Despite groundbreaking advances in basic and clinical science and technology, clinical trial methods have not kept up with the pace of change and are no longer fit for purpose — not for patients or for the life science industry. The reasons for this include regulatory uncertainty, economic factors, and cultural obstacles, not the least of which is an adherence to conservatism in a highly regulated industry.
Overcoming these barriers, especially breaking the culture of the status quo, is beyond the power of any one organization. What is required is an ecosystem-wide series of changes based on collaboration to adopt decentralized clinical trials and realize the benefits they bring.
One challenge facing the adoption of decentralized trials begins with language. We face a digital Tower of Babel, where the terms that one person uses with confidence can be easily interpreted differently by others. Without a common lexicon, it is easy for some people to be dismissive and conclude that new approaches like this are impractical or aren’t feasible for their work.
Is a decentralized trial one without a brick-and-mortar site and participants engage entirely online? Is it one where some “visits” may take place at home rather than in the clinic? Is it one in which some patients may participate using local labs and convenient health care sites?
For most people, the answer to all of the above is “yes.” The term decentralized trial encompasses a range of solutions that include digital tools for capturing consent data, the use of home health providers, direct-to-patient drug distribution, remote monitoring and diagnostics, and the use of local labs and imaging centers. In short, decentralized clinical trials are those that use novel technologies or processes to create options for participation outside of conventional clinical settings, improving access and convenience.
The work we are beginning as co-chairs of the Decentralized Trials and Research Alliance (DTRA) is a strong step toward that collaborative change. This global cross-industry coalition aims to increase access to clinical trials and research for all patient populations, especially those that have been traditionally ignored or overlooked.
At its launch in December 2020, leaders representing more than 50 organizations that included biopharmaceutical companies, regulators, patient organizations, investigator networks, key technology and service companies, and others shared approaches that foster needed change in clinical trial processes and, more importantly, shift organizational cultures to reinvent trials to benefit more patients. These founding organizations have since been joined by nearly 50 others that share the vision and mission of DTRA.
Covid-19 underscores the need for better representation
For too long, individuals in disadvantaged communities — which have been disproportionately affected by the Covid-19 pandemic — have been largely underrepresented in clinical trials. Decentralized research that leverages available technologies can help recruit a more broadly representative patient population by gender, ethnicity, geography, income, and more.
Remote monitoring and communications technologies make it possible for researchers to check in with participants as often as needed, rather than once a week or once a month, as is common in traditionally conducted clinical trials, and also to collect biometric information like heart rate or activity 24 hours a day, seven days a week. In addition to supporting the patient-centricity and engagement goals that most life sciences organizations have adopted, better access by participants across race, sex, age, geography, and other demographics offered by decentralized clinical trials provides the data needed to understand dosing, efficacy, and safety for the broadest possible range of patients.
Providing full optionality — participation when and where a patient has the ability — will be a challenge. Most protocols today are not written with such flexibility, and many trial technologies were not engineered to adjust for different locations based upon participant preference. Realizing this potential will also require thoughtful approaches that address the “digital divide,” not only enabling participants to use technology they may own, but also ensuring tools can be provisioned and provided for those who may not otherwise have access to necessary technologies.
Collaboration to drive change and adoption
While the benefits of decentralized trials are clear, the path to adopting them at scale has so far been challenging. Collaboration can foster understanding on what the research community can accomplish and offer confidence to stakeholders in the path forward that addresses concerns from feasibility to risk.
Stakeholders include sponsors and their ability to generate valid and reliable evidence; regulators, who must trust the data for decision-making; investigators, who must be able to execute studies and oversee safety, and for whom solutions must be improvements rather than create complexity and additional burdens; trial participants, who should have greater access and more control over their experiences; and technology and service partners, who must be ready to introduce solutions that meet these challenges in a complex environment.
Collaboration brings the opportunity to address the priority barriers identified by the founding members of DTRA:
- Remove the existing “Tower of Babel” of approaches by defining specific models for decentralized research as well as key performance indicators
- Use the collective experience of diverse members to understand and share best practices that reduce risk and improve the execution of decentralized clinical trials
- Gather data and information from across the clinical trial community to provide educational resources for all stakeholders and advance a supportive culture and environment to embrace decentralized research
- Identify remaining barriers to the global conduct of decentralized trials and chart pathways to resolution
Although DTRA has emerged as the largest multi-stakeholder collaboration focused exclusively on advancing decentralized research, no such organization can succeed by working in isolation. DTRA has begun to develop a network of other consortia and collaborations in the global research community that may have incremental initiatives considering decentralized trials, creating the opportunity to drive cross-organization meta-collaboration, avoid redundancy, and provide the research community with a clear roadmap for the future.
Now is the time for this community to seize the moment, enabled by the pandemic, to work together to harness the momentum toward decentralizing research and the technologies needed to improve trials for those they are intended to help — patients. If the health and social disparities that have become painfully evident show us anything, it’s that there are silver linings at our fingertips that we can best embrace together.
Amir Kalali is a physician scientist, clinical professor at UCSD, chairman of CNS Summit, and co-chair of DTRA. Craig Lipset is assistant professor of health informatics at Rutgers University and founder at Clinical Innovation Partners, and co-chair of DTRA.