Leveraging Technology to Empower Patients and Decentralize Clinical Trials

Health Storylines is a patient-driven, digital platform for people to track and manage their health, but it is also a tool to enable drug and device developers to conduct decentralized clinical trials and gather real-world evidence. Alira Health expanded its digital health offerings with Health Storylines through its acquisition of Self Care Catalysts at the start of 2022. We spoke Gabriele Brambilla, CEO and co-founder of Alira Health, about how the technology provides patients with greater control over their own health, how it is using it to drive decentralized clinical trials and the integration of real-world evidence in the drug development process, and the potential to leverage its Health Storylines platform to answer research questions outside of a traditional clinical study.

Daniel Levine: Gabriel. Thanks for joining us.

Gabriele Brambilla: Thank you for inviting me, Danny pleasure.

Daniel Levine: We’re going to talk about decentralized clinical trials, Alira Health, and how Alira is using digital health technology to improve data gathering while giving patients greater control over their own healthcare. Alira describes itself as a global healthcare advisory and technology company. It’s done a series of acquisitions lately, including a crowdfunding platform, CRO, and a digital health company. Help us make sense of what Alira does, who are its customers. How does it make its money and what value does it provide?

Gabriele Brambilla: Yeah, so Alira Health focuses on delivering services to pharma, biotech, and medical device companies through the continuum of development of a drug or a device. So, if you look at our services that go from a very early phase of development, which is called CMC, chemistry manufacturing control, through regulatory clinical trial management, market access, real world evidence—we do it with a very particular approach. About two years ago, we decided to go out in the market and buy technology that would empower [and] enable our services to be more patient centric. And what does that mean? More patient centric means that we focus on the collection of data through a partnership with patients to support our services throughout the whole continuum—it could be a decentralized clinical trial, platform technology in a clinical trial. It could be a technology used in a patient support program in a commercial drug already in the market. The mission is to what is called humanized healthcare or democratized life sciences research. So, bringing patients as partners in the different phases of development of solutions in life sciences. So that’s a different angle that we have.

Daniel Levine: Many of our listeners will be familiar with the challenges of conducting a clinical trial, particularly in the rare disease space, because those often involve small and geographically diverse patient populations. We’ve seen growing adoption of decentralized trials. This has been a long term trend that’s gotten a boost from the pandemic beyond the ability to make it easier for people to participate. Are there unexpected benefits that have resulted from decentralized trials?

Gabriele Brambilla: First of all, let’s define decentralized trials, right? For me, when we talk about decentralization, it doesn’t matter if it’s full or hybrid and we can discuss about that later if you want, but it is every time that you don’t have to go to a site to enroll a patient or you don’t have to ask the patient every time to come back to the site to be able to monitor his conditions through a particular therapy or a diagnosis. So, what happens when you use decentralizing technologies that usually are defined as EPRO—electronic physician reported outcomes—or eConsent or E-consultation or tele-consultation? It’s the fact that you’re going to be able to reach a much more diverse population because you don’t have geographical limitations. So, the health equity component, the ability to enroll patients from different status, different geographies, and they don’t have to be close to be in an oncology trial to Mayo Clinic, MD Anderson, or Dana Farber. You can really actually recruit them from everywhere. That’s a big advantage, right? It’s a big advantage that allows a higher diversity in the clinical trial. And then there are the typical things like not having to go back to the hospital, not having to have a monitor to visit every time. There is lower cost, higher speed, but it’s not applicable every time, Danny. The market is not huge as many people are talking about. It is a growing market, but there are still a lot of logistic limitations in using a full decentralized model when you run a trial.

Daniel Levine: Well, what problems do decentralized trials solve and what challenges do they create?

