Using Clinical Trials Digitalization to Optimize Healthcare

In today's world, digital technology has become an integral part of the daily routines of billions worldwide. The extensive adoption of this digital acceleration has benefited everyone, including clinical research, by providing insights into the daily lives of both consumers and patients.

Similarly, medical practice has advanced using interoperable Electronic Health Records (EHR), meaning, information can be drawn from multiple service providers and health organizations.

EHRs are among the best tools to protect the patients’ health and safety. It also helps to ensure the integrity of the trial if healthy patient volunteers attempt to participate in multiple trials, possibly with conflicting medications that could bias results or threaten their own health.

Wearable devices like Holter Monitors for ambulatory ECGs with remote reporting are becoming ubiquitous in the form of digital watches that perform the same function and can report through Internet of Things (IoT) technology. Even with virtual trials, investigators are no longer confined to periodic readings when the patient visits the clinic. They look at event reports as they come in, or program reporting on a schedule.

Clinical trials digitalization will also help counter widespread negative reporting bias. This is where non-conforming trials go unreported while those showing desirable results are published. If 80% show undesirable results, they vanish, and the “affirmative” 20% go into the journals. The new objective is that all reports are filed, irrespective of funding sources, so the information reflects reality.

The registration and reporting of every clinical trial can also prevent study duplication, enhance scientific scrutiny, and elevate overall quality. It showcases its significance for enhancing public health, improving medical care, and refining public perception of clinical research and discovery.

Accelerated adoption of RWE for clinical trials

“Real-world evidence (RWE) is medical evidence generated during routine patient care. There are multiple sources of RWE, including patient health records, pharmacy claims, registries, and even social media”, says the NIH. RWE follows patients after treatment, too, identifying problems or additional benefits post-treatment. This feeds directly into real-world data (RWD), which we will discuss shortly.

Real-world evidence (RWE) is now becoming the preferred standard because the medical industry is leaving the “volume-based” payment models behind in favor of models that deliver informational value. This includes those investigators utilizing personalized medicine, and demonstrating the remarkable adaptability developed out of necessity during the pandemic.

The better that global regulatory bodies understand these RWE factors, the more value we derive from pharmaceuticals, important medical device innovations, freshly developed applications, and improvements.

Artificial Intelligence (AI) has significantly increased our ability to analyze Big Data sets. Moreover, it has provided the opportunity to radically improve both efficiency and success in practical clinical trials. Digital transformation includes the prevalence of wearable devices (e.g. Apple & Android watches), digitized health records, and previously unused data sources. These provide invaluable data for clinicians and researchers.

It provides new insights that aid drug trial design, application, and execution. Whether you’re trialing generics or testing new treatments, information about the efficacy of similar drugs makes your choices in trial design more revealing and ultimately more useful.

A well-conditioned athlete from a visible minority, or a typical office worker will each have an effect on your trial results. If a participant has comorbidities, your product may have additional benefits or drawbacks to discover.

Real-world data provided marvels such as Bupropion Hydrochloride, a mild antidepressant. It performed as expected, but two to three weeks after beginning the course of treatment people began to inexplicably quit smoking. The mechanism is still unclear—but it works! That is the value of RWD.

Difference between RWD and RWE

RWD and RWE transcend the catchphrase mentality—they truly mean something and influence essential change. They help us assure that drugs and vaccines are safe and are used safely. They provide evidence that medical devices, healthcare products, and techniques are appropriate, and can be used by people all around the world.

With the vast volume of data being produced continuously worldwide, it would be foolish not to analyze it and make it part of our investigations. Simple clinical notes written in a digital format, billing claims, insurance data, and mobile device information are all valuable. Without AI to sort, collate, and extrapolate, much of its utility would be lost.

What is real-world data?

Real-world data (according to the US-FDA) is “the data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources, such as electronic health records (EHRs), claims and billing activities, product and disease registries, patient-generated data from in-home settings, and data from other sources, such as mobile devices”.

What is real-world evidence?

Robust analytics aggregate and transform real-world data (RWD) into real-world evidence (RWE). It informs us about what happens in the real world but with the professional insights of a clinical setting. It allows stakeholders to exploit the knowledge to improve efficacy, safety, and enhance patient outcomes.

