Wien, 01.10.2023 – Text: Catarina Carrão
With the impact of the COVID-19 pandemic, Decentralized clinical trials (DCTs) and digital health technologies (DHTs) gained a momentum in medical research, allowing for clinical study participants to remotely partake in trials using state-of-the-art DHTs.1-3
What are Decentralized Clinical Trials?
According to Dr. Leonard Sacks, associate director for clinical methodology in the office of medical policy at the U. S. Food and Drug Administration (FDA), in a decentralized clinical trial some or all of a clinical trial’s activities can occur at locations different than the traditional clinical trial site – these alternate locations can include the participant’s home, a local health care facility, or a nearby laboratory.3 This ability to perform dispersed clinical studies comes from the incorporation of digital health technologies, which can capture health care information directly from trial participants.
Which Digital Health Technologies can be used?
With the advancement of digital technologies, the use of portable instruments like glucose, blood pressure, or electrocardiogram monitors, with sensors that can be wearable, implantable, or ingestible, allows for the collection of health data in a remote dispersed form. Besides, the use of DHTs can also include synergistic mobile applications where clinical trial participants can rate their quality of Life (QoL), pain, depression, daily functioning; or even perform tests of functional performance such as cognition, coordination, and vision.3 As such, DHTs can collect data much more frequently than scheduled trial visits, sometimes even continuously, capturing information during the participants’ routine activities, providing an extra insight into the effectiveness and safety of the treatment in “Real Life.”
What are the benefits?
On a practical level, DCTs can reduce time and expenses in the long run by limiting or even eliminating the need for resources associated with traditional sites, although the costs are deferred to the needed DHT software/hardware. Additionally, clinical trial participants like to receive treatments at their homes or local clinic, rather than be obliged to travel to an unknown clinical trial site. With less of an inconvenience it can be easier to recruit people and keep them enrolled if they don’t have the burden of far distance travel. By reducing the barriers to participation, DCTs are more inclusive allowing for older individuals, or people with disabilities, or minority groups living in areas without clinical research facilities to participate. Furthermore, not only participants are benefited but also parents/caregivers, which are exempt from prescribed timeframes to visit the clinical site where treatment or follow-up is taking place. With these benefits, comes an improvement in trial efficiency, and facilitation of research on rare diseases, and/or diseases that affect populations with limited mobility.1-4
What are the challenges?
Clinical trial sponsors need to store electronic data securely, transport treatments safely (e.g., refrigerated if necessary), and allow participants to report adverse events (AEs).3 As such, participants and clinical research staff must understand how to use the technologies correctly and safely; and patients may also need to learn how to take the experimental treatment at home. On the other hand, not all people are comfortable with wearable sensors, or familiar with smart watches, and may opt out of participating, biasing the population to a younger a more tech savvy population.3 Also, sponsors should ensure that a DHT is fit-for-purpose (i.e., that the level of validation associated with the DHT is sufficient to support its use and interpretability in the clinical investigation).2
Additionally, collecting data from multiple remote sources may require strong data management skills, and data management platforms with cloud-based software solutions that support clinical trial management, reporting and data collection.
Hybrid Models
Although the number of DCTs is growing, the tendency is for hybrid models to become more common, where some trial activities take place at clinical trial sites and others at the convenience of a patient’s homes. For example, hybrid decentralized trials may be more appropriate in cases where the administration of a treatment or a complex medical assessment needs to be performed at a clinical trial site, and some follow-up assessments could be performed remotely through online patient-reported outcome measures, via telehealth or in-home visits, or by local healthcare providers.2
Conclusions
Both European Medicine Agency (EMA) and FDA have recently launched recommendations and guidance on DCTs elements and its hybrid form.2,5 Most importantly, all parties agree that the most fundamental rule is to protect the trial participants safety, in particular when patients are separated from their traditional care centers.
References
All references assessed 15th September 2023.
1. EMA. Recommendation paper on decentralized elements in clinical trials. Head of Medicines Agencies, European Medicines Agency, December 2022 https://health.ec.europa.eu/system/files/2023-03/mp_decentralised-elements_clinical-trials_rec_en.pdf (2022).
2. FDA. Decentralized Clinical Trials for Drugs, Biological Products, and Devices. Guidance for Industry, Investigators, and Other Stakeholders (draft guidance), May 2023 https://www.fda.gov/media/167696/download?attachment (2023).
3. FDA. The Evolving Role of Decentralized Clinical Trials and Digital Health Technologies. U.S. Food and Drug Adminitration https://www.fda.gov/drugs/news-events-human-drugs/evolving-role-decentralized-clinical-trials-and-digital-health-technologies (2023).
4. FDA. Decentralized Clinical Trials for Drugs, Biological Products, and Devices Draft Guidance Webinar: June 20, 2023 https://www.fda.gov/media/170858/download (2023).
5. EMA. EU Recommendations on Decentralised Elements in Clinical Trials. PCWP/HCPWP joint meeting, 28 June 2023, EMA Amsterdam https://www.ema.europa.eu/en/documents/presentation/presentation-eu-recommendations-decentralised-elements-clinical-trials-m-al-ccmo_en.pdf (2023).
