3 Reasons Why Decentralized Trial Models Using Mobile Research Nurses Yield Greater Financial Returns
Decentralized trials (DCTs) have grown in acceptance since the COVID pandemic when mobile research nurses were asked to step in and save hundreds of site-based clinical trials that were in jeopardy of being postponed or cancelled because of mandated lockdowns and fear of spreading the virus. Despite their proven ability to persevere against one of Biopharma’s greatest challenges, DCTs are still often an afterthought or an add-on option to site-based trials due to cost concerns.
The perception has been that deploying mobile research nurses to visit patients at their own homes would be more expensive than site-based trials. Even in some directly comparable examples, this may not be true e.g., if chemotherapy patients need to have their blood drawn a specific number of hours following a treatment. Such blood draws done at a traditional site may require extended office hours, phlebotomists and lab access—none of which would be required with a home visit. However, this simplified view of the cost of a home visit instead of a patient visiting a site does not consider the most expensive, albeit indirect, total cost/value of clinical trials. A more holistic (and better) comparison is to consider the total cost of a pipeline of clinical trials vs a portfolio of those which incorporate DCT elements.
The problem has been that, until recently, there was no verifiable cost data to document the financial benefits of a DCT. That’s no longer the case thanks to a recent study by the Tufts Center for the Study of Drug Development (Tufts). This study shows that, not only are DCTs more patient-centric, but they also provide comparatively high returns on investments for sponsors.
Tufts data available on the impact of DCTs shows that applying DCT methods in Phase II clinical trials results in an increase in value of $8.6 million, on average, per investigational drug, or nearly a five-fold return on investment (ROI). In addition, in a portfolio of Phase III drugs, DCT methods increase value by $41 million per drug, with a 13-fold ROI. There is an increase in value of $20.4 million per Phase II investigational drug when drug developers apply DCT methods to both Phase II and Phase III trials.
While the Tufts study is the most comprehensive, to date, on the financial benefits of DCTs, it is not the first or only source to suggest that, for many studies (especially those involving rare diseases), it is more cost effective to engage a few sites and extend their reach with mobile research nurses than to engage many sites, each with only one or two study participants.
A recent case study additionally confirmed DCTs to be less costly than site-based trials. Dr. Steven Cummings, executive director of the San Francisco Coordinating Center and lead investigator on the largest Parkinson’s study, estimates a cost savings of 80% per participant compared with a similar trial conducted at a traditional site.
3 key factors driving the positive financial impact of DCTs
How are these significant cost savings accounted for? The Tufts study revealed three main factors that impact development finances positively. There are fewer substantial protocol amendments, decreases in screen failure rates and reductions in clinical phase cycle times.
1. Fewer substantive protocol amendments
Substantial protocol amendments are often conducted to address difficulties in enrolling trial participants. Deploying mobile research nurses to visit participants at home has been noted to enhance recruiting. For instance, a recent survey of cancer patients conducted by the Mayo Clinic illustrates that patients favor DCTs. In the survey, 58% said traveling is a barrier for trials and 63% said a remote trial option would make them more likely to participate.
Benchmark data show that the number of substantive protocol amendments, on average, for Phase II trials is 3.3 for trials with no DCT elements and 2.4 for trials with DCT elements. For Phase III trials, the number of average substantive protocol amendments is 3.4 for non-DCT trials and 3.2 for DCT trials.
2. Decreases in screen failure rates
Screen failures occur when research staff put potential participants through a screening process to ensure they fit the inclusion criteria for a clinical trial, only for those potential participants to not enroll in the trial.
DCT elements offer increased opportunities to participate more easily, flexibly, conveniently and at a remote location of patients’ choosing. Recruiting patients is generally made easier when patients don’t feel like they must commit to driving repeatedly to a site over the duration of the trial, along with the typical issues of making family and work arrangements. While some visits may not be conducive to a home visit, many others are and could lower the threshold of patients’ willingness to enroll. The use of mobile research nurses to support remote clinical trial activity results in decreases in screen failure rates.
The Tufts data and analysis suggest that screen failures account for 11% of trial costs. The data also indicate that the average screen failure rate for trials without DCT elements is 31.5% for Phase II and 29.9% for Phase III vs those with DCT at 24.1% and 20.1%, respectively
3. Reduction in clinical phase cycle times
Mobile research nurses, like those directly managed and trained by PCM Trials, a leading provider of mobile research nurses, play a central role in reducing phase cycle times. Deploying mobile research nurses to patients’ homes allows better compliance to protocol, decreases missed visits due to patients encountering challenges (be it family or economic driven) and encourages patients to stay with the trials. Most of us have witnessed patients who tried their best to adhere to visit schedules, yet all it takes is a snowstorm or deep freeze, an unreliable car or family caregivers who normally drive the patient getting sick themselves, and the visit windows may be missed. DCT trials, according to Claudia Lucchinetti, director of Mayo Clinic Center for Clinical and Translational Research, could have up to a 90% higher retention rate than the more-traditional models.
Measured from the start of one phase to the start of the next, the Tufts study showed reductions in cycle times have the greatest impact on value in both Phase II and Phase III trials. Benchmark data specifically shows that Phase II and Phase III durations can be reduced at least 10%, or three months, for each phase. The reductions in time bring revenues for drugs that make it to market closer to the start of phase testing, thus increasing cash flows after discounting.
Benefits beyond the cost savings
While the Tufts study and various high-profile case studies prove DCTs are financially advantageous, they are increasingly important for inclusion and diversity reasons, as well.
In its November 2020 guidance, The FDA encouraged sponsors to make trial participation less burdensome on patients and recommended that sponsors “consider the use of mobile medical professionals, such as nurses and phlebotomists, to visit participants at their locations instead of requiring participants to visit distant clinical trial sites…”
Much of the reason behind the FDA guidance was based on a need to increase diversity in clinical trials so that participants better mirror the patients most likely to benefit from the investigational drugs.
According to Dr. Lucchinetti, Black people make up 13% of the American population but represent less than 5% of clinical trials. Hispanic people are about 18% of the American population but less than 1% of participants in clinical trials.
Besides enhancing diversity by removing many of the barriers that prevent certain populations from participating in clinical trials, mobile research nurses can reduce anxiety among patients, especially when they live in the same communities as the study participants, look like them, and visit them in the familiar environment of their homes. This enhances the comfort level for patients, as compared to what they may experience travelling to research sites which often are in unfamiliar neighborhoods far from where they live and where they must interface with unfamiliar personnel in an institutional setting that may feel cold and intimidating. PCM Trials is an example of a company that focuses on recruiting its mobile research nurses from the local communities to establish greater rapport with patients.
It’s important to note that mobile research nurse visits can be initiated during protocol development or later, as a rescue strategy. Evidence gathered to date suggests, however, superior in-window results when mobile protocols are built into the trial from the beginning, rather than treated as an afterthought that may require adjustments in the protocols. PCM Trials, for example, was able to maintain nearly a 100% in-window rate even during the peak of the pandemic because it was able to adapt to existing trial protocols.