Hurdles and Harmonization: Data Collection in a Digital Health World
Aligning best practices in eCOA and ePRO within today’s evolving technology ecosystem.
During Cambridge Healthtech Institute’s 6th annual Summit for Clinical Ops Executives (SCOPE) Europe, which was held in Barcelona, Spain, Oct. 17-18, Andy Studna, editor of Applied Clinical Trials, was on-site to moderate a panel on the interplay of electronic clinical outcome assessment (eCOA), electronic patient-reported outcome (ePRO), and wider digital health technology (DHT) approaches in drug development.
The participants exchanged insights on a variety of topics, including terminology, regulatory obstacles, and what the future for this space could look like.
The following panel discussion has been edited for length and clarity. A video of the roundtable in its entirety can be found here.
Andy Studna, Editor, Applied Clinical Trials: It seems to me, through our contributed content at ACT, that the industry is viewing ePROs/eCOAs and DHTs as two completely separate entities, even though they are trying to achieve the same thing. Why is industry viewing these spaces so differently?
Bart Roofthooft, Head of Digital Health Solutions, EBIS/DHCoE, Janssen R&D: I think the ePRO world is already well established. So many of our preferred suppliers at J&J are able to provide ePRO capabilities with the right systems and right technologies. And ePRO is just one part of the broader eCOA system. So you have the ePROs, you have the ClinROs (clinician-reported outcomes), and ObsROs (observer-reported outcomes). But you also have the other side of digital health technologies, which is still a business that is growing and that we still need to understand a bit more. We need to understand the regulations that are coming out and we need to also see how they are impacting digital health and the end-to-end landscape. I love to talk about end-to-end because it speaks about the different technologies and different suppliers we are implementing. But it also looks at the impact that these digital health technologies are bringing. When it comes to connected devices or wearables in that sense, there’s still a lot to discover, still a lot to implement, and to assess. Also, how that helps to define the site and then the participant journey.
Scottie Kern, Executive Director, eCOA Consortium, Critical Path Institute: I agree with those that don’t think they’re the same, because they’re not. Digital health technologies have the capacity to administer and present an electronic clinical outcome assessment. It’s an assessment of a clinical outcome. eCOAs are not DHTs, but there is a crossover. And I think this terminology issue is fundamental to a lot of the challenges around adoption. They need to be considered differently and [while they have] similar skill sets to deal with implementation, they are not exactly the same thing.
Bryan McDowell, Vice President, eCOA Clinical Science & Consulting, eCOA Science, Clario: I think, potentially, they are the same thing, just to throw that into the mix. It’s just that one is better-defined than the other. I think we’re trying to separate out a couple of components where we know eCOA very well; we know the regulations are very strong around that, we know how to implement them, how to embed them, and we can move forward, whereas DHTs are still in their infancy.
We’re putting something comfortable to discuss back into one area and another piece into a different area that can cause confusion in discussions. We’ve seen the use of terminology being misused for a long time around areas such as defining decentralized clinical trials. So we’ve just got to be careful how we partition them out. [For example,] can we call eCOA a mature DHT?
Estrella Garcia Alvarez, PhD, Director, Global Clinical Operations, R&D, Almirall SA: I think that terminology is key. We need to agree on what each of the different elements that we have on our tables mean. The diversity is huge; ePRO, eCOAs, DHTs, etc. But we need to unify because otherwise we are losing time, in order to have everything fully integrated. I think we need to establish an agreement, because each company is talking about different things depending on their tool or the area where they are working.
At the end of the day, we need to be aligned together and fully agree on a common terminology to move on ahead to the future.
Studna: How do we get aligned on the terminology in this space? And what barriers are preventing us from doing so?
Kern: It’s a really important point. Based on that previous discussion, we don’t have alignment on the table here. I think there’s a couple of different factors to it. There’s definitely efforts to harmonize in the context of eCOA. Something that the eCOA Consortium has been involved with is developing a common lexicon. It took a lot of time and a lot of effort—contributions from sponsors and the PRO Consortium as well. We don’t really know if it’s being used.
The second component is that from a marketing perspective, for some technology providers, they want to create novel terms to describe what their technology does. It’s not helpful, because we end up thinking some things are different when they’re not.
