Alison Holland

If 2019 was the year of personalized medicine, 2020 was the year of decentralized clinical trials (DCTs). Ready or not, the COVID-19 pandemic ushered in a new era of clinical research—the biggest advantages of which are just beginning to surface. In 2021, the life sciences industry will see more remote trial designs adopted by a larger and broader group of sponsors, sites, and patients. As a result, patient access and participation will rise to rates never seen before, more real-world evidence will be captured to enable better outcomes for more indications, and trial efficiency will jump by 50% or more.

Here are seven changes predicted to change the face of clinical research in 2021.

1. The flexibility inherent in a decentralized model will drive widespread adoption.

DCTs are each unique by design, and not one-size-fits-all. In contrast to the rigidity of traditional randomized control trials, DCTs offer a modular framework, affording choice to sponsors, sites, and patients. As adoption of this model continues to grow, these stakeholders will become accustomed to having options – just as consumers have come to expect flexibility in their retail shopping experiences. At that point, at least some aspects of trials will be stipulated to be decentralized in all protocol designs.

By the end of 2021, we will come to a tipping point where patients recognize how much easier it is to participate in DCTs, how technology enables rather than hinders, and how many ways sites and sponsors can streamline processes. The freedom to create trial designs that work best for each sponsor, each site, each patient, each protocol will be too great of a siren song. The industry will not revert to the rigid pre-pandemic way of conducting trials and will abandon long-standing behaviors to embrace opportunity.

2. Real-time patient care will change perception to increase study participation.

As DCTs become standard, researchers and clinicians will begin to adapt to receiving data in real-time. Clinical research associates will develop ways to verify the quality of data collected remotely, for instance. Likewise, healthcare professionals (HCPs) will adjust to engaging with patients more frequently, remotely at times, to ensure their health is being actively monitored and adjusted. In this new model, patients will receive the gold standard of care, and will turn to trials to receive this top-rate healthcare and be proudly part of the development of breakthrough medicines rather than viewing pharmaceutical R&D with skepticism.

For this to come together, however, DCTs will need to be built on a unified platform where all trial data is reported and visible in one system for all stakeholders. Sites must be alerted when patients input data so they can respond in real time. By the end of 2021, DCTs managed on a unified platform will garner greater trial participation, improve efficiency, and become the ultimate model for patient-centricity.

3. Remote technologies will triple patient access to clinical trials in one-third the time.

Historically, access to clinical trials has been one of the greatest hindrances to research.

Across the U.S., there is tacit acknowledgement of a two-tier system—those who have access to quality healthcare and those who do not. The difference between the two often tips the scales in terms of population representation in trials. DCTs that involve remote tools improve access by bringing the trial to the patient, removing the burdens of travel and distance to make trials feasible across demographics.

This is not theoretical. The National Center for Biotechnology Information (NCBI) found that a decentralized trial model recruited three times as many patients three times faster as the traditional model. The patients in the decentralized model also better represented urban and rural areas. These benefits are particularly important for rare diseases, as trials are few and far between and patients may be spread across wide geographic areas.

4. Greater awareness of social injustices will ensure diversity is no longer an afterthought.

As we continue to open our eyes to social injustices, more pharmaceutical companies will engage in real conversations about equality and have another important reason to embrace a decentralized approach to trials. But this is just the start. In 2021. diversity will be first-order priority from the beginning of a study rather than reverse-engineering racial equality into the trial design as a rescue mission.

Expect to see diversity increasingly baked into the early stages of study protocol design and product development this year. And it will extend beyond people of color to include different genders and ages. We will see patients from all backgrounds involved in the study process from the start. Medable’s Patient Advocacy Council (PAC) has patients ranging in age from 20 to mid-70s, a mix of female and male members, and is expanding to add minorities in early 2021. The PAC informs our products and trial design—not on the back end, but at the beginning when they can make the biggest impact.

5. Digital biomarkers will expose ‘simmering’ symptoms before they turn serious.

The fall-out of COVID-19 has accelerated the capture of digital biomarkers as more companies use DCTs and capture patient data remotely. In fact, researchers are now able to capture quality data, on a longitudinal scale, and harness that data across groups of patients to establish accurate markers that could signal important changes in the trajectory of a disease earlier.

While the digital biomarkers that can be captured remotely are often constrained by cost and logistics, there is rich data that can be collected reliably with digital tools. For instance, a daily video that shows progressive changes to a Parkinson’s patient’s shuffle, a wearable device that measures hand tremors continuously, and a digital sleep monitor that tracks REM sleep cycles all inject new factors into the research equation to help determine accurate digital signatures.

Using digital biomarkers, researchers will be able to identify “simmering” symptoms that could turn into an adverse event later in the trial. Since the development of symptoms has the potential to jeopardize patient safety, instill fear and panic—patients could drop out of a trial prematurely. Identify symptoms early, however, and they can be mitigated, preventing psychological harm and trial drop-outs. In 2021, the momentum of DCTs and remote technologies will allow clinicians and researchers to better identify digital biomarkers to help improve care and maintain the integrity of vital clinical trials.

6. The industry will (finally) proactively align on master protocols.

In 2006, the unprecedented approval of Novartis' Imatinib Mesylate for five new indications based on a single-phase II clinical trial bewildered the pharmaceutical sector. The Imatinib story is an illustration of the biomarker-driven precision medicine trial designed using the principles of master protocols. What was once used exclusively for oncology trials, master protocols are now used in other therapeutic areas. Innovative trial designs, such as DCTs, will drive greater use of master protocols in many more therapeutic areas by the end of 2021.

Many patient advocacy groups support the use of master protocols because they increase patient access and reduce the burden for all stakeholders by pre-contemplating important factors and baking them into protocol design from the start. While this approach requires more up-front work, it is far offset by the efficiency gains on the back end. For example, there are fewer administrative burdens for sites, patients are more likely to stick to the trial, and greater knowledge is gained faster.

As we move through 2021, expect pharmaceutical companies, patients, sites, and regulators to align on greater use of master protocols. Remote technologies so key to decentralized clinical trials will enable their use as well as make studies more adaptable, which benefits all stakeholders. Ultimately, this help cut cycle times in half and allow researchers to collect more robust data, in and out of the clinic, with much less burden on patients and sponsors/sites.

7. Greater access will drive radical improvements in trial efficiency and effectiveness.

As an unimagined consequence of the pandemic, the life sciences industry now sees the transformational impact that decentralization will have on clinical research. In 2020, it provided a vital stopgap to overcome the halt of clinical trials for thousands of shut-in patients and trial staff. What served as a band-aid in the short term quickly developed into a recognition that by enabling greater patient access to research, DCTs can broadly benefit sponsors, CROs, sites, and patients.

We saw faster enrollments and greater retention for patients participating from home. We saw more and higher quality, real-world data. We saw more efficient processes and greater patient experience. We saw the resistance to digital and mobile technologies melt away, as televisits and remote screening and enrollment made participation easier for more patients from more places.

As the life sciences industry continues to march forward with its adoption of a decentralized model, it is incumbent upon technology providers to continue evolving its platform and tools. Companies will not go back to pre-pandemic ways. If we continue to learn and innovate, the domino effect will drive 50-70% increases in trial access and efficiency—ultimately leading to more effective therapies for more people around the world.

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Alison Holland

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