Laying the Groundwork to Measure the Impact of Decentralized Clinical Trials (DCTs)

The Tufts CSDD Roundtable took place in November, and on behalf of Veristat, I was honored to participate in this invite-only program. It is part of our company’s DNA to stay ahead of the driving forces in the industry so that we can best serve as a knowledgeable partner for our clients, most of whom are small to mid size organizations involved in rare diseases and novel therapies. Ken Getz, the Tufts CSDD team, my pharmaceutical and CRO colleagues and I came prepared to engage in a frank and open dialogue on quantifying the value proposition of decentralized clinical trial (DCT) deployments.

The goals of the roundtable were to:

• come to a consensus on the meaning of DCT
• identify the challenges to DCT adoption
• begin to understand how best to evaluate
• the value proposition for DCT deployments
• identify the metrics and KPIs to measure their ROI

Following are my key takeaways from the roundtable discussion.

What exactly are DCTs?

In principal, DCTs are defined as studies executed through telemedicine and mobile/local healthcare providers, using processes and technology differing from the traditional clinical trial model.¹ In practice, a DCT can mean something different to each of us. Those of us participation in the roundtable came to collective agreement that, at their core, DCTs deliver the technology and process improvements in such a way so as to make a trial more accessible and convenient for patients. To make this happen, how then do we, as DCT champions, continue to improve trial design and operationalize the trial so that each study is fully focused on what is important to patients and encourage their ongoing participation. 

What are the challenges associated with sponsors adopting DCTs?

The challenges that rose to the top in terms of those hurdles most pronounced included:

• Site and PI engagement: It is important to make mention of the fact that there wasn't representation from a site at the roundtable. However, those in attendance shared their perspective that when a large majority of patient interactions in a study occurred virtually PIs could feel as thought they had lost 'touch' with their patient as they were not physically 'seeing' them. Adding to this, if PIs were not seeing them, or were not seeing them as often, they may question whether the care of the patient was being hindered. 
• Ensuring a purpose fit deployment model: Interestingly, some pharmaceutical companies have a policy in place requiring that 100% of phase 2 and phase 3 trials have a DCT component, or that one DCT component be considered for every trial conducted. The concern shared was whether sponsors would be promoted to implement DCTs to meet their employment targets, without fully factoring in whether it would make sense for the study. 
• Demonstrating a favorable ROI from up-front cost investments: Start-up costs that go into conducting a DCT could be perceived as out-of-reach, or a model that does not make sense for the small-mid size biotechs. How can we help to establish real-world scenarios that would facilitate the decision-making process for a sponsor so that they would be better able to clearly conclude whether investing in DCTs would be a smart move.

How do we work to remediate DCT adoption challenges?

The challenges outlined by roundtable attendees subsequently drove our thought process to arrive at the following must-haves as we support a sponsor's ability to weigh the benefits of DCT adoption:

• Whatever solutions we bring to bear, we need to account for patients getting the same level of care and service from their physician involved in a DCT study as they would when seen by a physician at a brick and mortar facility.
• Support elements of a DCT that were purpose-fit for the trial and established during the design of the study, rather than added in as an after-thought. I recall the days when the COVID pandemic focused us to retrofit virtual components into a study. As we emerge from the pandemic's grip, we now have an opportunity to be proactive with our solutions, not reactive. From targeted e-consent deployment to in-home patient visits all the way to a fully virtual trial, it is important that we select the right approach for that particular study. A DCT solution does not need to be all or nothing as a sponsor decides what makes sense for the patient, the sites, and the trial as a whole.
• Clearly demonstrate what DCTs can offer even the smaller sponsors. What could DCT deployment actually save, and what could a DCT deployment-ready stance do, to support a sponsor's long-term growth trajectory?

What were the DCT KPIs that made it onto our wish list?

The roundtable participants agreed that, overall, the experiences over the past few years have shed light on the ways in which decentralized solutions can support or augment clinical trials. The time as come to better define how we measure their deployment success.

