: 10.17226/26297.
×
Page 9
Suggested Citation:"1 Introduction." National Academies of Sciences, Engineering, and Medicine. 2021. Building Data Capacity for Patient-Centered Outcomes Research: Interim Report One - Looking Ahead at Data Needs. Washington, DC: The National Academies Press. doi: 10.17226/26297.
×
Page 10
Suggested Citation:"1 Introduction." National Academies of Sciences, Engineering, and Medicine. 2021. Building Data Capacity for Patient-Centered Outcomes Research: Interim Report One - Looking Ahead at Data Needs. Washington, DC: The National Academies Press. doi: 10.17226/26297.
×
Page 11
Suggested Citation:"1 Introduction." National Academies of Sciences, Engineering, and Medicine. 2021. Building Data Capacity for Patient-Centered Outcomes Research: Interim Report One - Looking Ahead at Data Needs. Washington, DC: The National Academies Press. doi: 10.17226/26297.
×
Page 12

Below is the uncorrected machine-read text of this chapter, intended to provide our own search engines and external engines with highly rich, chapter-representative searchable text of each book. Because it is UNCORRECTED material, please consider the following text as a useful but insufficient proxy for the authoritative book pages.

PREPUBLICATION COPY, Uncorrected Proofs 1 Introduction The Office of the Assistant Secretary for Planning and Evaluation (ASPE), in partnership with other agencies and divisions of the United States Department of Health and Human Services (HHS), coordinates a portfolio of projects that build data capacity for conducting patient- centered outcomes research (PCOR). The PCOR data infrastructure provides decision makers with objective, scientific evidence on the effectiveness of treatments, services, and other interventions used in health care. This research is frequently focused on analyzing existing data to study questions and provide objective information for the purpose of informing real-world health care decisions. BACKGROUND The legal framework that established funding for research on the outcomes and effectiveness of treatments and health care interventions dates back to the 2003 Medicare Prescription Drug, Improvement, and Modernization Act. This act provided authorization for the Agency for Healthcare Research and Quality (AHRQ) to support research comparing the outcomes and effectiveness of treatments and clinical approaches and to disseminate the findings from this research. In 2009, the American Recovery and Reinvestment Act provided additional funding to AHRQ, the National Institutes of Health, and HHS for research that compares the effectiveness of medical options. In 2010, the Patient Protection and Affordable Care Act provided further authorization for research that assists patients, clinicians, purchasers, and policy makers in making informed health decisions. To facilitate patient-centered outcomes research, in 2010 Congress established the Patient-Centered Outcomes Research Trust Fund (PCOR Trust Fund) with the Department of Treasury. The goals of the PCOR Trust Fund are to fund PCOR research, disseminate research findings, and develop a data infrastructure for PCOR. The PCOR Trust Fund has been reauthorized through 2029, through H.R.1865 of the Further Consolidated Appropriations Act of 2020. The most recent statute specified intellectual and developmental disabilities, as well as maternal mortality, as research priorities. The statute also called for PCOR studies to include consideration of the full range of outcomes data. Specifically, the law states that: Research shall be designed, as appropriate, to take into account and capture the full range of clinical and patient- centered outcomes relevant to, and that meet the needs of, patients, clinicians, purchasers, and policy-makers in making informed health decisions. In addition to the relative health outcomes and clinical effectiveness, clinical and patient- centered outcomes shall 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, non-medical costs to the 5

PREPUBLICATION COPY, Uncorrected Proofs patient and family, including caregiving, effects on future costs of care, workplace productivity and absenteeism, and healthcare utilization.1 The bulk of the PCOR Trust Fund funding (80 %) is allocated for research and is made available through the Patient-Centered Outcomes Research Institute (PCORI), a nongovernmental organization established by Congress for this purpose. Approximately 16 percent of the PCOR Trust Fund funding is set aside for disseminating research findings, incorporating findings into clinical practice, and training researchers in patient-centered outcomes research. The agency overseeing this work is AHRQ. The remaining funding, which constitutes 4 percent of the PCOR Trust Fund, is allocated for building data capacity for PCOR and is overseen by ASPE. Specifically, Section 937(f) of the Public Health Service Act instructed the Secretary of HHS to: … provide for the coordination of relevant Federal health programs to build data capacity for comparative clinical effectiveness research, including the development and use of clinical registries and health outcomes research networks, in order to develop and maintain a comprehensive, interoperable data network to collect, link, and analyze data on outcomes and effectiveness from multiple sources including electronic health records.2 Figure 1-1 shows how the PCOR funding and work is allocated across the three entities. This National Academies study is focused on issues relevant to ASPE’s continued work on the PCOR data infrastructure, in other words, on the priorities for the use of the 4 percent of the funding that is allocated for HHS. As the coordinating agency for the data infrastructure investment portfolio across HHS agencies, ASPE guides the PCOR data infrastructure’s strategic framework and vision, sets funding priorities, and coordinates interagency workgroups. ASPE’s work is assisted by a Leadership Council for the PCOR Trust Fund, which includes representatives from other HHS agencies, including the Administration for Children and Families, the Administration for Community Living, the Assistant Secretary for Preparedness and Response, the Agency for Healthcare Research and Quality, the Centers for Disease Control and Prevention, the Centers for Medicare and Medicaid Services, the Food and Drug Administration, the Health Resources and Services Administration, the Indian Health Service, the National Institutes of Health, the Office of the Chief Technology Officer, the Office of the National Coordinator for Health Information Technology, and the Substance Abuse and Mental Health Services Administration. The Leadership Council provides input on priorities for the portfolio, including projects to fund. During the period from 2010 to 2019, the PCOR Trust Fund funded 53 projects, which translated to 76 agency awards, totaling approximately $131 million. 1 https://www.ssa.gov/OP_Home/ssact/title11/1181.htm 2 https://aspe.hhs.gov/collaborations-committees-advisory-groups/os-pcortf/about-os-pcortf 6

