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Everyday Drug Discovery: How Tech Expands The Potential Of Clinical Trials And Recentres Participants

Throughout my career in the development and implementation of technological solutions to support clinical research, I've been lucky enough to work closely with a wide range of so-called "stakeholders" in the clinical trial lifecycle, from regulators, drug development research teams, site staff, and patients. 

While all groups have demonstrated an inspiring level of dedication and passion, I have always been moved by patients' willingness to sacrifice their time and adapt their care plans to take part in clinical trials, fully understanding they might not directly benefit from the treatment being tested, though so often desperately hoping they do.

Patients recognise the importance of clinical research to the long term success in treating diseases of all kinds, and in turn the industry has touted the importance of "patient centricity", or of giving "patients a seat at the table", and "putting patients at the centre of what we're doing" as a way of bringing some kind of balance to the scale of sacrifice we ask. While such aims are noble, a lot remains to be done to translate them to tangible outcomes for patients.

Making patient centricity tangible

Clinical trials can last for years, and researchers need to follow participants' responses consistently across time to gather high-quality, relevant data in order to properly understand the impact of a disease and treatments of interest. While we talk a lot about minimising burden for patients, we cannot shy away from the fact that asking patients to participate in these trials is a significant additional burden on their already busy lives – that is just the reality of clinical research today.

In our experience, trial participants are highly informed and interested in the mechanics, objectives, and milestones of the research they are involved in and with proper onboarding they appreciate the effort that will be asked of them in the trial. However, if patients feel hassled and overwhelmed by the day-to-day business of clinical trials – long-distance travel, record keeping, financial burden, time off work, childcare – at that point, they are much more likely to drop out. And every lost patient is an indictment of our failure to do better.

This is where thoughtful use of technology can meaningfully move the needle on our claims of patient centricity. Zooming out to understand the patient's full trial experience, and developing a technology solution that supports this entire clinical trial journey – from patient recruitment to in-trial activities and study completion and exit – simplifies the experience for patients, while optimising workflows for sites. In doing so, more efficient and successful trial experiences can be provided.

How technology can empower trial participation

My focus for the last 15 years or so has been electronic clinical outcome assessment (eCOA) technologies, which expand patients' and physicians' ability to flexibly carry out asynchronous data collection, either at clinical sites or closer to "real-time" in the patient's own home. But, eConsent is a complementary method of ensuring patients have comprehensive trial information without needing to set foot in a clinic. The growth in Bring Your Own Device (BYOD), allowing patients to use their own mobile devices or web browsers to access eCOA or eConsent systems, has marked a paradigm shift away from the rigidly controlled provisioned devices traditionally used in clinical trials, to a more patient-friendly world where they can use the technology they are familiar with.

Additionally, we are seeing a significant increase in the number of active studies and protocols where eCOA and sensors are paired together to achieve more powerful insights. For example, we are seeing growing use of activity tracking wearables used with eCOA, to measure a patient's comprehensive physical activity and quality-of-life, or to enable the patient to complete an at-home six minute walk test. We're also seeing continuous glucose monitors (CGM) and eCOA used together to measure a patient's glucose levels and response to real-world events and diet, and handheld spirometers with eCOA to measure asthma patients' response to treatment before and after rescue inhaler use.

Another factor that we believe is behind the uptick in use of eCOA and sensors during a trial is the 2024 FDA guidance on use of DHTs for Remote Data Acquisition. The new guidance helps provide a much-needed set of recommendations for sponsors when using these new technologies during a trial, including recommendations on fit-for-purpose, usability, training, endpoints, data security, and record retention. These technologies used together promise deeper insight as to what patients are reporting via traditional questionnaires, as well as providing new data streams to drive novel insights.

