The buzz these days in clinical research surrounds the term decentralized clinical trials (DCTs) which seems to promise it all for all stakeholders in the health ecosystem, from increasing patient participation in new trials to expanding the volume and the value of datapoints.
But is the notion of such a remote approach more hype than reality at present?
Some argue that maintaining the balance of traditional trusted approaches with innovative technological and digital advances could be the holy grail to deliver real efficiency, quality, and success in clinical research.
In recent years, orphan drug developers have led the way by designing studies that maintain the all-important, personalised connections, deliver trusted specialist support, and enhance overall transparency.
They have demonstrated that a hybrid, rather than a fully decentralized approach, is a model of success, and there is much we can all learn from the rare disease trailblazers for the future, of efficient drug development.
Can we really have it all?
For decades, drug developers have relied on traditional site model studies to assess the safety and efficacy of their products.
And for good reason. Site-based clinical trials are a tried and tested way to generate high quality data and monitor adherence to the protocol.
However, they are not without their challenges.
They can be slow and expensive, and they can place a high burden on the participants who are often asked to travel long distances to site visits.
DCTs replace site visits with remote monitoring, either through home health, telehealth, questionnaires, or sensors/wearables, making participation easier.
Yet most protocols will require at least some face-to-face participant/site interaction, and the European Medicines Agency (EMA) has advised sponsors to allow participants to visit sites should they wish.
In addition, the DCT model can erode site oversight, and, when sponsors deploy multiple vendors or platforms for different elements of a single study, increase site burden.
Hybrid: The best of both worlds
When deployed effectively, hybrid studies, which combine face-to-face and remote monitoring elements, offer the best of both worlds.
It is a model that acknowledges that each study will be different, and that is flexible enough to support the needs of participants and sites alike.
Sometimes, people prefer to travel to a site for their appointments, and some study procedures may not be suited to home health.
Consequently, not providing a “bricks and mortar” option may exclude some potential participants so it is important that patients be given the option to attend the site.
Technology and services designed to support patients, such as travel and expense reimbursement, can secure the participation of patients by making their journey, whether it is 12 minutes or 12 hours, as easy as possible.
Such advanced travel concierge services can provide a comfortable, stress-free trip, and specialist expense reimbursement solutions mean participants are never out of pocket when they chose to or are required to visit the site.
Another area of concern is that remote data collection can sometimes erode the link between investigators and participants, thus impacting trial retention.
People can feel unappreciated or disconnected, leading to dropouts and, therefore, missed data points.
Enabling two-way conversations and ensuring home health teams have all the information they need to answer questions and provide updates can help sidestep this potential pitfall.
Sponsors also need to ensure they are supporting sites.
Investigators are ultimately responsible for patient safety and the quality of the study data, so may be wary of losing oversight to home health teams.
Aligned with ICH GCP and the FDA and EMA guidelines for DCTS, home health providers will facilitate the site’s review of, or interview if preferred, healthcare professionals (HCPs) before they are deployed on any study visits.
They can also facilitate relationship building, through face-to face meetings or in-person HCP training, and enable site teams to join visits virtually, all of which increases transparency and provides assurance.
What is more, sites are often frustrated with the sheer volume of different technologies and solutions sponsors can ask them to engage with. Again, intelligent hybrid solutions can bridge this gap.
Crucially, sponsors can reduce the burden on sites by collaborating with partners who provide seamless, integrated solutions.
When services such as participant support, home health, travel and reimbursement are all operated through the same platform, it streamlines administration processes and reduces the number of moving parts sites are expected to track.
Hybrid in practice
We are living in a golden age of orphan drug development, with more new targets, candidates, and treatments than ever before now offering fresh hope to people who may previously have felt forgotten.
The shift from 100 per cent physical clinical trials, in which sponsors have been able to reduce participant burden without increasing site workload or degrading data quality, has been central to this story.
In one study, for example, the rarity of the neurogenerative condition necessitated recruitment from across the United States, in order to “widen the net” of eligibility. At the same time, the participants’ significant mobility challenges made travel to the one specialty site conducting the study highly burdensome.
To overcome these inter-impacting challenges, participants were identified via a patient recruitment company and, to enhance site oversight, consented by site personnel using telehealth. Mobile HCPs conducted remote screening, removing the need for an initial clinic visit.
A 10-day site visit was unavoidable, so the sponsor initiated a “white glove concierge” travel service which not only helped participants and their caregivers get to the site, but also supported them while they were there.
Easy to use expense reimbursement services were also initiated to remove financial barriers.
The original protocol also included two in-clinic follow up appointments, as the blood sample requirements were deemed too complex for home health.
However, working in lockstep with the sponsor and the sites, home health solution was designed that ensured sample quality was high enough to avoid degradation during transportation to the laboratory.
HCPs with Nurse Licensure Compact (NLC) licenses, meaning they can practice across multiple states in the US, were recruited.
They then underwent intense human peripheral blood mononuclear cells (PBMCs) collection training from the sponsor, and their competence tested.
The visits requiring PBMC collection were then able to be conducted in the home where desired by the participant.
The patient recruitment company that was utilised on the study have stated that without being able to offer these home visits, it would have been very difficult to get patients to agree to participate.
Importantly, the program was designed and conducted in collaboration with all stakeholders, allowing for open lines of communication right the way along the pathway.
In this case, the recruitment company stated that without being able to conduct these visits in the home, it would have been extremely hard to get patients to agree to participate.
As this case study shows, modern drug development can provide the best of all worlds, so long as it places the needs of people – whether that is the participant or the site – as the priority, with technology there to better enable and support the process.
That means embracing solutions that give people a choice on how to participate, involving everyone in study design, and enabling close and direct communication between all stakeholders.
In short, flexible, integrated solutions that can flex to the individual needs of the people involved in each individual trial are the secret to success.