PREPUBLICATION COPY, Uncorrected Proofs FIGURE 1-1 Patient-Centered Outcomes Research Trust Fund: Three streams of work and funding. NOTE: AHRQ = Agency for Healthcare Research and Quality; DHHS = Department of Health and Human Services; PCOR = Patient-Centered Outcomes Research; PCORI = Patient-Centered Outcomes Research Institute. SOURCE: Workshop Presentation by ASPE, May 3, 2021. Figure 1-2 is a visual representation of ASPE’s current framework for the patient- centered outcomes research data infrastructure. The bottom row shows the main data sources feeding into the PCOR infrastructure. Data collected as part of clinical care include data collected for health care delivery and for billing purposes. Examples of primary data collected as part of research studies include data from clinical trials and national health surveys. Other examples of data sources include: Medicare or Medicaid claims data; quality or outcomes data collected by health care providers for the purposes of improving health care value; Food and Drug Administration data on the safety of medications and medical devices; and Centers for Disease Control and Prevention (CDC) data on births and deaths provided by state public health authorities. The framework describes the relationship between the data sources and the current key functionalities and focus areas (middle row) which support the research. The key functionalities are described in further detail in Box 1-1. Major building blocks are the services, standards, policies, and governance that enable the use of the data for research, described in further detail in Box 1-2. The top row shows the key data users and contributors of data. A more detailed overview of ASPE’s work and the projects funded to date will be included in the final report, at the conclusion of the committee’s review. 7

PREPUBLICATION COPY, Uncorrected Proofs FIGURE 1-2 ASPE’s Strategic Framework for the Patient-Centered Outcomes Research Data Infrastructure. SOURCE: Workshop Presentation by ASPE, May 3, 2021. BOX 1-1 Key Data Infrastructure Functionalities in the Existing Strategic Framework for Patient-Centered Outcomes Research Standardized Collection of Standardized Clinical Data Researchers will be able to use standardized clinical data based on common data element standards across research projects and networks, thereby facilitating linkage and aggregation of data across data sources. Collection of Participant-Provided Information Participants, including those in safety net organizations, will be able to participate more fully in clinical research by directly providing information (i.e., data points provided by the participant such as Patient-Reported Outcomes). Linking of Clinical and Other Data for Research Researchers will be able to follow patients across the care continuum over time, including those enrolled in clinical trials. Researchers will be able to capture the range of variables influencing health outcomes and link clinical and other types of data (e.g., other clinical data, 8

PREPUBLICATION COPY, Uncorrected Proofs claims data, participant-provided information, and environmental data) required for research regardless of where the participant goes. Use of Clinical Data for Research Researchers will be able to utilize and analyze routinely collected clinical data for implementation of clinical studies (observational and interventional), including data relevant to assessing safety, efficacy, and adherence, as well as genetic data and Patient-Reported Outcomes. Use of Enhanced Publicly Funded Data Systems for Research Researchers will be able to readily use, retrieve, link, and aggregate publicly funded data for research due to enhancements in publicly funded data systems. SOURCE: https://aspe.hhs.gov/collaborations-committees-advisory-groups/os-pcortf/about- os-pcortf/building-data-capacity-patient-centered-outcomes-research. BOX 1-2 Building Blocks of the Patient-Centered Outcomes Research Data Infrastructure Standards represent information and meaning to patient-centered data to ensure that health specific information can be accurately (and securely) exchanged and used. In most cases standards should be nationally accepted, widely approved, or broadly adopted either through market forces, community approval or regulatory requirements. These include such items as data standards for capturing, storing, representing, and exchanging data in a secure manner such that accurate information is conveyed to the recipient of the data. Policies are standards of behavior that participants can rely on consistently to build patient- centered data for research. Policies may include federal policies, as well as models for standardized state and local policies, that will lead to a trusted framework within the patient- centered outcomes research (PCOR) data infrastructure that ensures productivity, protects the patient and the patient’s data, ensures that evidence generations remains in the center of PCOR, and ensures the use of agreed upon standards and services. Services refer to resources that entities can employ on demand to capture, store, or exchange either PCOR data or evidence through a centrally hosted model provided remotely (such as through the internet) rather that provided locally or on-site. Services make it easy for the research data to interoperate among different systems without having to start from scratch for every connection. Governance structures refer to entities that are needed to develop and apply the rules and policies needed for building an interoperable and sustainable research network. Governance structures support the efficient use of the data infrastructure for research across individual and 9

