Reducing patient burden in clinical trials – especially for rare diseases – is a key objective for drug developers. Decentralized clinical trials (DCTs) continue to remain relevant long after the Covid-19 pandemic made them a necessity. Increased regulatory acceptance, improved recruitment, lower patient burden and superior data collection capabilities are just some of the reasons that clinicians continue to conduct trials with decentralized elements .
Moreover, the ability to bring the trial to the patient is increasingly important as the pharma industry focuses on rare disease studies that necessarily involve a limited number of patients. Putting these patients at the center of any rare disease study through a mobile healthcare approach is essential for success, as failure to recruit or retain even one or two fewer individuals dramatically narrows the study’s patient pool, and with it chances of success. Through video checkups and remote monitoring, mobile healthcare can even expand to include patients around the world, which can further widen the patient pool in critical rare disease studies.
While wearable devices, web-based technologies, and mobile healthcare have been in use for over 20 years, the rapidity of adoption over the past decade is startling. The percentage of trials using decentralized or virtual components increased from 3.28% in 2016 to a peak of 6.07% in 2022, according to GlobalData.[i] This increase has been possible not only due to improvements in technology and the necessities of a global pandemic but changing attitudes within the pharma industry itself.
Although early hype around decentralized trials (DCTs) has subsided – 2022’s peak has not been matched in subsequent years – the accessibility and efficiency they can offer remains valuable, particularly in areas where traditional trials fall short. One of the clearest applications for DCTs is in research fields where accrual rates are highest and success rates lowest, like rare disease. Patient centricity, although a fundamental concept in modern clinical trials, is uniquely hard to integrate into these kinds of research, where logistical and recruitment barriers are immense. Adding decentralized components can circumvent these barriers, putting the patient back at the center of a study so clinicians can improve diversity and accessibility. In this article, we examine the link between DCTs and patient centricity in greater detail to better understand how decentralization can be used to improve the patient experience amid even the most challenging of trial conditions – like rare disease studies.
Why patient centricity?
Studies have shown that patient enrollment and retention remains a significant obstacle to the success of clinical trials. Globally, around 80% of trials fail to recruit patients within the recruitment timeframe.[ii] This results in adjusted schedules and delayed deadlines, ultimately preventing life-saving therapies from reaching the market more quickly.
Although coined in the 1990s, patient centricity has continued to grow in recent years as clinicians seek to rectify low patient accrual and retention rates. By putting the patient’s needs and preferences first, it is possible to design studies that are less invasive, less time-consuming, and more comprehensible. The ultimate goal is to empower the patient through informed decision making so that they become an active partner in the research. Rather than asking if the patient is right for the study, clinicians instead need to know if the study is right for the patient.
Patient centricity aims to build trust between patients and the study, encouraging them to ask questions, raise objections, and use their voice to improve their experience. Designing protocols around patients is one key way that clinicians can foster a sense of inclusion, as well as providing flexibility to ensure the patient’s daily life is not overly disrupted by participating in a trial. Additionally, building health literacy is essential: clinicians need to be providing patients with accessible materials that clearly explain their obligations to the trial without using overly complex medical information.
How decentralized trials support patient centricity
DCTs continue to grow in popularity because they can help further these aims. According to GlobalData, 52% of decentralized trials incorporated mobile healthcare, 22% web-based technology, and 18% wearable devices into their studies.[iii] The popularity of these three components is not only because they can improve data accuracy and reduce costs – they improve the overall patient experience and help them engage with the trial’s demands.
Mobile healthcare
By bringing the trial to the patient, mobile healthcare eliminates the logistical barriers that prevent many from participating in clinical trials. Whether a patient suffers from mobility issues that make travelling difficult or lives a prohibitive distance from the trial site, mobile healthcare fosters inclusion and reduces patient burden.
Mobile healthcare also has the added benefit of improving diversity by reaching populations that might previously have been underrepresented in clinical trials, further centering the patient experience. In rare disease trials, where failure to retain tiny numbers of eligible individuals can dramatically compromise a trial burdened by low accrual, taking a mobile-first approach may mean the difference between success and failure.
Web-based technology
Big data and digital software are additional tools in the patient centricity arsenal. The digitization of paper methods now includes electronic consent forms (eConsent), electronic patient reported outcomes (ePRO), and electronic clinical outcome assessments (eCOA).
Web-based technology doesn’t just improve data accuracy and trial efficiency – it boosts outcomes for patients as well. For example, new software can turn eConsent forms from incomprehensible medical jargon into engaging presentations that help the patient fully understand their responsibilities in the trial.
Patients frequently drop out of trials when they realize what they had expected to do does not align with the reality. Setting clear expectations from the start through eConsent improves patient retention and ensures the patient fully understands their role within the trial. Additionally, ePRO and eCOA not only provide clinicians with better, real-time data, but empower patients to feel more confident about the outcomes they are recording.
The use cases in challenging research areas are clear. In rare disease trials, for example, where participants may live far from specialized centers and often face cognitive or developmental challenges, digital tools like eConsent can significantly improve understanding and engagement. Building trust in the trial in this way is yet another advantage afforded by decentralisation – one which puts the patient first and cuts down on complexity in tricky trial conditions.
Wearables
Wearable health technology such as step counters, heart rate monitors, and other remote monitoring devices are becoming increasingly common in clinical trials. Collecting real-time data in this way means clinicians can develop holistic understandings of how health conditions progress. And they have the added benefit of improving the patient experience during a trial.
Rather than regularly attending a lab to monitor their vital signs, patients can go about their daily lives and have that data collected remotely. In rare disease studies, where even traveling to a trial site can be prohibitive, wearables enable researchers to gather high-quality longitudinal data without burdening the patient. This broadens otherwise-limited patient pools by enhancing participation rates, and once again facilitates increased diversity in trials: through healthtech, clinicians can study patients in remote areas or even completely different countries.
Overcoming challenges in DCTs
While decentralized elements can improve the accessibility of clinical trials, they can be a hindrance if not deployed correctly. Digital literacy rates vary, and some patients might struggle to use digital tools they aren’t familiar with, resulting in them feeling isolated and confused.
For patient centricity to be fully achieved through decentralization, it is essential that digital elements are easy to use and intuitive for all patients. Providing in-person consultation and guidance continues to be important to reassure patients and build that all-important trust.
But, with small, geographically scattered patient populations and high trial burdens, many of the most complex research areas and intractable diseases will struggle with recruitment, retention, and feasibility. Decentralized models help address these challenges by allowing patients to participate from home, use digital tools, and avoid excessive travel.
When executed successfully, a patient-centric clinical trial accommodates individual preferences and helps them engage with the trial’s work, ultimately improving outcomes for all stakeholders. The fact that trials continue to incorporate more decentralized and virtual elements demonstrates that the concept is an essential component to the success of a modern trial. A study is nothing without its patients – clinicians must recognize this and find ways to improve accessibility and diversity to improve healthcare for the better.
[i] GlobalData, The State of the Biopharmaceutical Industry 2024 (Mid-Year Update)
[ii] https://pmc.ncbi.nlm.nih.gov/articles/PMC7342339/
[iii] GlobalData, The State of the Biopharmaceutical Industry 2025
