Leveraging the health information technology infrastructure to advance federal research priorities

Abstract

Ensuring that federally funded health research keeps pace with the explosion of health data depends on better information technology (IT), access to high-quality electronic health data, and supportive policies. Because it prominently funds and conducts health research, the U.S. federal government needs health IT to rapidly evolve and has the ability to drive that evolution. The Office of the National Coordinator for Health Information Technology developed the National Health IT Priorities for Research: A Policy and Development Agenda (the Agenda) that identifies health IT priorities for research in consultation with relevant federal agencies. This article describes support for the Agenda from the Food and Drug Administration, the National Institutes of Health, and the Veterans Health Administration. Advancing the Agenda will benefit these agencies and support their missions as well as the entire ecosystem leveraging the health IT infrastructure or using data from health IT systems for research.

INTRODUCTION

Use of health information technology (IT) by U.S. healthcare providers and patient access to electronic health data have increased significantly in the past decade.^1–5 This has been facilitated by technological changes in information system capabilities and infrastructure,^6–9 as well as legislation, governmental programs, and policies.^5,10–14 In parallel, new health research information systems and capabilities are creating accelerated potential for research, discovery, and clinical translation. Large-scale efforts across the research enterprise are standardizing and increasing access to research data.¹⁵ New approaches, such as machine learning and advanced analytic techniques, have recently demonstrated possibilities for new discovery.^16–18 In addition, novel security approaches,¹⁹ new models of consent,²⁰ and open science initiatives²¹ are facilitating appropriate data sharing and enabling discovery. These efforts highlight the value of a robust research data infrastructure, while also revealing challenges. U.S. federal scientific policy has an essential role in addressing barriers and accelerating data-driven discoveries.

ADVANCING DISCOVERY THROUGH HEALTH IT POLICY AND DEVELOPMENT

The Office of the National Coordinator for Health Information Technology (ONC) is responsible for regulating the certification of health IT, including electronic health records (EHRs); promulgating health IT data standards; and coordinating nationwide efforts to implement and use health IT for clinical care and research in the United States.²² ONC has established a strategic goal to foster research, scientific knowledge, and innovation. Related projects, including data standard development,^23,24 interoperability functionality development,²⁵ and future-oriented policy development,²⁶ have been individually successful and have been conducted in partnership with the National Institutes of Health (NIH), Food and Drug Administration (FDA), Agency for Healthcare Research and Quality (AHRQ), and others, but the broader mission requires increased strategic coordination among all federal science agencies and initiatives.

Accordingly, ONC led the development of the National Health IT Priorities for Research: A Policy and Development Agenda (the Agenda),^27,28 which identifies priorities for policy and technological development in the United States, which are listed in Table 1. The Agenda has 2 overarching goals: (1) leverage high-quality electronic health data for research and (2) advance a health IT infrastructure to support research.

Table 1.

Health IT priorities for research policy and development

Improve data quality at the point of capture. Increase data harmonization to enable research uses. Improve access to interoperable electronic health data. Improve services for efficient data storage and discovery. Integrate emerging health and health-related data sources. Improve methods and tools to support data aggregation. Develop tools and functions to support research. Leverage health IT systems to increase education and participation. Accelerate integration of knowledge at the point of care.

IT: information technology.

The Agenda was developed in consultation with leading U.S. federal agencies that are using electronic health data and the health IT infrastructure to advance health research. Several federal agencies have a role to play in advancing the health IT infrastructure and use of electronic health data for research. For example, while it does not have a direct role in advancing scientific policy, the Centers for Medicare & Medicaid Services plays a role in the development and adoption of health IT. The Centers for Disease Control and Prevention (CDC) funds an extensive research portfolio and requires a robust IT infrastructure to advance public health science. FDA, NIH, and the Veterans Health Administration (VHA) are leading efforts to leverage electronic health data and infrastructure advancements for research across the full spectrum of health and disease. The Agenda represents the shared vision of these leading U.S. federal health research funding agencies for future federal science policies, as well as for technological development of health IT systems that support biomedical research and translation.

AGENCY INITIATIVES

The Agenda priorities are critical to achieve the missions of these federal agencies. In fact, several U.S. federal agencies already sponsor existing initiatives, described subsequently, which highlight how the Agenda priorities underpin meaningful improvement of research.

