On June 24, 2021, I was honored to moderate a dynamic roundtable discussion with an outstanding group of industry experts. Panelists from Pfizer, Merck KGaA, site networks, and decentralized clinical trial consulting firms shared their knowledge and focus on innovation in clinical research, in a highly informative discussion.
Our goal for this series of special reports is to provide you with the insights and pragmatic suggestions provided by our panel, as well as from the many valuable contributions from the 40+ industry colleagues in attendance. If you are interested in attending a future session of our clinical executive roundtable, please contact us.
Decentralized Clinical Trials: A Terrible Name for a Promising Innovation
When it comes to improving clinical trial efficiency, as well as promising a host of other benefits for patients and researchers, decentralized clinical trials (DCTs) have quickly dominated industry discussions and activities. In large part accelerated by the global pandemic, DCTs will play a key role improving clinical trials – but come with a host of challenges, according to our panelists.
And if our panelists had their way, the name decentralized clinical trial itself would disappear.
Why? While our industry has adopted decentralized clinical trials as the name for this approach, it really means nothing since clinical research has been decentralized for decades. Every trial has multiple vendors and investigative sites that are in various regions or countries. The DCT name detracts from our real goal: bringing clinical research closer to the patient and into their homes, making it far easier to participate in clinical trials based on their personal preferences.
Direct-to-patient trials, patient-centric trials, home-based trials: all are better ways to think about what we as an industry are trying to accomplish.
In fact, a key cross-industry initiative in this area is called "Trials@Home." Comprised of leading academic institutions, biopharmaceutical companies, and contract research organizations (CROs), the group’s mission statement sums up this new mindset well:
“The Trials@Home consortium will explore the opportunities of moving clinical trials from the traditional clinic setting to the participant’s immediate surroundings. These so-called Remote Decentralised Clinical Trials (RDCTs) make use of new – digital – innovations and enable participants to visit a clinical trial centre less frequently, if at all.”
So when thinking about it this way, the potential benefits of direct-to-patient clinical trials become clear:
Recruit from a larger and more diverse patient population. By moving clinical research closer to patients – for example to their homes or to their local clinic rather than to an inconvenient investigative site – we open up trials to far more people, making recruitment faster and easier.
Increase patient engagement and retention. According to a 2020 report by Tufts Center for the Study of Drug Development, the average patient dropout rate increased to 19.1% during the seven-year time period of the study. Adapting clinical research to how people live their lives means patient satisfaction and retention improve dramatically - significantly reducing the cost and risk of patients dropping out.
Collect higher-quality, real-world data. With the rapidly increasing adoption of technologies including smartphones, wearable sensors, and telemedicine, and by widening the patient pool, researchers are replacing data “snapshots” (limited data gathered from site visits) with a steady stream of real-world data (captured daily and in the moment, in the patient’s own environment).
Vision, Meet Reality: Implementing Direct-to-Patient Clinical Trials from the Site Perspective
As our panelists pointed out, however, this promise of increased clinical trial efficiency and quality comes with a host of challenges. Among them:
- A direct-to-patient / decentralized trial approach is not effective for every therapeutic indication (e.g. serious and / or rare diseases)
- Even in indications conducive to a DCT approach, increasing patient options and choice does not mean they will all choose home care – for many patients, a visit to the doctor or a medical facility provides a critical social element in their lives
- Many underserved and hard-to-reach patient populations do not have broadband Internet capabilities, smartphones, or the technology “savvy” required to take advantage of remote monitoring technologies
- Decentralized trial approaches and the requisite new technologies and processes involved often increase complexity rather than reduce it, for example when an electronic informed consent process is combined with a paper-based one
- The increased volume, variety, and velocity of data coming from patient devices has found many organizations woefully unprepared to manage this flood of data to unlock its value
One challenge stood out in our roundtable discussion: the added demands of new technologies on people and processes at investigative sites. Principal Investigators and investigative sites will continue to play a critical role in decentralized trials; in fact, for safety and endpoint management that role will actually increase in importance. Yet with a heavy focus on technology implementation, DCTs are introducing more complexity for people and processes to manage.
Patient-Centric Trials Require Patient-Centric Technology and Trial Design
Sites are already inundated with different clinical systems dictated by the various trial sponsors and CROs they work with. Clinical professionals at sites are expected to learn dozens of different systems, all while managing patient care. The result is cognitive overload causing increased error rates and reduced efficiency – and adding new DCT technologies to the mix, without forethought and proper processes in place, will simply make this problem worse.
When sponsors and CROs take the proper steps, however, decentralized clinical trials can demonstrate genuine improvements in efficiency, while maintaining high standards of patient care and delivering quality data.
Our panelists explained some key principles behind these successful patient-centric trials:
1. Utilizing technologies and processes designed with patients and sites in mind: Before implementing technology for technology’s sake in a decentralized clinical trial, consider the following questions from the patient and investigator point of view:
- What existing trial processes does this technology improve or eliminate?
- What processes does this technology make more difficult?
- Does this technology eliminate or add the need for other technologies?
- How much company-specific customization is required to utilize the technology? How much training?
- How will patients in the trial access this technology?
- Will every patient have the technology infrastructure in place required to use the technology, such as broadband, a smartphone, tablet, or PC?
- Does using the technology properly require patients to be “tech-savvy”?
- What are the options if the technology fails?
- Are there ethical or regulatory considerations we need to take into account because of the technology, such as patient privacy?
- Can we reduce trial complexity? For example, is that secondary endpoint absolutely necessary?
- Can we take trial components that would force patients to travel to a site and replace them with digital endpoints that can be done at home?
- How can we build in more choice and flexibility for patients? For example, accommodating different patient preferences for how often they see their physician?
- Would I be inclined to participate in the clinical trial the way it is structured? What would keep me from doing so?
- Have we developed a change management plan for internal staff, CROs, and sites that incorporates all new or changed processes and technologies?
Closing Thought: A New Business Model for Sites In Patient-Centric Clinical Trials?
With investigative sites being a critical component in the clinical trial infrastructure, our panelists expressed concern that sites could see less revenue per patient in a DCT model. If a patient-centric trial is not designed for success from the beginning – with accelerated and expanded enrollment and more streamlined processes – the best clinical research sites and best investigators could start declining to participate in decentralized trials.
One proposal is for sponsors and CROs to compensate sites for all the work they do – not just per patient. This means paying sites not just for patient-focused activities, but for all required additional work as well, ranging from technical help desk to system administration.
Food for thought – and an important consideration as our industry continues to explore the innovation coming from the simple act of putting patients where they should have always been: at the center of our universe.
- Tufts CSDD Impact Report, January/February Vol. 22 No. 1: “New Global Recruitment Performance Benchmarks Yield Mixed Results”; https://csdd.tufts.edu/impact-reports