Gabriele Brambilla: In terms of solving, I think that the biggest problem in a trial at the beginning is really how do I find patients? And you want to be able to find them in a bigger pool or bigger geography. And so when you think about the use of what is called eConsent, so the ability to recruit patients throughout a digital platform and not having to see them at a site—could be an outpatient center, an inpatient center—that accelerates the whole process and creates a lot more diversity in the recruitment; that’s for sure. So, that’s great. The challenge, though, is that not every time you can apply that model, not every time you can use eConsent. For example, in the U.S. it is much simpler to use an eConsent than in Europe [where] there are certain markets where the eConsent is not fully validated for certain types of trials, thinking about my home country in Italy, for example. So, there are some technical limitations around that for sure. And central is also the need that sometimes you really need to take certain types of tests, right? A full decentralization means you don’t have to visit the patient physically anytime during the study—very hard, not a lot of studies are like that. You still have to do a blood draw. You still have to do an x-ray. You still have to do a particular analysis and you still have to see the patient. So, that’s why most of the decentralized clinical trials, I think over 80 to 90 percent right now are done in what is called a hybrid model. You have maybe the use of the EPRO, you maybe have the use of the eConsent in just one part of the study, what is called a virtual site or a meta site, but still you need patients to go back to the site to have certain things done. So again, the hybrid model probably is the one that is the most utilized right now in the market in the United States and Europe.

Daniel Levine: As sponsors and regulators have both gotten more experienced with the centralized trials. What’s been learned?

Gabriele Brambilla: Get a lot more experienced—I put a question mark, Danny, because let me tell you this—I’m not the smartest guy on earth, but in the last few years, because of all these acquisitions and money we had to invest in these acquisitions, we did a lot of study and we had to understand a lot of the nitty, really small details of the technologies. And I have to say a lot of the people that I talked to that are claiming to be KOLs, claiming to be front runners in this change of moving from a site central to a decentralized, they do not know the complexity of running from a clinical operation, a clinical trial. And so when people are saying, and sorry if I get too technical, but maybe some people will understand, I can dispose of what is called a EDC, which is the heart of where all the data are collected in a clinical study and just run everything through my EPRO, which is the dream of some companies out there, I think is a very difficult battle, extremely difficult. So, still 90 percent of the studies—phase 1, phase 2, phase 3, not phase 4—they want an EDC because the data managers that are running the study are trained to use an EDC. So, when we think about the learning experience, that the change between the site centric to the centralized is going not as fast as people expected and is faster in phase 4, because you can really not utilize an EDC and bring everything to an EPRO. It’s a lot more difficult in phase 2 and phase 3. So, it becomes almost an addition to the standard model, but it’s not totally changing it. And so that when you say challenges, there are a lot of technical challenges and legacy situations that you cannot change as quickly that are slowing down the penetration. I say that as one of the major players in the market, that’s a challenge. That’s a barrier we’re coping with.

Daniel Levine: Even though the physical trial is decentralized, are you saying that the data gathering is not happening in a single platform at once?

Gabriele Brambilla: No. What I’m saying is that when you run a clinical trial, all the data that you collect, usually going in what is called an EDC, which is the heart to where all data go. When you decentralize, most of the players out there are saying, you don’t need an EDC just grow to an EPRO. So, the patient controls everything and all the repository calls are in the EPRO. That’s a major change. And that change is not happening yet. Especially in phase 2, phase 3 clinical trials where pharmaceutical companies are reluctant to move away from a model they’re very comfortable with where they’re data managers that are driving the choice of a technology platform to run a clinical trial, [and] are dictating what they want because they’re trained on that. So, the change is slower because they’re used to using a certain approach that is not as easy changeable, right? And the decision makers are really sometimes not the chief medical officer of the company, but the data manager, the manager who will have to process the data. I always work with EDC. I’m not going move away from that. You can sit on top of the other technology to decentralize, but I still want my EDC. So, that is the type of things that are happening in the market, where again, it’s part of the process. I just want to be realistic with the audience and the people. It is an incredible, interesting market that is growing, but it’s not yet the multi-billion market that media or other players are trying to say it is right now.

