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Suggested Citation:"1 Introduction." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
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Page 1
Suggested Citation:"1 Introduction." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
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Page 2
Suggested Citation:"1 Introduction." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
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Page 3
Suggested Citation:"1 Introduction." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
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Page 4

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1 Introduction1 Clinical trials are a cornerstone of drug development, providing scientific evidence on the safety and efficacy of novel pharmaceutical compounds and informing clinical care. At the same time, traditional clinical trials are slow, expensive, and inefficient (Fogel, 2018). Additionally, given the requirement to travel to trial sites, clinical trials can place a time and financial burden on research participants, depending on the number of clinical visits required by the study protocol (Fogel, 2018). Narrow eligibility criteria for participation in clinical trials also creates an issue in that studies may not fully reflect the patient population for which a new therapeutic is intended to treat (i.e., patients in the real world who may receive a specific therapeutic intervention are generally more diverse than study participant cohorts when it comes to age, gender, race, ethnicity, disease severity, or comorbidities) (Blumenthal et al., 2017; Heneghan et al., 2017; Hill et al., 2008). As a result, the link between clinical research and clinical practice is also frequently misunderstood by patients and providers. There is growing recognition by stakeholders from across the clinical trials enterprise that transformational change in the way traditional clinical trials are conducted is needed to address these challenges and meet the needs of patients. An emerging trend in the clinical trial landscape has been the incorporation of digital health technologies—such as mobile devices, mobile apps, remote monitoring devices, and online social engagement platforms—into study design (see Appendixes D and E for examples). Such virtual clinical trials can leverage digital health technologies for collecting information at each stage of the clinical trial, improving trial participant recruitment and retention, enabling online-based informed consent, measuring real-time clinical endpoints, and continuous tracking of adverse events. Digital health technologies may give trial participants a choice of participating from the convenience of the home rather than traveling to a trial site, which can increase participant engagement and retention (Sharma, 2015). This may be particularly important for engaging patients with mobility issues or those who live in rural areas, which can be far from the research centers at which studies are conducted. Additionally, data collected through digital 1 The planning committee’s role was limited to planning the workshop, and the Proceedings of a Workshop was prepared by the workshop rapporteurs as a factual summary of what occurred at the workshop. Statements, recommendations, and opinions expressed are those of individual presenters and participants, and have not been endorsed or verified by the National Academies of Sciences, Engineering, and Medicine, and they should not be construed as reflecting any group consensus. PREPUBLICATION COPY: UNCORRECTED PROOFS 1

2 VIRTUAL CLINICAL TRIALS: CHALLENGES AND OPPORTUNITIES health technologies may enable continuous real-time data collection of endpoints during the course of a trial participant’s daily life (Gold et al., 2018) rather than periodic data collection during site visits, which may only offer a snapshot of relevant health information. Researchers and providers can use information collected through digital health technologies to enhance monitoring and improve understanding of treatment effects and disease progression. Despite the benefits, virtual clinical trials also come with risks, as described by workshop participants: • Patient privacy concerns, such as the risk of sharing sensitive health information over the Internet, said Deven McGraw from Ciitizen Corporation; • Operational challenges, such as the lack of community and provider engagement, said Craig Lipset from Pfizer and Silas Buchanan from the Institute for eHealth Equity; • Technical barriers, such as digital health technology user interface, said Donna Cryer from the Global Liver Institute; and • Cultural barriers, such as concerns over data integrity and fear of technology failing, said Leonard Sacks from the U.S. Food and Drug Administration (FDA). It is important to note that virtual clinical trials are not a “one-size-fits-all” model and only a fraction of clinical trials are fully virtual. In the near term, digital health technologies may only be accepted in a few settings, such as disease areas in which telemedicine is already an accepted practice or for evaluating medical products with a known safety profile and endpoints that can be measured remotely noted Kimberly Hawkins from Sanofi Genzyme. However, in the longer term, virtual clinical trials have the potential to streamline the process of drug development and may offer new opportunities for a modern, more patient-centric clinical trial enterprise, noted Lipset and Ray Dorsey from the University of Rochester. To explore the current clinical trials infrastructure and highlight potential opportunities for supporting the practical implementation of virtual clinical trials, the National Academies of Sciences, Engineering, and Medicine’s Forum on Drug Discovery, Development, and Translation (forum) hosted a 2-day workshop on November 28 and 29, 2018, titled Virtual Clinical Trials: Challenges and Opportunities. Linda Brady of the National Institute of Mental Health, National Institutes of Health (NIH) and Clay Johnston of the Dell Medical School, The University of Texas at Austin, opened the workshop by reflecting on the barriers and inefficiencies that currently exist in the clinical trial enterprise and how digital health technologies could be leveraged to address some of these challenges. The following workshop sessions aimed to advance discussions and common knowledge among key stakeholders about opportunities for a modern, patient-centric clinical trials enterprise in light of digital health technologies that could enable virtual clinical trials for new medical product approval (see Box 1-1 for the workshop Statement of Task). This proceedings builds on a body of related forum work. Proceedings based on previous workshops hosted by the forum include Examining the Impact of Real-World Evidence on Medical Product Development (NASEM, 2019), Advancing the Science of Patient Input in Medical Product R&D: Towards a Research Agenda (NASEM, 2018a), and Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020 (IOM, 2012). PREPUBLICATION COPY: UNCORRECTED PROOFS