Building flexibility into patient technologies from the beginning, for example, granting patients some choice in how they engage with their clinical trials tasks – whether it's as simple as allowing them to use their own smartphone, or as significant as allowing them to choose whether they want to do their scheduled assessments at a clinic site or from the comfort of their own home – is a significant way to help build and maintain long-term patient relationships and is a fundamental philosophy in how we approach product development. I don't believe we should think of these so-called "decentralised" technologies as a revolution or a whole new research methodology. Rather, it is an adaptable study design element – one amongst many available to us. We should have no interest in wholesale discarding methodologies proven to produce life-altering innovations, nor denying patients face-to-face time with their doctors if that's what they want; rather we should be building technologies that empower patients in their clinical trial experience.

Such technologies can play a particularly significant role in rare disease research, where the handful of people affected may be spread all over the world. Research teams cannot expect patients to travel internationally to partake in studies of potentially life-saving experimental treatments. Bringing the trial to the patient ensures study teams maximise the ability to capture every essential data point, and decentralisation can generate a sense of global connectedness to the study. Complex therapeutic areas such as the central nervous system (CNS), which are woefully lacking in good treatments, also offer fertile ground for leveraging technology to streamline trial operations and improve data quality. We are already working on this alongside our partners to deliver a fully unified platform for CNS assessment delivery and oversight.

Of course, new technologies generate new considerations. In the context of clinical research, we can face unique challenges in the form of language barriers, time zones and local regulations. Technological capacity and internet access vary considerably across geographies – yet another reason to allow flexibility in the kinds of devices we allow patients to use. However careful upfront planning, and involving patients during the study design process, ensures mitigation strategies are set up before they are needed. Importantly, introducing elements that help to decentralise clinical trials for patients is not a one-size-fits-all solution.

Building the future of clinical research

With a bit of creativity, coordination and commitment (all of which are abundant in the biopharma industry), research teams can balance global scale and the fundamental needs of the trial to capture data, with human centricity. COVID-19 vaccines, for example, were developed at the astonishing rate they were because existing decentralised data capture and delivery methods were deployed en masse in novel, interconnected ways.

At Medidata, we really value these diverse points of engagement and data capture. A traditional clinical study is a highly controlled and sterile environment. Daily life isn't. That's why flexibility, with control, is central to how we build our technologies. It also nods to a future of meaningfully lower burden, with data passively drawn from sensors worn while a patient is commuting, sleeping, at the gym or out to dinner, building out a more complete picture of how they are living with their disease or the experimental treatment.

The biopharma industry runs clinical trials to develop new, better therapies; patients participate in them because they want to help themselves and other patients. We have to remain mindful of the many emotions a patient may experience in the course of treatment.

We in the healthcare industry naturally love innovation of all sorts, but we shouldn't be innovating just for innovation's sake. Sometimes "old fashioned" methods – in-clinic assessments with a real-life human, for example – are preferred by patients. Let's take advantage of the full suite of tech solutions available to actually put participants' best interests at the fore.

2 Groundbreaking Clinical Trials That Have Helped Black Health

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Clinical trials have long been the cornerstone of medical advancement, but their impact is especially meaningful when they represent the full diversity of the populations they aim to serve. During a recent panel discussion on Clinical Trials Day, industry leaders shared powerful examples of both interventional and observational studies that have made significant differences in healthcare outcomes, particularly for Black Americans.

Moderna's COVID-19 Vaccine Trial: A Commitment to Representation

When Moderna launched its pivotal COVID-19 vaccine trial, the company made an unprecedented decision to prioritize diversity in its study population. As Nader Daoud, Associate Director of Clinical Trial Health Equity at Moderna, explained, "When we were running the trial… we saw that we had an imbalance in the number of participants of color, particularly Black, Hispanic participants."

Rather than proceeding with an unrepresentative sample, Moderna took a bold stance. "The company realized that we would have to extend the timeline in order to allow, to actually encourage people of color to be more enrolled in the trial," Daoud shared. This meant "working closely with our community partners, with investigators of color, with sites that actually had these populations."

The decision wasn't made lightly, as it delayed the vaccine's market entry. However, the results spoke for themselves—Moderna achieved approximately 37-38% enrollment of people of color, closely mirroring the U.S. Population demographics.