Daniel Levine: Well, explain to me how Alira works with its customers with regards to decentralized clinical trials. What role does it play in terms of design, recruitment, and actual operations of a decentralized trial?

Gabriele Brambilla: So, Alira Health. There are different players in the market. There are players that are purely SaaS players and selling only the technologies, like a metadata, and doing very well. And there are companies that are selling a consulting and service component trust technology. So we are part of this second bucket. And we entered very early. We entered at the regulatory level, even for an approval of a study. So, we really impacted the clinical protocol design. We impacted the clinical strategy. And then we bring our technology. So, the technology is empowering services, not a purely SaaS offering for Alira Health. And we ran technology with our own data managers with our own CRAs that are the clinical operators of a clinical trial. So, it’s more of a bundle between technology and services, which is becoming very successful because the market is not as mature to just buy technology only. The market has to be educated on how to use the technology and our service approach might help in certain cases to convince the client to move to a decentralized model.

Daniel Levine: Earlier this year, Alira acquired the digital health company Self Care Catalysts and its digital health platform, Health Storylines. What is Health Storylines and how does it work?

Gabriele Brambilla: Health Storylines is a little bit different than the other technologies out there and we decided to go in and acquire them. We’re super excited about it because it’s the only that we are aware of, is the only technology that is engaging with patients even before there is the need of a clinical trial or the start of a clinical trial. So, what happens is that Storyline has been developed as an application for patients to manage their disease. You go on the platform, you can download it from the app store and you have the platform for epilepsy patients, for prostate cancer patients. And they use it to treat and better manage their disease and their conditions because of that. We already have 120,000 patients on the platform. What we do then, we plug in on this app all the other part of the platform that allows to collect clinical grade data from patients. So, it’s not just a platform that you deploy once you have a clinical trial and you recruit only for the clinical trial. It is a patient community app that develops a community. And then we can tap into that community to run eventual trials. So, it helps a lot from the recruitment perspective because you already have a patient community utilizing the platform that you can plug into a clinical study or a real world evidence study.

Daniel Levine: How does this fit in with other earlier offerings to build out your capabilities in decentralized clinical trials?

Gabriele Brambilla: It’s beyond decentralized clinical trials because out storyline works in the clinical environment to support our clinical operations and data management phase 2, phase 3, and phase 4, but it also supports our commercial services in what is called a PSP—patient support programs. Whenever you launch a drug, most of the time you also launch a digital PSP. So, you launch a drug and you launch an app that will help the patient to correctly use or take the drug and try to increase the effectiveness of the drug. The platform, the technology, is also used for PSP purposes, not only for clinical research, but for also commercial use. So, it is really one single platform that can support the whole spectrum of our services from early clinical research to product launch and commercial and lifecycle management.

Daniel Levine: How much control do patients have over their own data and the data that’s collected?

Gabriele Brambilla: Full control because whenever you are on the platform, for example, Self Care Catalysts in the case of Storyline, they have a waiver. They have to go through and give us access, according to HIPAA compliance, to their data. They know that data can be used for particular purposes. They know they can be invited to the app to participate in clinical trials. So, the most important part is really what to develop the app for the patient first so that they download it, not because they want to be part of a study, but because they want to find an application that will help manage their condition. So you become partnered with them that way. They’re not just a subject of a study, they’re human people with a condition that we’re trying to help to manage better. From there, you gain the trust. And if you’re very transparent on how you are recruiting them or informing them, or educating them on how they’re going to be involved in a clinical trial—that’s how we manage it. And obviously then, everything is HIPAA compliant and GDPR compliant. All the things have to be done legally, but everything starts from a real true partnership with the patient at the beginning, independently from a clinical study.

Daniel Levine: What’s the consent process like and what’s a patient does consent, how easily can they change their consent decisions?

