Maurice Solomon

For many pharmaceutical organizations, decentralized clinical trials (DCTs) have quickly become a strategic priority, one that is expected to endure long after the COVID-19 pandemic ends. Once viewed as nice-to-have pilot projects, DCTs—trials conducted remotely or through local healthcare providers to improve the patient experience—arrived when Pfizer launched a virtual trial

 in 2011. While drug-based interventional DCTs only experienced a 7% CAGR between 2014 and 2019, they jumped to 77% between the second halves of 2019 and 2020 after the onset of COVID-19, according to ZS’s DCT database, referencing data from more than 1,000 trials.

Decentralized trials offer a patient-centric approach, addressing various patient needs that often go unmet in traditional designs. DCTs benefit sponsors by accelerating clinical development, enabling more representative patient access and developing a stronger evidence package than traditional trials. Yet, DCT implementation doesn’t have to be all or nothing; trial decentralization can be viewed as a spectrum, with most DCTs combining in-person and remote activities to make clinical trial participation easier for patients. But one thing is certain: COVID-19 has shifted the balance of tradeoffs for the pharma industry, and despite operational challenges that may arise during decentralization, the benefits outweigh the investment. In the era of disruption, now is the time to take the risk.

Through our work with pharma clients, we have observed four primary drivers that suggest DCTs are here to stay, even in a post-pandemic society:

 As a result of COVID-19, regulatory agencies including the FDA and European Medicines Agency have clarified their guidance on key decentralized modalities, such as telemedicine across state lines, direct-to-patient investigational product shipments and more. The FDA’s new Digital Health Center of Excellence

 to advance the use of DCTs indicates a smoother regulatory landscape through 2021 and beyond.

 Evolving technology—fueled by record-level venture capital investments

 in digital health—is enabling trial decentralization. Furthermore, horizontal and vertical integration among technology solution providers is creating more end-to-end technology solutions to deliver DCTs.

Pharma investment and capability development:

 Pharma is creating focused DCT capability groups with dedicated leadership and investing in processes and technology to operate DCTs at scale.

 Patients’ increasing use of digital healthcare, such as telemedicine and digital pharmacies, have enhanced expectations of their clinical trial experience, which we don’t expect to revert moving forward.

DCTs offer patients a more streamlined experience, reduce the burden of time-consuming in-person visits and enable wider patient access, especially in rural and underrepresented communities.

Like patients, sponsors also benefit from DCTs. As of 2016, 85% of trials failed to finish on time, financially impacting sponsors by up to $8 million per day, according to Pharmafile.

Decentralization can reduce these hurdles for sponsors by speeding up recruitment, increasing compliance and reducing drop-out rates. Additionally, collecting high-quality clinical and real-world data through DCTs can build stronger evidence packages and better reflect the value created for patients. Sponsors can use novel digital endpoints to generate differentiated label claims through DCTs as well.

Still, decentralization—like most new innovations—adds complexity to the clinical trial process. It requires stakeholders to devote time and resources before the benefits can be realized. Sponsors must learn how to effectively and flexibly implement DCTs, as well as support patients and sites on their respective learning journeys. From a regulatory perspective, DCTs are still nascent and without consistent standards, meaning sponsors may have to initially focus on select priority markets. Lastly, while initial proof points to DCTs providing value are emerging—especially for increasing recruitment speed

—more work is needed to understand their impact. Sponsors should consider ways to benchmark DCT performance versus traditional trials to understand and capture the value of DCT.

Sponsors considering DCTs must root their approach in a deep understanding of patient needs and experiences. Designing approaches and solutions that reduce the patient burden and provide the right behavioral incentives for patients will lead to better patient engagement and improved trial performance. It’s also important to recognize that sites will remain key to patient experiences: As sponsors develop DCT solutions, providing options that are adaptive to site workflow and capability will be critical.

It’s easy to assume solely decentralizing trials and introducing new technology will improve the patient experience, but this isn’t necessarily true. In fact, a multitude of disjointed study apps may even increase the burden, while home nurse visits may be more stressful than visiting a community center. Sponsors must first listen to patients and understand their needs, and then create a more personalized trial experience that meets those needs. It’s important for sponsors with diverse clinical development pipelines to tailor this exercise to specific trial patient populations, endpoints and intervention modalities. Furthermore, within each trial, sponsors should strive to enable optionality for individual patients based on their engagement preferences.

Beyond the approach itself, sponsors also must ensure that decentralization options are considered early in clinical development planning, allowing sufficient time for patient research, endpoint validation and vendor selection activities. If decentralization is an operational afterthought, the process will be more difficult to incorporate and, moreover, the extent to which the trial can be decentralized will be constrained by traditional, site-centered protocols. Shifting this thinking upstream will enable study teams to assess how decentralization can improve the patient experience.

Developing the digital capabilities needed for DCTs is also critical. There are new types of data collected in DCTs and oftentimes new insight generation opportunities from continuous data collection. Digital solutions must enable a unified user experience and one source of truth for data integration. Additionally, while the infrastructure can be gradually built—with numerous digital solutions and providers in the market—sponsors must have an enterprise architecture blueprint to guide the development to enable connectivity and optionality. Developing the DCT infrastructure without a plan would lead to future challenges with technological integration and agility.

Finally, since DCTs are cross-functional in nature, they require collaboration across sponsors’ trial design, trial operations, regulatory, digital health and innovation functions. These teams must overcome functional silos and establish new ways of working to successfully drive DCTs. Sponsors must also establish consistent processes to facilitate scaling DCTs from a proof-of-concept to an organizational capability. One key step here is organizational knowledge sharing: applying learnings rapidly and efficiently across study teams.

For sponsors wondering where to begin or focus DCT efforts across a portfolio, a combination of their business needs and feasibility of decentralization between therapy areas can act as a guide. Some therapy areas are a better fit for decentralized trials, based on factors such as the investigational medicinal product’s administration route (e.g., oral vs. infusion), type of assessment procedure (e.g., non-invasive vs. invasive biopsy) and whether the condition being studied is chronic or acute. Although ZS’s DCT database found cardiovascular, neurology and diabetes as the most common therapy areas for DCTs, they can also be applied in other therapeutic areas. In fact, oncology and infectious diseases make up 5% and 4%, respectively, of all DCTs historically. That said, for many therapy areas, full decentralization may not be feasible or right for patients; instead, identifying specific procedures that can be decentralized or combined into a single visit can drive valuable improvements to the patient experience.

As DCTs become more common, they will raise the bar for patient experience and patient data collection. They offer an opportunity to fundamentally change how we perform clinical research. As pharma companies embark on this journey, they can’t do it alone. The good news is that patients, investigators, sites and regulators are prepared to make the change and unlock the value of moving clinical trials closer to patients.

https://www.pfizer.com/news/press-release/press-release-detail/pfizer_conducts_first_virtual_clinical_trial_allowing_patients_to_participate_regardless_of_geography

https://www.fda.gov/news-events/press-announcements/fda-launches-digital-health-center-excellence

https://www.appropriations.senate.gov/imo/media/doc/AGRept.pdf

https://rockhealth.com/reports/2020-market-insights-report-chasing-a-new-equilibrium/

http://www.pharmafile.com/news/511225/clinical-trials-and-their-patients-rising-costs-and-how-stem-loss

https://www.sciencedirect.com/science/article/pii/S2451865420300740

https://www.sciencedirect.com/science/article/pii/S2451865418300358

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