PREPUBLICATION COPY, Uncorrected Proofs organizations’ boundaries of control and ownership. Governance structures are distinguished from “governance” which is what a governing body or governance structure does. SOURCE: https://aspe.hhs.gov/collaborations-committees-advisory-groups/os-pcortf/about- os-pcortf/building-data-capacity-patient-centered-outcomes-research. ISSUES FOR THE COMMITTEE ASPE asked the National Academies of Sciences, Engineering, and Medicine to appoint a consensus study committee and identify issues critical to building data capacity for PCOR and for generating new evidence to inform health care decisions. The input provided by the committee will contribute to ASPE’s strategic planning for their work related to the data infrastructure over the next decade. The study is part of a broader initiative by ASPE intended to update the strategic plan in light of the reauthorization of the PCOR Trust Fund and advances in health information technology and interoperability tools in recent years. The study is a collaboration of three units of the National Academies: the Committee on National Statistics, the Board on Health Care Services, and the Computer Science and Telecommunications Board. The consensus study committee has a diverse membership, its 15 members including experts with decades of experience, as well as emerging leaders, in the broad fields of: (1) patient-centered outcomes research; (2) research methods, statistics, and demography; (3) computer science and data infrastructure; and (4) patient engagement and patient perspectives. Appendix A contains the biographical sketches of the committee members. As part of its information gathering activities, the committee was asked to organize three workshops to collect input from stakeholders on aspects of the charge developed in consultation with ASPE. The workshops focused on key topics that the committee believed would particularly benefit from broad input from a variety of data users and other stakeholders. The conclusions from each workshop are summarized in interim reports. This first interim report summarizes the discussion and conclusions from the first workshop, which focused on looking ahead at data user needs over the next decade. The second workshop in the series centered on data standards, methods, and policies that could make the PCOR data infrastructure more useful. The third workshop discussed research and data collaborations. As an interim report focused on one in a series of information-gathering activities, the scope of this report is limited to a subset of the topics relevant to the committee’s charge and the conclusions reached by the committee are, at this stage, fairly high level. Some aspects of the topics discussed will be examined in further detail in subsequent workshops. For example, the first workshop focused on the additional data that stakeholders would like to have access to for PCOR. The second workshop will examine ways that the existing data could be better utilized to meet data needs, as well as the challenges associated with data sharing and addressing privacy concerns. After completing all of its information gathering activities, the committee will issue a final report, which will integrate and examine these topics in further detail. Box 1-3 shows the committee’s Statement of Task for the overall study; the committee will address this charge in its final report, integrating what was learned from the workshops, and from all other forms of input, including public meetings with HHS staff and background documentation available on the history and operations of the PCORTF. The final report will 10

PREPUBLICATION COPY, Uncorrected Proofs contain overall findings and conclusions from the study, on the basis of the committee’s further deliberations and integrated judgment on the input received and materials reviewed. BOX 1-3 Statement of Task for the Overall Study The National Academies will appoint an ad hoc committee to conduct a series of three one-day public workshops and develop conclusions to help guide the data capacity development for patient-centered research from 2021 through 2030. Each workshop will seek input from key stakeholders on topics relevant to the committee charge, and the specific focus of each workshop will be determined by the committee in consultation with the Assistant Secretary for Planning and Evaluation. As part of its activities, the committee will also  Consider the published review of the history and trajectory of the Office of the Secretary Patient-Centered Outcomes Research Trust Fund (OS-PCORTF) portfolio of investments and the OS-PCORTF roadmap;  Assess anticipated changes to health care priorities and priorities for health data and their impact on building data capacity into the foreseeable future, as identified by the Assistant Secretary for Planning and Evaluation;  Evaluate the feasibility and utility of developing a phased-in approach to building the interoperable data capacity for patient-centered outcomes research with existing databases in the Department of Health and Human Services, other federal departments, and the private sector in a phased approach, such as projects identified in the Cures Act Title III Section 4003 (Interoperability);  Consider other existing legislation, regulations, and the like, as deemed relevant; and  Receive input from individuals or groups that represent stakeholders including patients and their caregivers or families and their health care providers. The committee will issue interim reports after each public workshop with conclusions, and will produce a final written report with findings and conclusions to help guide a future course to continue building the data capacity for patient-centered research. All reports will follow institutional guidelines and be subject to the National Academies review procedures prior to release. Appendix B shows the agenda for the workshop, which was held on May 3, 2021. The committee’s goal for this event was to bring together researchers and representatives of patient organizations to understand the needs of these two important data user groups. Specifically, the goals of the workshop were to:  Provide a high-level overview of the types of data included in the data infrastructure for PCOR; 11

