Trials of the century

Over the past two decades, clinical trials have undergone significant transformation.

Historically, trials followed a paternalistic and analogue model with sponsors and investigators dictating the process to participating patients, often with little consideration for how they felt or how they wanted to engage with the activities they were asked to complete.

Trial activities took place on-site and relied primarily on physiological endpoints – imaging, biomarkers, lab tests – while disregarding the lived experience of the patient.

Communication to participating patients was generally limited, often leaving them confused about what was expected of them. To put it simply: trials were largely designed for the convenience and curiosity of principal investigators, rather than the comfort and ease of mind of patients.

In the early 2000s, as it became increasingly clear that physiological improvements in patients did not necessarily translate to improvements in the way they felt about their condition or treatment, clinical trials started to shift towards a more patient-focused process.

For instance, self-reported measures or outcomes, enabled by tools such as electronic Clinical Outcome Assessments (eCOA) technology, were being used more widely across the industry.

These technologies offered sponsors and investigators a more comprehensive understanding of how a treatment was impacting individual patients and provided detailed insight into how patients were feeling about their condition and treatment.

Modern clinical trials are fundamentally different from their predecessors. The ‘ideal’ trial is as patient-centric as possible – designed around the patient’s preferences, lifestyle and needs. It goes far beyond decentralisation, to focus on integrating patient choice and flexibility into the entire trial process from the beginning.

After all, what patients ultimately want is flexibility – specifically, the ability to engage with study tasks in their own, preferred environment and at their own pace. Technology has evolved to the point that this can all be made possible.

We no longer talk about ‘decentralised clinical trials’ (DCTs) as a standalone model. Instead, decentralisation is a method that can be embedded within ‘traditional’ trial designs and a capability within a wider technology framework. This framework is designed specifically so that the technology supports the patient-centric focus of the trials.

For example, patients can often complete all their study tasks through accompanying apps, which act as a central hub for accessing eCOA questionnaires, reviewing consent documents, interacting with wearable sensors and completing study visits.

Whether it is smartphone-based or web-based, the platforms are designed to offer the most pertinent information to patients at the right time, ensuring that they receive consistent, tailored access to what they need, without the burden of switching between platforms or logging into different sites unnecessarily.

This technology allows for continued scalability, which is key to speeding up the modernisation of trials.

One of the biggest technological shifts in recent years has been the adoption of Bring Your Own Device (BYOD) strategies in eCOA studies.

While there had initially been significant concerns within the industry around data comparability and operational logistics, BYOD has since proven to be both reliable and effective. Today, it is a significant element of many modern eCOA trials and part of what we call the ‘silent revolution’ in clinical trials – a move towards greater accessibility and patient control.

eCOA technology has been instrumental in this shift towards patient flexibility and comfort in the modern trial process. By enabling reliable – and valuable – data to be collected directly from patients, eCOA is providing a means for wider technology adoption. Importantly, this technology also enables better insights into what is working, what is not and how to adjust in real time.

At an earlier point on the adoption curve, we are starting to see sensor technology being used to gather real-time, deeper data from patients without the need for them to come in for time-consuming site visits.

Let us take the example of a remote six-minute walk test: patients wear a sensor at home and follow the instructions provided to them on their eCOA device. Data is then collected and fed back to the study team in near real time for analysis. We are seeing results comparable to the ‘gold standard’ methodology that is collected in a clinic.

This offers a more flexible way to monitor mobility without the need for a physical site visit. Similarly, eConsent platforms allow patients to remotely review and sign consent forms, removing the inconvenience of travelling to a site for the simple purpose of signing a form.

Without a doubt, there has been significant progress over the last decade to shift towards patient-focused trials. However, this has not been without its challenges.
For many sponsors – particularly those with less experience with the modern clinical trial – transitioning to this new process can be challenging.

Indeed, while eConsent platforms have gained a significant amount of interest from sponsors, the added complexity of managing the idiosyncratic requirements of the numerous IRBs that may be involved in a global trial has so far proven to be a challenge in terms of their adoption into trials.

The real hurdle, however, is not the technology itself, which is more accessible and user-friendly now than ever before. Instead, it is the internal change management required to prioritise patients and introduce technology processes into the organisation.

Change requires a shift in mindset, not just the integration of new tools. For sponsors to successfully engage with the modern trial, it is important that they understand the need to completely restructure how trials are designed – from talking with patients before protocol design even begins, to finding ways to foster ongoing communication with patients throughout a trial and beyond – to be more inclusive, more flexible and more tech-enabled.

Different therapy areas have adapted to this modernisation process in different ways. Rare and complex diseases, such as those relating to the Central Nervous System (CNS), have often led the way in patient-centricity due to the urgency and complexity of these conditions.

Technology has helped widen the participation pool, making it easier for patients across different geographies to join studies.

Oncology, too, has benefited from the decentralised process – for example, enabling very sick patients to reduce site visits by completing tasks from home.

Conversely, however, it is also an area where patients typically value face-to-face interactions with their care providers, serving as an important reminder to us that flexibility means offering choice, not simply eliminating the site altogether.

For conditions with event-driven symptoms such as migraines or chronic pain, the benefits of technology become even more profound.

Traditional site visits are limited in what data they can capture as they provide only a snapshot of the patient’s condition at a given time. But when data is collected at home, over several days, weeks or months, through sensors or regular eCOA assessments, a more complete picture can be provided.

This longitudinal view can lead to better diagnostics, improved treatment tracking and, ultimately, better patient outcomes.

As we look to five or ten years down the line, we anticipate that trials will become increasingly flexible and conversational.

AI will play a larger role, not as a buzzword, but as a functional tool to streamline and speed up repetitive study operationalisation tasks like edit checks, data cleaning or even real-time translation of patient-facing content.

Trials will become more natural, allowing for dynamic questionnaires that evolve based on patient input. The industry is expected to shift toward studies that are continuously adapting, providing real-time insights on study design and conduct.

Yet for this vision to become reality, regulatory frameworks must also adapt. Sponsors and regulators will need to collaborate to validate these new approaches, ensuring that flexibility does not clash with scientific integrity.

Change is never easy, especially in an industry that is so driven by control and compliance. But the opportunity is clear: faster, more efficient trials that generate higher-quality data, improve patient experience and get life-changing treatments to market faster.

As for the often-stated concerns for those patients who may be less tech-savvy, we have found that with proper training and support, these groups engage just as effectively as ones who are more accustomed to technology.

The key is to build systems that are intuitive, accessible and backed by support. When done correctly, compliance and engagement follow naturally.

Ultimately, modernising clinical trials is not just about integrating technology – it is about rethinking the entire patient journey.

It is about asking how trials can fit into a patient’s life, rather than asking patients to fit into a trial. With this approach, and with the right tools, we can build a future where trials are more inclusive, more efficient and, most importantly, more human.

Paul O’Donohoe is a Senior Director at Medidata Solutions