"It took longer for our vaccine to come to market, but we managed to enroll with thirty-seven, thirty-eight percent people of color in the U.S., which is representative of around 40% people of color that are living in the U.S. Across generations," Daoud noted.

Groundbreaking Genetic Discoveries in Parkinson's Research

Observational studies are equally vital to advancing healthcare equity. Alyssa O'Grady, Vice President of Clinical Research for the Michael J. Fox Foundation for Parkinson's Research, highlighted a remarkable discovery from 2023.

"The Global Parkinson's Genetics Program… discovered a new genetic change in people with Parkinson's of African ancestry," O'Grady explained. "People with this genetic change were found to be at a higher risk for developing Parkinson's disease."

This finding was particularly significant because "prior to the launch of this genetics program that led to this discovery, over 90% of all studies of Parkinson's genetics were focused on people of European ancestry." The discovery demonstrates the critical importance of diverse representation in medical research.

"This was a really powerful example of how much more we can learn about disease when we include folks from all communities in research," O'Grady emphasized.

The Power of Intentional Inclusion

A common theme across all successful clinical trials was intentionality. As Rolondo Enoch, MS, Director and Head of Diversity and Inclusion in Clinical Trials at Kite Pharma, succinctly put it, "Inclusive science is good science."

This perspective has transformed how companies approach their research. Daoud noted that at Moderna, "It's really about making an intentional effort to think about, okay. We did this once, but how do we do it again and again and again?"

For the Michael J. Fox Foundation, setting clear intentions was crucial. O'Grady explained their approach as "setting that intention, putting money behind funding research across the globe to make sure that we have significant numbers of people across all communities participating in research."

Personal Impact

Ricki Fairley shared her personal experience with clinical trials: "I would be dead if I didn't have a trial. I had triple-negative breast cancer. I did all of the standard of care stuff. I had a mastectomy, and my cancer came back in a year. And my doctor said, "You have two years to live. We don't have anything for you."

Fairley found and advocated for herself to join a clinical trial, taking a drug that is now the standard of care for triple-negative breast cancer. "Not only is it important to do all the right science, but it also can save your life," she emphasized.

As these examples demonstrate, when clinical trials are conducted with intentional diversity and inclusion, they not only advance scientific knowledge but also directly improve health outcomes for communities that have historically been underrepresented in medical research.

On Clinical Trials Day, CVS Shares Lessons Learned From One Year Providing Trial Services

CVS Health has come a long way from its humble beginnings as a health and beauty products store in 1963. In recent years the retail pharmacy chain has begun to delve more and more deeply into the healthcare sector – by constantly expanding the basic healthcare services offered at their instore Minute Clinics, and by buying into the health insurance insurance space with their $69 billion purchase of Aetna in 2017.

Last year, the company added clinical trials to the list with the launch of CVS Health Clinical Trial Services, an initiative that works with pharma companies on clinical trial recruitment and even hosts trial sites at certain retail locations.

On the one-year anniversary of the launch, which also coincides with International Clinical Trials Day, pharmaphorum caught up with Josh Rose, CVS's VP and head of decentralised clinical trials, site solutions, and strategy, to talk about the venture and lessons learned in the first year. This interview has been edited for length and clarity.

Take us back to May 2020. What made CVS decide to enter into the clinical trials space?

Josh Rose (JR): In many ways, it's somewhat of a natural extension of the broader strategy that CVS has in terms of becoming a much more relevant healthcare provider for patients. The unbelievable relationship that CVS has with patients through pharmacy, through Minute Clinic, through Health Hub, is a super strong relationship and one that only strengthened and intensified with COVID, where everybody associated becoming vaccinated and prepared to deal with the pandemic with their visit to a CVS for a vaccine. So if you take that as a foundation, we have a legitimate reason to play in that space because of the familiarity trust that patients have with CVS.