PREPUBLICATION COPY, Uncorrected Proofs  Identify key questions that stakeholders are most likely to want answered going forward, including general themes that cut across health conditions and circumstances;  Discuss the implications of the broadened statutory scope for PCOR;  Identify gaps in what stakeholders need and what the infrastructure allows. Consider both limitations in the existing data and improvements that could be made to new data collections;  Discuss what questions cannot be answered and who is not served by the current PCOR data infrastructure;  Discuss what matters HHS is best positioned to address and how the agency could maximize resources available for the PCOR data infrastructure (representing 4% of the PCOR Trust Fund), in the context of HHS’s public mission, authorities, programs, and data resources. Invited speakers in each of the sessions were asked to reflect on the general topics above. The specific questions for each session are described in Chapters 2 through 4. An obvious limitation of any activity of this type is that only a small number of stakeholders can be invited to speak. To compensate for this limitation to the extent possible, the committee invited representatives of patient groups with a broad reach, representing a variety of different interests and medical conditions. The researchers included were also individuals with broad research interests and an understanding of the PCOR infrastructure. In addition to sessions focused on the data needs of patient groups and researchers, the workshop also included a session on health disparities research and the data needed to explore this topic in more depth. The workshop also featured a case study on data challenges encountered as part of a study related to COVID-19. Appendix C contains biographical sketches for the presenters. A recording of the workshop and the presentation slides used by the speakers are available on the National Academies website at www.nationalacademies.org/PCORData. Information about the workshop was disseminated through National Academies mailing lists and on the project website. To collect additional stakeholder input, members of the public were invited to provide comments on topics related to the workshop (or any other topic related to the committee’s charge), using a public input form available on the National Academies website. OVERVIEW OF THE REPORT This report is organized around the three main sessions of the workshop. Chapter 2 discusses presentations on the data needs related to health disparities, Chapter 3 focuses on patient organization needs, and Chapter 4 describes the input received from researchers. The points conveyed by the workshop participants do not necessarily reflect the views of the committee; instead, in each chapter, a summary of the input received is followed by the committee’s conclusions. These conclusions 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. Because this is an interim report, the committee’s conclusions at this stage are big-picture conclusions, which will be integrated with additional input over the course of the study. 12

Next: 2 Health Disparity Data Needs »
Building Data Capacity for Patient-Centered Outcomes Research: Interim Report One - Looking Ahead at Data Needs Get This Book
×
MyNAP members save 10% online.
Login or Register to save!
Download Free PDF

The Office of the Assistant Secretary for Planning and Evaluation (ASPE), in partnership with other agencies and divisions of the United States 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.

ASPE asked the National Academies 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. This report, the first in a series of three interim reports, summarizes the discussion and committee conclusions from the first workshop, focused on looking ahead at data user needs over the next decade. The workshop included representatives of patient groups with a wide reach and researchers with broad research interests as well as an understanding of the PCOR infrastructure.

  1. ×

    Welcome to OpenBook!

    You're looking at OpenBook, NAP.edu's online reading room since 1999. Based on feedback from you, our users, we've made some improvements that make it easier than ever to read thousands of publications on our website.

    Do you want to take a quick tour of the OpenBook's features?

    No Thanks Take a Tour »
  2. ×

    Show this book's table of contents, where you can jump to any chapter by name.

    « Back Next »
  3. ×

    ...or use these buttons to go back to the previous chapter or skip to the next one.

    « Back Next »
  4. ×

    Jump up to the previous page or down to the next one. Also, you can type in a page number and press Enter to go directly to that page in the book.

    « Back Next »
  5. ×

    To search the entire text of this book, type in your search term here and press Enter.

    « Back Next »
  6. ×

    Share a link to this book page on your preferred social network or via email.

    « Back Next »
  7. ×

    View our suggested citation for this chapter.

    « Back Next »
  8. ×

    Ready to take your reading offline? Click here to buy this book in print or download it as a free PDF, if available.

    « Back Next »
Stay Connected!