There’s an undeniable gap between the clinic and the real world. Clinics often exclude comorbidities because they want “pure” results, but it is not reflective of the real world—just an idealized setting.

RWE addresses that issue and makes the results more generally applicable to people at large. It also helps to remove biases based on race, income, locale, age, and numerous other factors.

Applications for RWD/RWE with case studies

Randomized control trials (RCTs) are considered one of the most robust forms of evidence because of how adherence is tightly controlled. Everyone takes their medication at a specific interval, and blood tests ensure results are recorded precisely over time.

This however, is not reflective of the real world where people don’t follow schedules, skip doses, or end treatment early for numerous reasons. By pushing trials out into the community, you acquire results more in line with reality.

One example of RWD becoming RWE is the Salford Lung Studies. The treatment of COPD took place with thousands of patients in the community over three years. They were selected for exacerbated COPD (eCOPD), but there were no exclusions for smoking, Forced Expiratory Volume (FEV), or other spirometry values.

Their new treatment regimen turned out to be 8.4% more effective in the community than typical treatments for a similar comparison group. That is RWD becoming RWE.

In another case, the drug Avelumab received orphan drug status for the treatment of a metastatic Merkle cell carcinoma (mMCC), a rare aggressive skin cancer. Soon after, a metastatic intractable bladder cancer was added where non-hospitalized patients received infusion treatment every two weeks. They achieved a clinically meaningful and durable overall response rate (ORR) of 33% (11% complete, 22% partial). Once again, RWD became RWE, offering a treatment option for unresponsive solid tumors.

Finally, there was the case of PatientsLikeMe and amyotrophic lateral sclerosis (ALS). Here, some ALS patients were using Lithium Carbonate as a treatment, not approved for that purpose. PatientsLikeMe provided the opportunity to see if there was any efficacy. After 12 months it was revealed that the drug did not provide a clinical effect. This is a case of a negative result not being ignored, and thus providing useful data to the medical community.

Benefits of digitizing clinical trials (RWD & RWE)

Advantages

Improving evidence in digital clinical trials | RCTs are exclusive of comorbidities, adherence, demographics, etc., and don’t translate well to the real world. Using RWD and turning it into RWE tells us more about how treatments will work in practice.

Additionally, digital tools and electronic data capture systems contribute to more accurate and reliable data collection. This minimizes errors associated with manual data entry, ensuring data quality, and integrity.

Accelerating the development of new drugs and medical devices | RWD allows us to design better trials. By using clinical trial simulation it becomes easier to include a broad demographic, and, ultimately, the more efficient design lets the trials be shorter, speeding up the approval process.

As regulatory bodies become more accustomed to and supportive of digital technologies, there is potential for faster regulatory approvals and increased acceptance of RWE in decision-making.

Holistic optimization of the healthcare system | Patients can benefit throughout every stage of medical care, ranging from preventive measures and diagnosis to treatment and subsequent care. For example, in epidemiological studies, RWD can speed the assessment of risk-to-benefit comparisons.

Information contributed by patients from their own devices can significantly improve the design or treatment options for themselves. It is likely to shorten diagnoses times, meaning faster treatment.

Challenges

RTCs are exclusive. Getting enough participants can be problematic, especially for those with limited pools of participants. Ideal candidates could be too far away. Virtualized trials allow the selection of people from anywhere, as long as they or their caregivers can follow directions.

Retention can be problematic if it requires travel, attendance, scheduling, or other complications. Virtualization lets patients feel more engaged while they stay in their normal, perhaps more supportive, environment. As a result, they are more likely to finish the trial.

Clinical trials digitalization – powering clinical research with data

Real-world data and real-world evidence are powerful tools that drive growth in the clinical trials sector. They do this to the tune of a 7+% CAGR, meaning the sector will hit 152% of its current value by 2030. You can be a part of that, but it is necessary to start your digital acceleration journey today!

RWD becoming RWE—being accepted by the agencies and authorities as valid evidence—is a foregone conclusion at this point. They all see its superiority in enhancing healthcare delivery. Remember, no one leads from the middle of the parade! If you need help with your digital acceleration, then get in touch – we will gladly discuss your options.