Driving Patient-Centered Trials with Outcome Measures
Sonia Bothorel, Managing Director of ICON’s Outcome Measures solution, highlights how outcome measures can strengthen regulatory alignment, reduce trial burden, and improve data quality from Phase I through regulatory approval, post-marketing studies and beyond, as regulators and patients push for more meaningful evidence in clinical research.
Why is there growing urgency around outcome measures in clinical research?
Over the past decade, regulators have made it clear that patient experience data isn’t simply a “nice to have,” but rather, a requirement. Sponsors are now expected to demonstrate not only that a treatment is effective, but that it also improves aspects of health that matter to patients. The FDA’s guidance series on patient-focused drug development, the rise of real-world evidence, and the adoption of digital endpoints all reflect this shift.
Outcome measures, which span COAs and DHTs together, are central to meeting these expectations. They help define what success looks like from a patient’s perspective and ensure that clinical trials generate the data that supports both regulatory review and real-world relevance.
How do COAs and DHTs work together in a trial?
COAs, which include PROs, ClinROs, ObsROs, and PerfOs, offer structured, subjective insights into how a patient feels, functions or survives. DHTs provide continuous, objective data through wearables, mobile apps, and remote monitoring tools. When used together, they give sponsors a more holistic picture of treatment impact.
For instance, a PRO might capture fatigue severity, while a wearable measures changes in activity levels. These are two views of the same patient experience. This combined approach enhances data quality, reduces patient burden, and allows for real-world monitoring between site visits. Most importantly, this approach ensures that we are putting the patient experience at the heart of trial design.
How do outcome measures improve trial efficiency and regulatory outcomes?
A well-designed outcome measures strategy can streamline the entire development process, from phase 1 through to regulatory approval. By selecting meaningful measures early, sponsors can:
- Avoid unnecessary protocol amendments
- Reduce recruitment and retention challenges
- Align more closely with regulatory expectations
- Generate more compelling evidence for approval and labeling
- Translate clinical impact into real-world benefit
Our clients have also seen downstream gains such as higher patient engagement, better data quality, and fewer delays tied to unclear endpoints or inadequate instruments. For these reasons, we believe that an outcome measures-first approach isn’t simply good science. It is also good strategy.
What is ICON’s approach to outcome measures?
ICON offers an end-to-end service that integrates scientific, operational, and technical expertise. We support sponsors in identifying, selecting, and implementing the right combination of COAs and DHTs, from early-phase design through regulatory submission. That includes:
- Strategic advisory on fit-for-purpose COAs
- Selection and licensing of validated instruments
- Data strategy, DHT, and digital endpoint consulting
- Integration of actively and passively collected data
- eCOA implementation and training
- Multilingual support and localization
Our work is grounded in decades of experience through Mapi Research Trust, the leading independent curator of COAs and the ePROVIDE platform, as well as the expertise of the patient-centred outcomes team within ICON, and enhanced by tools like Atlas, ICON’s proprietary platform for selecting evidence-backed digital measures.
What challenges do sponsors face when implementing outcome measures?
Too often, the choice of COAs or DHTs is made without fully assessing how they will be implemented in practice. This can lead to operational hurdles later in the trial, such as difficulties integrating the measure into site workflows, technology incompatibilities, or higher-than-expected patient burden.
Selecting the right outcome measures requires both a scientific perspective, for example - ensuring they are valid, reliable, and aligned with the concepts of interest - and also an operational perspective, whereby we evaluate whether they can be feasibly deployed at scale, across sites, languages, and patient populations. Without this dual lens, even a scientifically sound measure can create costly delays or data quality issues.
What challenges are you seeing post-selection of measures?
We often see challenges in four key areas. First, is in planning and coordination. Implementing outcome measures cannot be an afterthought. Effective implementation of an outcome measures strategy requires early cross-functional alignment across clinical, regulatory, digital, and site teams.
Next is training and compliance. Site staff must have clear, consistent protocols for administering COAs and explaining the use of DHTs, particularly to patients who may not be technologically adept. In addition, patients must have access to effective onboarding and ongoing support for any technical issues that may arise throughout the trial.
Technology integration can pose another challenge. Managing multiple data streams from eCOAs and digital sensors requires robust infrastructure, which we find that many sponsors are still in the process of building.
And lastly, a challenge we see sponsors facing is stakeholder buy-in. At many pharmaceutical organizations, COA and DHT teams work in silos and rarely collaborate. A successful outcome measures strategy, however, requires shared goals and a unified evidence plan across DHTs and COAs.
Sponsors need outcome measures that reflect patient priorities, are feasible to implement, and stand up to regulatory scrutiny. ICON understands and foresees these challenges, and we help sponsors mitigate these risks by offering integrated delivery and expert consulting throughout the trial lifecycle.
What role does ICON’s Atlas platform play?
Atlas is the industry’s most comprehensive platform for insights on AI-powered biomarkers, multi-modal technologies, wearable sensors and other DHTs. Leveraging the insights from Atlas, we support sponsors in selecting and validating digital measures, helping bridge the gap between DHT innovation and regulatory-grade evidence. Atlas contains more than 19,000 digital measures and biomarkers from 3,600+ connected devices, covering more than 1,300 medical conditions.
What is the impact on sponsor decision-making when using Atlas?
Through this dataset, sponsors can evaluate available technologies, understand how specific digital measures have been used in past trials, and reduce risk when incorporating new endpoints. Atlas helps ensure digital measures are not only scientifically sound but aligned with patient priorities and operational feasibility.
The Atlas team also offers additional customizability with Digital Consulting services, tailored to those who are looking for a white-glove approach to measures or device selection. Leveraging Atlas’ data, we can provide support in defining the measurement strategy across a single or multiple trials or phases, can provide a list of fit-for-purpose DHTs for selected measures, a deep dive of available evidence in support of a DHT, amongst other capabilities.
What’s your advice for sponsors looking to get started with COAs and DHTs?
I encourage them to think strategically from the start. This means:
- Beginning with the patient and identifying meaningful aspects of health through qualitative research
- Translating those priorities into measurable concepts of interest
- Choosing fit-for-purpose tools that combine both COAs and DHTs as appropriate
- Validating their measures early and aligning with regulators on endpoint strategy
- Investing in training, site readiness, and ongoing data monitoring
- Using partners who understand both the science and the operational realities
Patient-centered research requires intentionality. With the right planning and support, outcome measures can help bring better treatments to patients faster, and with greater confidence.
