Successful DCTs: Design Thinking for Optimized Clinical Trials
Bill Byrom
Signant Health

ecentralization of clinical trials is a new term but not a new concept. If we consider the objective of ensuring an empathetic patient experience, optimal trials from the patient perspective may not always be characterized by fewer site visits but should certainly be characterized by a simpler site experience. This benefits both patients and sites. This broader view of trial optimization is important as we design future studies through this wider lens.

Design Thinking

Design thinking is an approach used for practical and creative problem solving. In this approach, designers follow five phases:

  1. Empathize: Empathizing with the user to understand their wants, needs, and objectives.
  2. Define: Defining the problem, based on findings from the empathize phase.
  3. Ideate: Developing potential solutions, then narrowing these down to a few ideas to take forward.
  4. Prototype: Experimentation and turning ideas into tangible solutions.
  5. Test: Testing and refining the solutions based on insights and feedback gathered during testing.

While design thinking may be more common in software development, the principles hold true in study design, particularly in developing more patient-centered designs such as DCTs: Understanding the true needs of the patient and the problems to be solved (steps 1 and 2), developing designs and exploring them with patients through feasibility research (steps 3 and 4), and then continuing to refine and develop our thinking through experience of practical implementation (step 5).

We use the term DCT, decentralized clinical trial, a lot. But, behind the acronym, many of us have nuanced definitions, so while we talk about DCTs, we don’t always mean the same thing. Were DCTs born from the pioneering work of Orri and colleagues, who implemented the industry’s first completely siteless trial, the REMOTE study, much more than a decade ago? That would certainly be a key inflection point, combined with a renewed drive toward greater patient-focused drug development, steering a different perspective on how to implement clinical trial protocols. However, when you think about it, as an industry we started the journey, and have been in the business, of decentralizing trials for many years.

An alternative name I prefer is “optimized clinical trials.” As an industry, we seek to optimize the way clinical trials are run and how clinical evidence is collected, ensuring speed with accuracy, while delivering a more empathetic patient experience, and to enable all that at scale. Decentralizing aspects of a clinical trial by reducing the number of on-site visits needed through enabling certain procedures and assessments to be conducted away from the site is one important tactic to trial optimization. However, the concept goes beyond that.

Consider a cancer trial of a new treatment taken in addition to radiotherapy. Patients need to attend clinic most days, for a number of weeks, to receive radiotherapy treatment; on-site visits cannot be reduced or eliminated. However, thinking about trial optimization enables us to consider how to make those on-site visits the most efficient and least burdensome as possible, for both patient and site. In this case, patient-reported outcomes measured every week at specified site visits can instead be completed at home to reduce the time spent at the site.

Or consider a pediatric trial where caregiver and child need to attend an initial site visit to review study information and determine whether to consent to participate. The face-to-face caregiver and child discussion with the doctor is invaluable. But access to the full study information, with the ability to flag areas needing discussion ahead of the site visit, may focus discussion on the most important points, reduce the time needed in clinic, and eliminate unnecessary travel and disappointment for families who decide the study is not for them.

In both cases, we have not reduced the number of on-site visits. But we have simplified the on-site experience by considering elements that can be accomplished remotely to reduce on-site activity to only those elements that need to be done in-clinic. Is this a DCT, a hybrid study? If our definition is reducing on-site visits, then perhaps not. If, however, our broader definition is the thoughtful optimization of a clinical trial by decentralizing certain trial components to simplify participation, then yes, this is exactly the kind of study design thinking we are talking about and what, as an industry, we have been carefully doing for years, years before REMOTE, and years before the advent of the DCT acronym.

We’ve been collecting clinical outcome assessment data from patients at home for decades. Beginning with (and in some cases persisting with) paper, we’ve collected valuable disease and symptom data in the form of patient diaries to understand and measure intervention effects. We’ve subsequently optimized data quality and integrity by collecting these data electronically. We’ve migrated in-person clinician assessments, ClinROs like depression ratings, to telephone and video conduct, with early published validation studies providing evidence of measurement comparability between video and in-person dating back to the early 2000s. We’ve performed remote central ratings for important primary endpoints in CNS studies to drive data consistency and limit inter-rater variability affecting the ability to derive treatment-related effects. For longer still, we’ve collected informative data on activity and sleep patterns using technology worn by the patient during everyday life.

Technology is a powerful enabler, but trial optimization isn’t just about technology. Successful trials, including and especially successful DCTs, rely on comprehensive and great technology, combined with scientific expertise, to drive the highest quality clinical evidence generation and the most sensible use of enabling technologies (like eConsent, telemedicine, and direct-to-patient trial supply management). All trials, regardless of the setting in which they are conducted, rely on accurate and reliable measures of efficacy and safety, and optimization is specific to each study. It depends on aspects including the disease indication, the unique characteristics of the patient population (such as cognitive ability, dexterity, technology literacy, available time, and travel distance from the site), the concepts of interest and their associated measures to be studied, and the geographies involved. These should drive our thinking on how we select and thoughtfully apply our technologies to ensure accurate and reliable clinical endpoints, and to ease trial operation and participation for patients and sites.

Why is this important? We’re not going to apply technology for technology’s sake. We’re not going to apply decentralization for decentralization’s sake. Yet, we hear others setting targets for a percentage of trials to be DCTs in the coming years. The aspiration seems right, and maybe reflects the drive to demonstrate increasing patient centricity. But a blanket target like 40%, 50%, or 60% makes less sense. One hundred percent of trials should be optimized clinical trials: trials that thoughtfully apply technology with the aim of collecting informative, accurate, and reliable clinical evidence while simplifying participation for patients and sites. Depending on how you define it, this may or may not include “decentralization,” and it may not always mean fewer site visits for patients, but it will always mean thoughtful, science-led implementation that is sensitive to the study objectives and is empathetic to the patient population. This empathy is the first foundational principle in design thinking. It’s the first principle in clinical trial design thinking, in study optimization, and in successful DCTs and CTs.