Food and Drug Administration

FDA has broad responsibilities to protect the public health.^29,30 FDA’s regulatory responsibilities are supported by a significant IT infrastructure, which receives and manages large quantities of data from application submissions, surveillance, research, and other sources. To be successful, FDA requires access to timely, detailed data, which would be advanced by Agenda priorities 1, 3, and 4. In particular, previous FDA experience with clinical data and claims data—such as the development of the Sentinel System—has highlighted limitations that can affect current data sources, including limited detail and lagging data updates, further emphasizing the need to advance relevant priorities outlined in the Agenda.^31,32

A modern IT infrastructure capable of consuming, aggregating, and analyzing large and diverse datasets is needed to achieve FDA’s broad goals and highlighted by priorities 6 and 7 of the Agenda. In September 2019, FDA published its Technology Modernization Action Plan, which is focused on modernizing its technical infrastructure, enhancing its capabilities to develop relevant technology products, and collaborating with key stakeholders to achieve interoperability.³³ To be successful, FDA will require improved data storage and services, along with new tools for aggregation and research, which are highlighted by priorities 4, 6, and 7 in the Agenda.

As data collected in the health system are increasingly used to inform FDA’s regulatory decision making, interoperability and harmonization efforts outlined in the Agenda are needed for the efficient collection and use of high-quality data. The 21st Century Cures Act of 2016¹³ underscored the promise of real-world data, which can be derived from EHRs, mobile devices, claims and billings activities, product and disease registries, and other sources, and evidence to support FDA’s regulatory decision making.³⁴ In particular, FDA recently published a framework for the Real-World Evidence Program and related use cases to evaluate the use of new types of data and the subsequent analyses in regulatory decisions for drugs and biologics and understand practical application.³⁵ FDA also provided guidance for the use of real-world data in the evaluation of medical devices and has used real-world data as part of medical device regulatory decisions.^36,37 However, interoperability, standardization, and harmonization are needed for the efficient collection and use of high-quality real-world data for these purposes, consistent with the need to advance priorities 2, 3, and 4 of the Agenda.

The increasing complexity of data that inform the regulatory process has led the FDA to develop novel IT tools for those purposes. For example, precisionFDA is a next-generation DNA sequencing platform that allows researchers to compare their genomic sequencing data against reference datasets and analyze their data using online genomic information libraries.^38,39 This initiative has provided researchers with access to comparative data on genomic datasets and powerful analytic tools, which demonstrates the need for advanced aggregation functions and tools to support data analysis and research as noted under priorities 6 and 7 of the Agenda. In addition, other federal agencies such as the National Cancer Institute (NCI) and CDC are participating in precisionFDA, highlighting the fact that several agencies have similar research-related needs and creating an example of the type of cross-agency collaboration that is needed to advance Agenda priorities.

National Institutes of Health

NIH is the nation’s medical research agency. To achieve its mission “to enhance health, lengthen life, and reduce illness and disability,”⁴⁰ NIH-funded researchers need access to high-quality electronic health data and research data infrastructure and tools.⁴¹ Advancing the Agenda priorities would help meet these needs. NIH is already advancing some of these priorities through several initiatives. In particular, in 2018 NIH launched the Science and Technology Research Infrastructure for Discovery, Experimentation, and Sustainability Initiative, which provides commercial cloud storage and computer support for high-value datasets resulting from NIH-funded research, addressing Agenda priority 4, promoting efficient data storage and discovery.⁴² In 2018, NIH also published its Strategic Plan for Data Science (the Plan), which aims to connect NIH data systems, support storage and sharing of data, increase data management and analytic capabilities, enhance the relevant workforce, and implement good stewardship policies.⁴³ The Plan is aligned with Agenda priorities 3, 4, 6, and 7 in highlighting the need for data to be findable, accessible, interoperable, and reusable, which can be achieved, in part, through more consistent use of data standards.

Most recently, to improve interoperability of research data, NIH released a notice encouraging researchers to make use of the Health Level Seven International^® Fast Healthcare Interoperability Resources^® standard to accelerate the use of clinical data for research purposes and the exchange of research data.⁴⁴ Not only will this advance priority 3 in the Agenda, but also for NIH to reach goals outlined in the notice, it will require coordinated collaboration with ONC, researchers, and developers to continue to advance standards development as outlined under priority 2 of the Agenda.

