In recent years, the field of clinical research has witnessed a steady shift towards decentralised clinical trials (DCTs). This transformation has been driven by advancements in digital health technologies, increasingly patient-centric approaches, and the urgent need for more efficient and adaptable trial methodologies. As the landscape of clinical trials evolves, DCTs have emerged as a promising alternative to traditional site-based trials, offering several potential benefits such as enhanced patient convenience, recruitment and retention, improved data quality, faster trial timelines, and lower costs.
Patient-centric trials align with the broader movement towards personalised medicine, focusing on tailoring medical interventions to individual patient needs and preferences. Companies like Medable have been leading the charge, pioneering the DCT space with innovative digital platforms and solutions. Their cutting-edge work enhances clinical trials by improving patient access and engagement, enabling remote monitoring, and ensuring high-quality data collection. Furthermore, organisations like the National Institute for Health and Care Research have given free access to helpful resources like guidance documents, practical examples and templates, which serve as an invaluable tool for researchers and sponsors looking to navigate the complexities of DCTs and ensure their successful execution.
Pandemic-driven shift to DCTs
The COVID-19 pandemic significantly accelerated the adoption of DCTs. During the pandemic, many clinical trials faced disruptions due to lockdowns, travel restrictions, and concerns about patient safety. In response, regulatory agencies and research organisations rapidly adopted decentralised approaches to ensure the continuity of essential clinical research activities. Regulatory bodies like the TGA (Therapeutic Goods Administration), FDA (Food and Drug Administration) and EMA (European Medicines Agency) have issued guidelines encouraging the adoption of decentralised elements to ensure continuity and resilience in clinical trials. This regulatory support has provided a conducive environment for the implementation of DCTs, reassuring sponsors and researchers about compliance and quality standards.
Pros and cons of DCTs
A major appeal of decentralised trials is that they can offer significant cost savings. Traditional clinical trials are often associated with high costs due to site management, travel reimbursements for participants, and logistical complexities. By decentralising trial activities, sponsors can reduce expenses, making clinical research more financially sustainable and accessible. The economic efficiencies of DCTs align with broader industry trends towards value-based healthcare, focusing on better outcomes at lower costs. Furthermore, apart from the cost optimisation, the use of digital health technologies in DCTs draws upon telemedicine and mobile health, which have demonstrated the efficacy of remote healthcare delivery in various contexts.
Despite the numerous advantages, the implementation of DCTs is not without challenges. Ensuring data integrity and maintaining regulatory compliance in a decentralised environment requires robust digital infrastructure and rigorous validation of remote monitoring technologies. Additionally, addressing issues related to patient privacy, data security, and the digital divide is critical to the successful execution of DCTs. Researchers must also consider the potential variability in data quality due to differences in patients' ability to use digital tools and adhere to remote protocols.
Remote N of 1 trials: Decentralisation in action
The work of Jane Nikles, Director at N-of-1 Hub, provides a compelling example of how the principles of decentralised trials can be applied to innovative N of 1 trial designs. N of 1 trials involve repeated cross-over comparisons of treatments within a single patient, making them inherently personalised and suitable for remote execution using digital health tools. Nikles’ pioneering research using remote N of 1 trials, which began around two decades ago, offered valuable insights into the use of decentralised methods to deliver individualised trial methodologies. In her research, Nikles and her team demonstrated the feasibility of remote N of 1 trials, for example to test treatments for pain in people with osteoarthritis, and stimulants for children with Attention Deficit Hyperactivity Disorder (ADHD). She initially used telephone and mail-based methods to provide education and monitoring and collect data, and later incorporated digital technologies like email, mobile Apps, telehealth and remote monitoring devices to enhance patient engagement and data collection. Because all of Nikles’ N of 1 trial research in the last two decades has been successfully conducted using decentralised methods, her team seamlessly transitioned to using remote N of 1 trials during Covid-19 (during the times when clinical research was able to be conducted). This is a notable example that demonstrates the value of decentralised innovative trials.
While remote N of 1 trials differ from the larger, group-based randomised controlled trials (RCTs) typically associated with DCTs, they share several key principles. Both approaches emphasise patient convenience, real-time data collection, and the use of digital technologies to enhance trial efficiency. By integrating these elements, researchers can design trials that are more responsive to patient needs and adaptable to diverse clinical settings. The work of researchers like Nikles underscores the value of decentralised methodologies and provides a framework for future innovations in clinical trial design. As the field continues to evolve, embracing the principles of decentralisation will be crucial to addressing the challenges and opportunities of modern clinical research.
N-of-1 Hub is a consulting company that specialises in designing, conducting and analysing personalised clinical studies using single-case designs. N-of-1 Hub provides consulting and collaborative services to companies, clinicians, researchers, and healthcare consumers who wish to conduct personalised clinical studies using single-case designs. N-of-1 Hub also offers data management and analysis services and customised workshops and courses. Additionally, our training programs are tailored to equip researchers and clinicians with the skills needed to excel in personalised clinical studies.
For more information on how N of 1 trials can revolutionise your clinical research and improve patient outcomes, we invite you to contact us for a free consultation. Don’t forget to follow us on LinkedIn for the latest updates and insights in the field of personalised clinical studies
References
Nikles, C. J., Clavarino, A. M., & Del Mar, C. B. (2005). Using n-of-1 trials as a clinical tool to improve prescribing. British Journal of General Practice, 55(512), 175-180.
Nikles, C. J., Mitchell, G. K., Del Mar, C. B., Clavarino, A., & McNairn, N. (2006). An n-of-1 trial service in clinical practice: Testing the effectiveness of stimulants for attention-deficit/hyperactivity disorder. Pediatrics, 117(6), 2040-2046.
Nikles, C. J., Yelland, M., Glasziou, P. P., & Del Mar, C. (2005). Do individualized medication effectiveness tests (n-of-1 trials) change clinical decisions about which drugs to use for osteoarthritis and chronic pain? American Journal of Therapeutics, 12(1), 92-97.