Clinical Trials Day 2024: Forging the Path to Better Trials for All

As science becomes more complex, we must simplify the collective experience of sponsors, sites, and patients.

Each year, Clinical Trials Day invites us to reflect on the scientific developments that are revolutionizing clinical research — and patients’ lives. Trailblazing programs in precision medicine are leading the charge and need to become everyday occurrences: from Genomics England’s 100,000 Genomes Project supporting patients affected by rare diseases and cancer to the I-SPY2 Trial reimagining trial designs in oncology.

Yet complex science often undermines operational efficiency. Over the past seven years, the average number of amendments per protocol increased by 60%, while the typical time to implement an amendment has almost tripled.¹ Applying even simple adjustments to a gene therapy trial, such as expanding the number of participants, can make or break a study by causing costs to escalate sharply. Over-hyped technologies (like DCT solutions) have failed to deliver on their potential, leading to lower operational efficiency rather than sought-after improvements.

To avoid a tug-of-war between scientific rigor and operational efficiency, we must prioritize the user and data journeys of sites, patients, and sponsors. Only by simplifying their collective experience will we be able to deliver the trials we need rather than what the technology allows.

Moving to a mindset of removing problems

The analogy I keep coming back to is that of solving a Rubik’s Cube, where each of the six sides represents a different research stakeholder: for example, patients, sites, data management, regulators, pharmacovigilance, and clinical research. It is not possible to see the other sides without changing your orientation and/or that of the Rubik’s Cube itself. So, if you try to introduce a change to benefit one user group (e.g., a new system for sites), you can’t anticipate the impact on at least three of the other users/sides. And just as no one can solve a Rubik’s Cube with one move, the industry won’t be able to address operational challenges in one go. This is all the more telling when you realize that many people are trying to solve the Rubik’s Cube simultaneously, leading to positive and negative impacts on all users.

Taking one critical group, sites have been voicing their concerns for years about the growing technology burden. Common pain points include navigating 15+ portals per study, organizing password changes every six to eight weeks, and accommodating each sponsor’s unique definitions, standards, and database setups. Not only do disconnected tools take site staff away from patient care and absorb their time in training, but they also undermine data quality by forcing repeated data entry. One leader likens the site experience to being stuck “in a really bad escape room”.

Thankfully, we are turning the corner on delivering better site support. An aspiration of fewer systems will reduce the site admin burden with positive knock-on effects on both patient recruitment and engagement. A better patient experience would then hopefully widen access to life-enhancing treatments, particularly in rare diseases. Reflecting on her experiences and needs as a rare disease patient, Helen Shaw, co-founder of the virtual site VCTC, observes: “I see how hard it is to take part in a clinical trial. But patients do want that opportunity to be offered something that they wouldn’t get in their standard care, whether additional MRIs or new medicines.”

Simplifying at a time when science is becoming more complex can feel counterintuitive. But when sites and sponsors shed the legacy systems holding them back, they can finally determine which processes they need to run the trials they want.

Orchestrating a better data flow

With cell and gene therapies accounting for a bigger share of the drug development pipeline, we can expect a changing research profile: more studies with relatively small patient populations and rolling regulatory approvals, leading (hopefully) to compressed timelines. Yet, paradoxically, even a study of 30-40 patients can still ingest and generate huge volumes of relevant data (e.g., DNA-related, molecular information) because each person is treated as an individual rather than a study average. This data is then used to develop highly personalized and effective treatments.

As we transition into a non-EDC-centric world, we will need more flexible data management so that sponsors can drive science forward while delivering complex studies efficiently. Rather than a one-size-fits-all approach, systems and technology must be able to support the majority of protocols with enough flexibility for niche trial requirements.

To solve the Rubik’s Cube, each research contributor should benefit from our data approach. This is only possible when all relevant study data can be captured, cleaned, and managed on the same platform. Centralized data management would alleviate site monitoring activities as trial data can be reviewed and acted on in real time. Patients can then participate effectively with more choice and control over their data.

Finally, we must prepare for new technologies and scientific advances to bring vast amounts of distributed study data. AI/ML will have an important role in transforming raw data so it is clean and usable: machine learning is already having an impact by taking the noise out of edit checks, for example. However, we must avoid the pitfall of our previous thinking on DCTs and assume that AI/ML will be a panacea to all our clinical data challenges.

Better trials for all

The time is right to interrogate and leave behind old habits, including excessive data collecting, cleaning, and querying. Regulators are already moving in the right direction by encouraging us to apply a risk-based approach to trial design and focus on the data and processes that safeguard trial quality.

If our goal is to bring all data together with the right processes and trained people, clinical teams will be able to focus on the science while data management can help systematically find patterns within (and across) studies. The benefits of a centralized approach go beyond operational efficiency and, over time, could change the economics of clinical trials. Having medical, investigators, and site coordinators on one connected platform will make it easier to meet ambitious patient recruitment timelines, and tackle emerging drug development challenges sooner.

Moving toward a common data framework will make it easier for sponsors, sites, and CROs to allocate their resources efficiently. Patients will be able to participate more flexibly and with more control over their data. These advances will put us on the path to better trials for all.

Listen to industry experts share their perspectives on the future of clinical trials.