Why is that now important for clinical research? As you know, one of the challenges that exists in clinical research is that while patients like the idea of clinical research, there's a big hurdle for their participation, whether it's challenging for them to get to a clinical site just because of logistics and distance, or whether it's lower trust in investigational research.

When we juxtapose that with what patients think about CVS as a partner in clinical research, it's dramatically different. So that is, in essence, the primary impetus that led CVS to say, okay, let's leverage what we have in order to improve clinical research.

You have a broad audience, which works in your favour. But is there any worry about self-selection bias of the people who use the Minute Clinic?

JR: Let's flip it. What happens today in clinical research is that there is self-selection in traditional clinical research, but it tends to be actually for folks that look more like you and me. They tend to be more educated, more affluent. But in contrast, though, if you look at our national demographics, they look a lot different. They're more diverse, there's different equity. And so I think what happens in traditional clinical research is there's this bias towards folks that are a very small representation of the actual patient community. In contrast, CVS is actually much closer to the majority of patient populations, those that demonstrate and represent the real kind of diversity and demographics that we have. And because of that, we're able to attract them and make clinical research much more appealing and available.

So how does it work? Is CVS a CRO or is it more complicated than that?

JR: No, I think it's less complicated than that. Pharma comes to a CRO and says, hey, we need to find patients and we need to find investigational research sites in order to conduct our study. Whereas we have patients and we have sites, so when pharma comes to us, they don't need to act through an intermediary like a CRO to say, hey, can you go find third parties that could do it?

CVS, by nature of the business that we're in, we have access to over 130 million identified patient lives. This is through the various databases that we have, like Aetna on the insurance side or retail pharmacy or central pharmacy. We just have a plethora of data at the de-identified level, and we have the tools to be able to analyse and determine if Josh Rose, potentially based on my medical history, am I a good candidate for clinical research? And they can reach out to me.

And on the other hand, we are in the process of building out what I believe will be the industry's largest fully integrated clinical research site network, meaning we're taking our Minute Clinics and our Health Hubs, and we're converting them into fully legitimate, trial-ready clinical research locations. So we combined access to patients and clinical research sites all in one, which really allows us to provide clinical research in a way that's (A) much more human and patient centric and (B) much more efficient because you don't have all those layers of oversight that pharma is paying for.

So one year in, what are some of your big successes? What have you learned?

JR: We're seeing fantastic adoption, we're seeing very broad and strong interest for what we do on the recruitment side for pretty obvious reasons because that's an unmet need and one that needs to be fulfilled daily. The same thing for our real-world evidence business. That's one that's growing nicely.

The community-based clinical research one is one that we believe has tremendous potential, maybe even the highest growth potential in the market. But it's one that it's taken us a little bit longer to get into that space. And so that's kind of I think lesson number one is just that clinical research is a complex, regulated, established market with its own adoption curve. The path to getting adoption of our community-based clinical site solution was a little bit longer than we anticipated.

In terms of successes, we recently launched a study and our objective was to recruit 100 patients within a three- to four-week period. And within the first week we were oversubscribed.

It really demonstrates that if you have this brand that customers trust and you have the right analytics and access to be able to identify where those patients are, and then you're also able to establish an investigational research network that is friendly, consumerised and in their backyard, so to speak, you can drive the kind of metrics like you've seen on this one study.

A lot of people, when they think of decentralised trials, think telehealth. How does your model fit into that future?

JR: One of the things that I've learned is that while in theory trying to move everything to the patient's home sounds good, the reality is, (A) you can't move everything, and (B) the patient actually wants more of an omnichannel experience, if you will, with clinical trials. The patient wants to be able to interact with studies on their own terms.

So we take a hybrid study and essentially create all the channels, whether it's in one of our clinics, in a patient's home with a CVS home research nurse, or remotely with one of our staff, bring them all together in the same environment and make that choice available to the patient. We believe that is really the optimal solution, as opposed to either always ivory tower medical centre or always at the patient's home.

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