INTRODUCTION 3 BOX 1-1 Workshop Statement of Task Clinical trials are a cornerstone of medical product development—supporting the evaluation of efficacy and identification of safety issues of new drugs—and a necessary regulatory requirement for bringing novel therapies to market. This workshop will examine opportunities for a modern, patient-centric clinical trials enterprise in light of digital health tools that could allow virtual clinical trials (e.g., studies that deploy various digital health tools or virtual site visits) for new medical product approval. Participants will discuss the current state of the clinical trials enterprise; highlight opportunities for systemic improvements; and discuss mechanisms to facilitate participation in clinical trials, including enhanced collaboration among sponsors, researchers, regulators, patients, providers, and health systems. Subject-matter experts will be invited to participate in the workshop through presentations and discussions that will: • Highlight opportunities for systemic improvement to support virtual clinical trials, including o Potential implications of virtual trials for cost, speed, regulation, and knowledge generation and dissemination of clinical trials; and o Elements of an IT infrastructure, including integrating data from electronic health records, mobile health applications, remote monitoring, virtual visits, and other relevant technologies with the capability to enhance the interface between clinicians and clinical trial participants. • Explore potential opportunities to use digital health tools to engage with patients and potential research participants, facilitate recruitment of participants to join a clinical trial, and maintain participation of diverse populations in the trial: o Collaborative approaches and incentives involving sponsors, researchers, patient advocacy groups, patients living with the particular condition being studied, and health systems—including regulations, quality measures and outcomes, or reimbursement strategies—to support the implementation of virtual clinical trials; and o Opportunities and challenges to enhancing equity in access and participation through virtual clinical trials. DEFINING VIRTUAL CLINICAL TRIALS For this proceedings, different terms were used by workshop participants to refer to clinical trials in which all or part of the study incorporates digital health technologies and enables remote participation outside of the traditional brick-and-mortar clinical trial site. Johnston observed that although the term “virtual clinical trials” is included in the workshop title, an adequate umbrella term for the variety of clinical trials under discussion is not easy to define. He noted that terms such as “decentralized,” “remote,” or “site agnostic” may describe some types of trials that incorporate digital health technologies, but many study activities still require a centralized location—a comment that Dorsey made in his presentation (see Chapter 2). PREPUBLICATION COPY: UNCORRECTED PROOFS

4 VIRTUAL CLINICAL TRIALS: CHALLENGES AND OPPORTUNITIES Additional terms referred to by workshop participants include “direct-to-participant” (see Chapter 3), “location flexible” (see Chapter 2), and “mobile” (see Chapter 5) clinical trials. Each of these terms highlights different aspects of how digital health technologies may be incorporated into study design. Nomenclature was a topic of discussion during the final session of the workshop (see Chapter 6). While identifying one umbrella term to describe all of the trials discussed at the workshop would be difficult, as noted by Steven Cummings, director of the San Francisco Coordinating Center, individual workshop speakers and participants suggested a few terms, including “flexible,” “modern,” and “21st century” clinical trials. ORGANIZATION OF THE PROCEEDINGS OF A WORKSHOP This proceedings is intended to provide a faithful summary of the presentations and discussions that took place during the workshop. In accordance with the policies of the National Academies, the workshop did not attempt to establish any conclusions or recommendations and instead focused on the information presented, questions raised, and improvements recommended by individual workshop participants. Chapter 2 summarizes workshop presentations and discussions regarding the inefficiencies of the current clinical trial enterprise, the boundaries of what might be considered a virtual clinical trial for medical product development, the opportunities to expand access for patients, and regulatory questions regarding the remote collection of endpoints. Chapter 3 explores perspectives and experiences with digital health technologies in interventional and observational studies, as well as in clinical care—highlighting the impact on participant recruitment, engagement, and input on research and study design. Chapter 4 discusses how the use of digital health technologies may alleviate or exacerbate access and equity in the context of clinical trials. Chapter 5 reviews the current and future policy landscape governing clinical trials and their relevance for virtual trial methodologies. Chapter 6 presents reflections by the panel moderators on their key takeaways from the workshop and a summary of discussions on possible opportunities for future action. PREPUBLICATION COPY: UNCORRECTED PROOFS

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Successful drug development relies on accurate and efficient clinical trials to deliver the best and most effective pharmaceuticals and clinical care to patients. However, the current model for clinical trials is outdated, inefficient and costly. Clinical trials are limited by small sample sizes that do not reflect variations among patients in the real world, financial burdens on participants, and slow processes, and these factors contribute to the disconnect between clinical research and clinical practice.

On November 28-29, the National Academies of Sciences, Engineering, and Medicine convened a workshop to investigate the current clinical trials system and explore the potential benefits and challenges of implementing virtual clinical trials as an enhanced alternative for the future. This publication summarizes the presentations and discussions from the workshop.

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