Clinical Researcher—June 2022 (Volume 36, Issue 3)

FORM & FUNCTION

Scott Gray

 

Though the concept has existed for quite some time, the emergence of COVID-19 thrust decentralized trials (DCTs) into the mainstream. Practically overnight, quarantines, site closures, travel restrictions, and the health risks of the virus itself drove clinical trials across the globe to a screeching halt. Clinical research organizations (CROs) and pharmaceutical study sponsors were uncertain about how to move forward while keeping their patients safe.

As the pandemic spread, life science organizations pivoted to technology to continue data collection and engage with patients. In a global survey of companies involved in clinical trials, the vast majority of respondents agreed COVID-19 accelerated the adoption of DCTs within their organizations. Ready or not, this real-world test allowed the industry to continue its studies in the face of unprecedented disruption.

Decentralized Trials: Here to Stay

DCTs leverage wearable medical devices, telemedicine, home visits, and local healthcare providers and laboratories in place of visits to traditional study sites, such as large academic medical centers, to manage drug administration and data collection. These solutions come with many potential benefits, such as better data quality and a more convenient experience for the patient. In many cases, they assist in shortening the time it takes to get new drug treatments to market.

For example, wearable devices offer near real-time patient data compiled in real-world scenarios as patients go about their daily lives. Using the same instruments and processes to collect data among participants reduces inconsistencies and streamlines the researchers’ data analysis. These devices also eliminate the need for patients to travel to central trial site locations, making it easier to participate by reducing the costs and logistical barriers which derail enrollment and retention. Engaging patient populations who otherwise might be unable to participate due to financial, geographic, or other personal constraints could, in turn, have a positive impact on increasing patient diversity.

Recognizing the challenges presented by COVID-19 and the growing prevalence of DCTs, the U.S. Food and Drug Administration (FDA) issued guidelines for conducting trials during the pandemic to help protect patient safety while minimizing risks to trial data integrity. For the first time, these guidelines provided CROs and trial teams with a regulatory perspective and suggested protocols for implementing remote patient care. These recommendations supplied guidance for conducting patient visits by phone and video conference, dispensing investigational treatments for self-administration at home, managing lab and imaging needs for patients, and detailing how remote efforts might impact study protocols.

Given their advantages and the new thinking provided by organizations like the FDA, it is safe to assume the use of DCTs will only continue to grow. However, this trial method’s quick adoption also revealed many shortcomings. Though some challenges will be naturally mitigated as the DCT process matures, others have demonstrated why DCTs will never be a one-size-fits-all solution for patients and clinical trial teams.

Where Decentralized Trials Fall Short 

Historically, clinical research teams have stored patient information in secure data management systems. By increasing the number of data collection methods used in a given study, DCTs can run into compatibility and consistency issues, making it difficult to reconcile data and security issues and creating risks to patient privacy.

The same technology enabling DCTs to succeed can also contribute to their downfall. Wearables and monitoring devices, for example, require patients to have access to reliable internet connectivity and the ability and desire to learn to use them as they are intended. Virtual participation may not be an option for patients unfamiliar with technology or those living in rural or poverty-stricken areas. Thus, inclusion criteria requiring participants to use smartphones and apps could unintentionally hinder patient diversity. Likewise, factors related to reliance on self-reporting and long-term adherence to these collection methods prompt the question of data accuracy, which could raise alarms among review boards and regulatory bodies.

Further, trials for patients with rare diseases are often too complicated for a DCT approach. Self-reporting and home visits are not always feasible for these patients and their caregivers. Many rare disease patients prefer the reassurance of meeting with specialized staff to address their complex medical concerns. Decentralized trials are not designed to offer the high-touch support these patients and their caregivers often require.

Additionally, due to the nature of rare disease patient populations, researchers and sponsors must often enroll patients from multiple locations across the world. With each new country comes an additional set of regulatory requirements for collecting and sharing patient data and the potential of language barriers and cultural nuances impacting communication. In many cases, having patient support staff from the same regions and countries as patients is the most efficient way to ensure trials proceed as planned.

A Hybrid Approach to Trial Design

A study conducted in 2021 found insufficient evidence to confirm which trial methods are most effective in terms of recruitment, retention, and cost. Despite the potential advantages of DCTs, brick-and-mortar trial sites remain a critical component, as a decentralized approach cannot apply to every clinical trial. As travel restrictions and public health risks ease, and we embrace a return to normalcy, adding DCTs to our collective toolbox is a strategic move, as is learning to select the right tool for the job.

In many cases, combining the personal care provided by traditional clinical trials with patient-centric elements of DCTs will be the most effective solution for study design. A study published by the American College of Cardiology found that clinical trials for drug approval often include decentralized elements. DCTs tend to incorporate aspects of traditional trial design with decentralization of the patient/medical staff interactions.

For clinical trials focusing on rare diseases, a hybrid (onsite/offsite) approach could offer the convenience of home care and telemedicine to reduce the number of in-person site visits while administering complex treatments in the safety of a controlled clinical setting. This hybrid application of the protocol would make the overall travel commitment more manageable throughout multi-year studies, reducing the burdens placed on patients and their caregivers.

Conclusion

Moving forward, we must continue to assess the value provided by DCTs in ensuring patient experience and safety remain of the utmost priority. We should continue to build upon what has been learned since the onset of the pandemic. It is up to clinical trial stakeholders to determine the best approach for balancing the needs of patients and their caregivers against the goals of CROs and pharmaceutical study sponsors. Whether that means a decentralized, traditional, or hybrid course, prioritizing clinical trial patients’ health and best interests ultimately encourages higher enrollment, increased retention, and more robust data collection results.

Scott Gray is the co-founder and CEO of Clincierge, a provider of patient support services for clinical trials. Since 2015, Clincierge patient coordinators have managed logistics and reimbursements in more than 300 clinical trials worldwide. For more information, visit www.clincierge.com.