By Pamela Divack and Manasi Kapoor
Editor's note: This article is part of a Q&A series about clinical trial innovation during and after the Covid-19 pandemic. For more related insights, check out our Q&A articles with Optum's Digital Research Network and VirTrial.
The Covid-19 pandemic has brought together regulators, life science companies, health care providers, academic health systems, patient groups, and others to develop safe and effective diagnostics, treatments, and vaccines to address the immediate public health crisis. These efforts have created unprecedented collaboration, flexibility, and speed in starting new, streamlined clinical trials and innovating on the clinical trial process overall.
Notably, clinical trial innovation during the pandemic has led to increased prevalence of decentralized, virtual, and digital clinical trials. A decentralized trial, also known as a remote or virtual trial, is conducted through telemedicine, mobile/local health care providers (HCPs), and/or mobile technologies. For example, a participant may interact with a clinician via a telehealth visit or a nurse may travel to a participant's home to collect a blood sample. A digital trial uses technology to improve recruitment and retention, data collection, and analytics (e.g. using social media to recruit and engage trial participants, deploying machine learning to analyze trial data). Clinical trials that use digital health technologies, such as smartphone applications or sensors, can be considered both decentralized and digital trials.
To better understand the future of clinical trial innovation—and what's changed during the pandemic—we spoke with Leanne Madre from the Clinical Trials Transformation Initiative (CTTI), a public-private partnership (established in 2007 by FDA and Duke University) to develop and drive adoption of practices that will increase the quality and efficiency of clinical trials.
Question: How has Covid-19 changed the clinical trial landscape?
Leanne Madre: Covid-19 has accelerated the use of decentralized approaches, such as e-consent, telehealth visits, remote monitoring, and even shipping investigational medical products directly to participants. These approaches can increase opportunities for participation in clinical trials and decrease the burden for patients (e.g. by not having to travel for trial-associated visits). However, careful planning is required to understand the responsibilities and impact on all affected parties. For example, telehealth visits may be easier for trial participants but may affect the providers who are interacting with participants in new ways.
In addition, master protocols, such as the U.K. RECOVERY trial and the U.S. NIH ACTIV trials, are increasingly being used to quickly evaluate treatment options for Covid-19 patients. A master protocol is a single, overarching protocol designed to answer multiple questions and may involve one or more interventions in multiple diseases or a single disease, each targeting a particular biomarker-defined population or disease subtype.
Lastly, Covid-19 has also highlighted the need to address a long-standing challenge of clinical trials: the underrepresentation of certain populations, despite scientific evidence of treatment response differences. Covid-19 has disproportionally affected racial and ethnic minority communities, and it has highlighted the importance of ensuring these communities are not only represented in trials but also actively engaged as equal partners in trial design, execution, and oversight.
Q: What are biggest opportunities for innovating clinical trials moving forward?
Madre: The pandemic has accelerated our knowledge of how to better leverage clinical and non-clinical data to understand medical products. This gives us the opportunity to expedite the understanding and approval process of medical products in the future. For example, the COVID-19 Evidence Accelerator (launched by the Reagan-Udall Foundation for FDA, in collaboration with the Friends of Cancer Research) has used data aggregation, analytics, and rapid information sharing to inform and safely speed diagnostic and therapeutic R&D.
We also have an opportunity to build bridges and involve underrepresented populations in clinical trials, and then reflect on these experiences to do a better job diagnosing, preventing, and treating all diseases and conditions. Moving forward, the makeup of trial participants should reflect all people who could receive vaccines and therapies.
Q: What are the remaining barriers to clinical trial innovation?
Madre: We must not revert to our burdensome pre-pandemic practices. Instead, we should build upon the lessons we've learned about how to improve the efficiency and quality of clinical trials, and how we can generate evidence on medical products to elevate the health of all people. Many organizations are already capturing such lessons learned and laying a foundation for discussions to make sure we more routinely do those things that have worked.
Q: What is one thing you want other parts of the health care industry to know about virtual or decentralized clinical trials?
Madre: There is an opportunity for cross-industry stakeholders to leverage and combine data from various sources to better understand the benefits, risks, and appropriate use of therapies to improve care within the population and at the individual level.
For example, telehealth and in-home visits used in virtual trials highlight the interplay between clinical care and clinical research. Clinical trials of the future can further integrate this relationship to improve efficiency, avoid duplication of activities, and enable better long-term evidence generation. We must continue to find ways to quickly design meaningful clinical trials that address important scientific questions in an efficient, fit-for-purpose protocol.
Q: What type of cross-industry collaboration is needed to advance virtual/de-centralized trials further?
Madre: Even before the pandemic, there were a number of multi-stakeholder initiatives aimed at improving the R&D of medical products, including the CTTI and the Medical Device Innovation Consortium, in the United States and globally. In addition, government agencies, professional societies, trade associations, and other organizations are having discussions to advance the continued use of virtual aspects of trials.
Now, we need to bring in more new voices—from data science and tech companies, health care providers and systems, and payers—to think about sustainable system changes. With the momentum of this moment, we have a great opportunity to collaborate and build upon these advances to improve clinical trials, clinical care, and public health.