United States: Advancing Adoption Of Decentralized Clinical Trials: Rationale, Current State, And Policy Recommendations

Foreword and Acknowledgements

Since 2022, the Initiative to Advance Decentralized Clinical Trials (the Initiative) has come together to identify issues and policy opportunities to advance decentralized clinical trials (DCTs). The Initiative is collaborating to improve advancements in health by identifying issues, to identify approaches that will address those issues, and to advocate for policy change that will support greater use of DCTs. The Initiative has engaged a number of health care industry stakeholders, namely patient and cancer advocacy groups, health care associations, pharmaceutical companies, and health technology companies. Through a series of round table meetings, participants have been meeting to discuss policy issues related to DCTs and to develop recommendations to advance widespread adoption of DCTs. The Initiative will continue to discuss issues related to advancing DCT with the goal of advancing policy recommendations, beginning with the ones contained in the attached white paper.

This white paper was discussed and developed through virtual roundtable discussions. While participation in the workgroup does not imply affiliation with or endorsement of the recommendations issued in this white paper, we would like to thank the following organizations for their participation:

• Amazon Web Services
• American Cancer Society Cancer Action Network
• Association of Clinical Research Organizations
• Epic
• Evidation Health
• Genentech
• EMD Group
• National Health Council
• National Partnership for Women & Families
• Thread Research

Executive Summary

Leveraging technology, electronic clinical data, and remote monitoring tools have enabled advancements in clinical trials to drive improvements in clinical and biomedical research. These advancements, primarily driven by developments in digital health technology (DHT), have the potential to improve health care delivery across diverse populations by changing how information is gathered from patients and health care providers.

DCTs are an approach to conducting clinical research in which technologies (including mobile devices) are used to remotely recruit, communicate with study participants, conduct virtual visits, and collect clinical data.¹ Widespread adoption of the electronic health record (EHR) and technological improvements driven by DHT companies have enabled the increased use of decentralized and hybrid clinical trials. DCTs are accomplished using technology and tools such as wearable digital health devices, patient apps, telehealth and smart phones to communicate with patients and collect data.

DCTs exist along a continuum: they include hybrid DCTs where a participant needs to visit the clinician (e.g., primary and specialty care physicians) at some times, and full DCTs where participants complete the entire clinical trial from their home. Some decentralized trials involve the patient's preferred physician administering the majority of standard medical care and data collection, while validating and submitting the details through technology to the physician's team. Most trials are not fully decentralized because of sponsor-mandated inperson interactions for safety and regulatory purposes. ² DCTs allow for the remote collection of data, including in the home or at the doctor's office, to become integrated into the clinical trial.

Currently, a patchwork of federal laws and regulations outlines requirements for DCT sponsors. In recent months, federal health agencies have also released proposals and frameworks on advancing DCTs. In general, traditional clinical trials and DCTs are subject to regulation by the Food and Drug Administration (FDA). Other DCT-related issues, including incentives for clinicians to recruit patients in clinical trials and DCTs and health information exchange issues, involve the Centers for Medicare & Medicaid Services (CMS) and Office of the National Coordinator for Health Information Technology (ONC). As a result of the COVID-19 pandemic, legislators and policymakers have acknowledged the need to further advance DCT adoption. The COVID-19 pandemic also demonstrated the value of DCTs for patients and supported clinicians' use for certain DHTs (i.e., flexibilities and expanded reimbursement for telehealth remote patient monitoring).

In the Consolidated Appropriations Act, 2023³ , Congress required FDA to issue or revise draft guidance that includes recommendations to clarify and advance the use of decentralized clinical studies. The guidance must include recommendations for several DCT issues such as remote data collection, use of digital health technologies in DCTs, and privacy and security issues. Congress also directed the Department of Health and Human Services (HHS) to evaluate agency practices to ensure that providers and patients have tools to identify and participate in decentralized and remote clinical trials. Congress has directed the HHS Secretary, in collaboration with FDA, ONC, and CMS, to evaluate agency practices across HHS and deliver a report to Congress with its findings and recommendations to accelerate DCTs and increase trial diversity by mid-2024. The policy recommendations contained in this report are intended to advise on HHS and agencies' upcoming report to Congress.

As explained further in this paper, DCTs offer several advantages over traditional, site-based clinical trials, which often place a significant burden on both patients and family caregivers. Because DCTs reduce the time and financial burden on patients, they can increase patient recruitment and maintain retention rates. DCTs allow access wider to prospective patients and increase the diversity of the patient population.

This paper outlines several policy recommendations to advance widespread adoption of DCTs. It provides an overview of the current environment on decentralized and hybrid clinical trials, including a discussion on benefits and challenges that DCTs may pose. It provides an overview of existing legislation and regulation that have been promulgated by the Administration and various agencies. It also provides background on programs and policies, not specific to DCTs or clinical trials, that can be leveraged to support DCT adoption.

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Footnotes

1. U.S. Department of Health and Human Services, Food and Drug Administration, Digital Health Technologies for Remote Data Acquisition in Clinical Investigations, Guidance for Industry, Investigators, and Other Stakeholders (December 2021) https://www.fda.gov/media/155022/download

2. Taiwo Adesoye, Matthew H.G. Katz, and Anaeze C. Offodile, "Meeting Trial Participants Where They Are: Decentralized Clinical Trials as a Patient-Centered Paradigm for Enhancing Accrual and Diversity in Surgical and Multidisciplinary Trials in Oncology," JCO Oncology Practice, 2023, https://doi.org/10.1200/op.22.00702.

3. Consolidated Appropriations Act, 2023, Pub. L. No. 117-328 (2022).

Originally Published November 2023

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