I think the third most compelling issue is around the divergence in definition amongst the regulators, because ultimately, that’s what really matters. Where we have the issue of clinical outcome assessments is it being very clearly defined by FDA and biomarkers being very clearly defined by FDA, but those definitions don’t map exactly to what [the European Medicines Agency] has. And [the Innovative Medicines Initiative’s] Mobilize-D project is a great example of that. Because when they’re developing assessments and digital measures to assess mobility, they themselves define them as biomarkers. But when we look at them, they’re COAs. I feel very strongly that we need to start addressing this on a global scale.
Alvarez: I fully agree with you. For us it’s not clear—imagine for the investigators and for the patient. I love technology; we try to innovate in Almirall SA in clinical trials in every single project and study, but at the end of the day, we have the patients in the core of everything we do. And if we are adding new hurdles to the investigators and site staff and to the patients, we are not going to complete our objective, which is developing drugs. I think that if we only talk about technology and how to integrate, and we don’t carefully analyze the impact on a daily basis at the hospitals and to the patients, we are going to be lost. We need to bring the hospitals on board and agree with them on the technology—the need to integrate and how we can save them the time in selecting the patients, collecting the data from the patients, etc. This is my particular point of view as a pure operations person.
McDowell: I agree, it would be great to get harmonized terminology, but we could spend 15 years trying to do that. Languages evolve into different areas and have different meanings of words. I think we’ve got to understand or be on the right page, at the very least, to have these discussions rather than [introduce] a clear, strong, one definition in a single language. That’s going to be quite difficult to achieve globally in a huge industry that spans across the world and that touches upon all the investigators, patients, etc. It’s a big challenge that we could spend too long working on rather than getting on with putting the right solutions in place to bring new drugs to the market.
Roofthooft: Digital health is a broad term. It covers a lot of areas. You can speak about digital health capability, speak about digital health technology, or a digital system. It has different aspects depending on where the term of digital health is going to be used. Also, coming back to the operational impact to a patient or to a site, it’s really important to understand that journey from a participant perspective, [such as] implementing a tablet, a wearable, or a watch that may have additional hardware and software concerns. That also has a broader impact to the rollout of these technologies overall.
Studna: What should be top of mind for professionals in this space as we move forward into the new year?
Roofthooft: From a sponsor perspective, it’s looking at what we will be implementing for the future. So, looking at the capabilities of digital technologies, the ePROs, and the innovations that are coming our way to see how they best fit our goals around clinical achievements. How are they impacting treatments? How do we get the ideal set of preferred suppliers implemented to help drive that landscape?
McDowell: From my perspective, when we look at what has been going on for a number of years about inclusivity into trials, accessibility, and making sure everybody can get involved in trials, we’re going to come full circle. We need to get back to understanding that we’ve got to make sure that the integrity of the data is maintained. Again, we’ve got to absorb it, not just collect the data, so that we’re collecting the right data at the right time, frequency, and location. And to make sure it’s done all within the right parameters, so that the data is valid and can be used for regulatory decision-making. There’s lots of different ways we talked [in the past] about using paper—mixed modalities, etc. We’ve got to make sure that the data integrity is maintained [in digital as well]. We’ve got to do that work, not just talk about it.
Kern: I want to connect back to the regulatory discussion. FDA put out its patient-focused drug development guidance (see: bit.ly/3TkcViP). That’s a very important document, and we’re going to see that embedded into the new year as well as we adjust. FDA recently announced it’s putting together an advisory committee around digital health technology (see: bit.ly/3sRpXtb). That’s going to be an important opportunity to expand the dialogue with regulators involved. Also in Europe, the Accelerating Clinical Trials in the EU (ACT EU) initiative published its report recently at its workshop and began to put together an advisory committee to help drive things forward. So, there are opportunities to have a broader discussion about the challenges we face in 2024.
Alvarez: My dream is to make everything very simple and try to unify. For instance, I would like to have one unique electronic record in all the hospitals around the world, and one unique ePRO, eCOA, and EDC (electronic data capture) in order to save all of us the time and money that we are investing. We should be focusing more on personalized medicine, among other things, but that is our job and we need to continue to improve and maximize the data that we obtain.