Gabriele Brambilla: Oh, they can get off of the platform very easily. The consent to participate in the app to manage the disease is one type of consent. And then there is the consent that you have to go through if you want to be part of a study. Being part of a study in a real world evidence study, where it is not interventional so you don’t have to take a drug, you’re just part of a cohort and they monitor you, is a type of light consent, I would say. Whereas when you go in a phase 2, phase 3, which is an interventional study, and you are supposed to take a drug and then be monitored for that drug, the consent paper is a lot heavier and a lot more detail. So, depending on which study you are going to be enrolled in, or not even a study, just depending on just being registered on the app, the consents are very different and always very transparent because we need to be very, very open with our partners, the patients.

Daniel Levine: There’s growing interest in real world data, in both capturing that and leveraging it. Are there ways Alira can leverage this platform to answer research questions outside of a clinical study for using data in ways that it may not have initially been gathered for?

Gabriele Brambilla: Absolutely. And that’s why we decide to buy Health Storyline because they already have patients on the platform and, independently from the study, are filling their diary, are giving information about their conditions. They’re talking to their peers, because there is a community feature about their conditions. And therefore, all that data can be used in an anonymous way, de-identified way, can be used for real world evidence analysis, right? And that’s what we do. And we have data in certain type of core, could be neck cancer, could be epilepsy, ADHD. Depending on the number of patients we have on the platform, we can run all sorts of analysis to help research, regulatory, and also to understand the effectiveness of a particular let’s say drug or diagnosis, or therapy in a particular cohort.

Daniel Levine: At the same time, what’s the potential for patient organizations to use this platform to conduct natural history or other types of studies?

Gabriele Brambilla: That’s the narrative strength for ours because they like the fact that the app doesn’t have to work only in a clinical trial, inside of a clinical trial. We have partnership with over, I think 10 now, patient advocacy groups and patient associations that are using the platform to engage with their members, their patients, or even to collect their real word evidence that then is used by sponsors to do their analysis. I think we have different ones that goes from diabetes to myasthenia gravis, which is a particular condition, to asthma and COPD. So, all those are public out there as partnerships. And we’re very proud of those because again the most important thing that the patient advocacy group has is the trust of their patients. The fact that they’re selecting our platform to be deployed to their members is a great honor and a confirmation that it is a platform built for patients first.

Daniel Levine: As you think about the work you’re doing and the ability to harness technology, how do you ultimately see this changing the way we gather data and conduct clinical trials?

Gabriele Brambilla: As we said before, I think that the health equity component or diversity component is going to be huge, right? The fact that because we had to go through a site centric approach, sometimes we cannot reach the minorities we would like to reach, the diversity we like to reach. Even the FDA came out with new guidance about diversity in clinical trials. These new technologies allow that and that’s great. I think it is a major fact. Think about vaccines where we didn’t have enough real world evidence, data, or clinical data for certain minorities and minorities were not comfortable in taking the vaccine. So, all that is going to be improved or changed. And that’s great beyond clinical trials. I think that there is a strong empowerment of patients boosted by COVID too. Patients now talk about vaccines, talk about clinical research, talk at their dinner table about phase 2 and phase 3. I do that at home and my kids now understand better what I do because it’s part of the media. It’s part of what people are talking about out there. So, the patient feels a lot more empowered, the patients understand now that their data is very valuable. So according to me, five years from now, patients will start to say, “Hey, what do you mean, I want to be paid for that data.” They’re going to start to ask for some sort of remuneration for the sharing of their data, because that’s very valuable information. How to pay them—cryptocurrency or whatever, but whoever is going to figure that out and be there in the next five years to offer that option to patients is going to be on the forefront of innovation, because patients are feeling empowered. Now they know that the data are very valuable

Daniel Levine: Gabriele Brambilla, CEO, Alira Health. Gabriele, thanks so much for your time today.

Gabriele Brambilla: Thank you, Danny. Thank you so much.

This transcript has been edited for clarity and readability.