Together we arrived at a DCT KPI wish list:

Quantitative metrics:

• Patient participation in clinical trials. Did we see an increase... a decrease... no impact?
• Cycle times. Was the first patient/last patient in faster... did the data lock happen more quickly?
• Faster time to recruit. Did the recruitment stage close more quickly?
• Increased patient retention. Was the retention rate higher in the DCT-depolyed trial?
• Cost/value ROI. Was the rate of return on the initial DCT investment a favorable one?

Qualitative metrics:

• Patient diversity
  Did we see a more diverse patient population through expanded access?
  
  
• Gender/ethnicity equality
  Was there balance improvement across and among gender and race?
  
  
• Patient satisfaction
  Were patients more satisfied participating in a DCT trial when compared to a traditional trial?
  
  
• Change in the roles of those involved in conducting studies
  What was the impact of the inclusion of digital/virtual component on the roles of the study team (e.g., CRAs, project managers, data managers...)?
  
  
• Change in staffing requirements
  What was the impact of the DCT on staffing levels as digital/virtual/at home components were introduced?
  
  
• Change in work processes and data requirements: What was the impact on study workflows, data monitoring and management, data types collected when the DCT was deployed?

We acknowledged the work ahead as DCT's KPIs are finalized and brought to the forefront. Complicating matters further, the headwinds we face are significant, and include:

• ROI parameters are not yet well-defined
• Mechanisms by which to universally measure the impact of DCTs are not in place
• There is no reliable baseline from which to company DCTs against non-DCT trials

Further discussion pointed to the need to integrate study demographic data in order to holistically and realistically evaluate DCT's impact. From my perspective, I shared that enrollment data will be influenced by such demographic data as the sites selected, the therapeutic area, patient recruitment activities (e.g., social media, patient advocacy groups, TV spots, etc.), and such. This broader context will be critical to the small-mid-size biotechs as the demographic study profile of a rare disease trial with a narrow patient population is vastly different from a trial being conducted by a large Pharma for a drug involving thousands of patents. 

As we progress forward on quantifying the value proposition of DCT deployments, having Tufts CSDD take the helm on this important endeavor as an independent, non-biased authority and visionary in this space will no doubt serve the needs of patients and our global clinical research community exceptionally well.

DCTs and their future value at Veristat

While our clients at Veristat are sensitive to the costs associated with deploying DCTs, there is an underlying enthusiasm for the many aspects of DCTs that uniquely address the needs of patients, the study, and the sites.

DCTs should not be a separate standalone consideration. Their evaluation requires that sponsors take into consideration not only the technologies being deployed, but also the people and the processes being impacted. At Veristat, we have uniquely combined our full-service offering with our DCT solutions to work synergistically in order to have a positive impact on patient centricity, site engagement and data optimization.

Veristat has successfully implemented a number of DCT solutions across several studies. In a recent clinical trial involving pediatric patients for Canavan disease, for example, DCT integrations helped our sponsor far exceed their enrollment targets.

We suggest to sponsors that they consider a walk-before-they run approach to DCT deployment. With e-consent, for example, this aspect of the study can be implemented with a relatively low upfront investment. Through its use, we are seeing e-consent deliver a solid return on efficiency.

Undoubtedly, DCTs are evolving and we will continue to see increased adoption, but in a thoughtful way. As we do, Veristat is ready to help our clients optimize their value in order to bring novel therapies to patients faster and more effectively.

Cindy Henderson

Chief Strategy Officer, Veristat

Cindy Henderson is the Chief Strategy Officer at Veristat, responsible for commercial strategy and execution. With more than 30 years of industry experience, 15 with Veristat, Cindy provides the innovative leadership necessary to support the intricate and dynamic requirements of Veristat's global client base.

Meet Veristat. Learn More.

• Integrated DCT Solutions
  
  Listen to the DTRA Conference Presentation Showcasing an example of DCT solutions for a rare and complex study

1 - Clinical Trial Transformation Initiative, CTTI Recommendations: Decentralized Clinical Trials 2 (2018), available here