The National Library of Medicine, the world’s largest biomedical library and a leading funder of informatics research, provides valuable data and information resources for researchers, healthcare professionals, and the public. The National Library of Medicine Strategic Plan 2017-2027 identifies 3 goals, which focus on providing the tools for data-driven research, enhanced dissemination and engagement pathways, and building the needed research workforce.^45,46 To reach these goals, it is critical to create an ecosystem that addresses Agenda priorities 7, 8, and 9. In addition, molecular and genomic databases provided by the National Center for Biotechnology Information support next-generation sequence alignment and clinical variation discovery and documentation, highlighting the need to address Agenda priority 5 regarding emerging health data.⁴⁷

The All of Us Research Program at NIH intends to “collect and study data” longitudinally “from one million or more people,” leading to precision medicine treatments and prevention strategies based on individual differences.⁴⁸All of Us is both generating primary data (eg, individual genetic sequences) and collecting data from healthcare providers. This initiative has required new policy and IT to address challenges in data acquisition, curation, and analysis, identified across Agenda priorities.^48–50All of Us will require ongoing advances in data aggregation and analysis, as noted under priorities 6 and 7 of the Agenda, as well as increasing access to interoperable health data, as outlined under priorities 3 and 4 of the Agenda.

NCI is also gaining new insights from large datasets and leveraging health IT for research. NCI’s efforts under the Precision Medicine Initiative⁵¹ and the Cancer Moonshot^52,53 have funded the collection of tumor genomic information, relevant clinical trials, and related cloud data sharing and computing capabilities. The Center for Biomedical Informatics and Information Technology supports the needed advanced data infrastructure and capabilities.^54,55 In addition, the Surveillance, Epidemiology, and End Results Program is creating linkages between state registries, EHRs, pharmacies, and Medicare data.⁵⁶ To be successful, these and other NCI programs must have access to a robust health data infrastructure that aggregates and harmonizes health data from diverse and novel sources for advanced analysis, such as environmental sensor data or wearable technology, which will be achieved via implementation of the Agenda.⁵⁷ Specifically, NCI’s programmatic goals require advancement of key informatics issues outlined under priorities 3, 4, 5, 6, and 7 of the Agenda for improved interoperability, data access, storage, aggregation, and analysis of both traditional and emerging health and health-related data.

Veterans Health Administration

VHA, which provides care for more than 9 million veterans at 170 medical centers around the nation, is also a major biomedical research funder and uses health information systems for care delivery and research.⁵⁸ VHA’s strategic research priorities require access to high-quality electronic health data and would be accelerated by implementation of the Agenda. The ability to make discoveries from these data depends on efficient storage and would benefit from advancing priority 4 in the Agenda. In addition, as veterans’ health care increasingly includes care outside of VHA facilities, interoperability with private sector health information systems is essential, making it critical to address priorities 2 and 3 in the Agenda.

The Million Veteran Program (MVP) is a massive cohort program, with more than 800 000 veteran volunteers to date.^59,60 MVP includes electronic health data, genomic information, and survey data, some of which is drawn from the VA Informatics and Computing Infrastructure, which also supports thousands of other VA research projects.⁶¹ MVP recently expanded to include veterans who do not receive care through VHA, underscoring the centrality of interoperability. The VA Informatics and Computing Infrastructure and MVP rely on interoperable health and health-related data and advanced storage and analytics, which will be well served by addressing Agenda priorities 2, 3, 4, 5, 6, and 7.

CONCLUSION

ONC, FDA, NIH, and VHA individually have missions, goals, and programs that require the advanced use of electronic health data for research and public benefit. Federal health research funding agencies face common barriers and would benefit from collective action on electronic health data policies and technological development. They also have significant roles in improving the health IT infrastructure through their funding priorities and related policies so that that infrastructure can be more effectively leveraged for research use of health data. While several separate efforts currently underway are addressing some Agenda priorities, these agencies support the Agenda and recognize the need for comprehensive and coordinated action. Other agencies such as Centers for Medicare & Medicaid Services and the CDC also have aligned authority or interests and are exploring supportive collaborations. Several agencies are already advancing cross-agency collaboration such as through NCI and CDC participation in precisionFDA, and relevant consultation regarding implementation of pertinent 21st Century Cures Act provisions. Successful implementation of the Agenda will include strategic coordinated action by federal health research funding agencies, yielding a variety of benefits for the agencies and beyond, including efficient use of financial and technical resources, shared policies across research initiatives, increased impact through common policies, and—most importantly—improved health through scientific discovery.

FUNDING

This work was partially funded through U.S. Department of Health and Human Services Contract Number HHSP233201600021I, Task Order Number HHSP23337008T, with RTI International.

AUTHOR CONTRIBUTIONS

TZ-C and PJW led the conception of the article, through ongoing collaboration with APA, PFB, SD, ARK, and RR. TZ-C and PJW led drafting of the article. All the authors revised the article critically and provided intellectual content; and approved the final version for submission. The order of authors listed in the manuscript has been approved by all authors.