Spencer Health Solutions
ong-term retention of patients in clinical trials is both difficult and costly, which is challenging in an era in which drug development costs are increasing while success rates decrease. High dropout rates are likely to compromise study power and lead to trial failure, which can have catastrophic financial implications for drug developers. Clinical trial dropout can exceed 30% in pivotal phase 3 trials, and nearly 85% of trials fail to retain enough patients. Recruiting a single clinical trial patient can cost nearly $7,000, and the cost to replace patients that have dropped out is nearing $20,000. High dropout rates also place additional burdens on study recruitment, as failing to recruit sufficient numbers of patients is itself a cause of clinical trial failure.
Reducing Dropout Rates Through Patient-Centric Technology
Long-term patient retention relies on reducing trial participation burden and improving patient engagement, including continually reinforcing the motivation to participate in the trial, ensuring that clinical trials are designed around the patient (e.g., including endpoints that are meaningful to patients), and monitoring patient experience in the trial. Patient engagement and retention in clinical trials becomes even more problematic as trials become longer in duration and/or more complex.
The close relationship between patient engagement, retention, and clinical trial success creates a unique opportunity for technology to support and reliably engage trial participants in the home. Daily engagement in a patient’s real-world environment is far more durable than episodic engagement at clinical sites, while also improving trial convenience and reducing participation burden. Indeed, reducing travel to clinical sites has been identified as one of the most important study participation burdens, and incorporating convenience-enhancing technology significantly improves study satisfaction.
The Effectiveness of Device-Based Engagement Over Mobile Apps
When considering how best to engage patients in the home, mobile apps are a tempting choice; however, apps are not effective at long-term patient engagement. One study reported only 9% patient retention using fitness-based health apps after 28 days, while another reported only 4.1 days of mean engagement time. In another example, a symptom-tracking app was only utilized for a median of 15 days in patients with Parkinson’s and 1 day in non-Parkinson’s patients. Furthermore, app usage decreases by 42% in individuals with low incomes compared to those making more than $100,000 per year, indicating that engagement via apps may be biased.
As an alternative to mobile apps, device-based engagement is more effective. A recent report of an in-home medication-dispensing smart hub reported 98% daily medication adherence over 6 months. This type of technology repeatedly engages patients, for example, by pairing medication dispensing with basic survey questions around quality of life and health outcomes, with survey response rates exceeding 90%.
In addition to improving patient engagement, technology that enables clinical trial participation in the home can also increase patient accessibility, as an estimated 70% of patients live more than two hours away from traditional clinical study centers, and only 35% of people are willing to participate in trials at traditional research centers. Importantly, increasing trial access to a broader array of patients is a critical component to promoting clinical trial diversity, which is an imperative for promoting health equity.
For patients to willingly use engagement technology in the home over long periods of time, it needs to be easy to use, with their needs considered throughout the design process—which is particularly true for fragile populations, including children, the elderly, and the mentally ill. For example, around 40% of patients with mental illness lack foundational digital skills, placing a higher usability bar on the usage of technology for patient engagement in this population.
Any in-home technology being used in clinical trials must be fit-for-purpose, designed around the patient, and rigorously tested for patient accessibility, acceptability, and consistent engagement and utilization over time. The above-referenced study of an in-home medication-dispensing smart hub reported that 96% rated the platform as easy to use and 87% indicated that they needed a technology like this to help simplify their medication routines, while caregiver burden was also significantly reduced.
Aligning with Regulatory Initiatives and Promoting Health Equity
Importantly, the use of in-home patient engagement technology aligns with multiple FDA initiatives and guidances. First and foremost, the FDA generated the Patient-Focused Drug Development Draft Guidance Series to improve inclusion of patient voice data in clinical trials and throughout the drug development process. Based on this guidance, drug developers will be asked to:
- Collect patient voice data on symptoms, disease impact, and other factors that are critical to patients in the target population;
- Measure clinical outcomes (using clinical outcome assessments, COAs) that are most important to patients; and
- Evaluate how patients feel and function as a result of treatment to confirm that the clinical outcome assessments being collected and the magnitude of change produced by treatment are meaningful.
These types of data could be easily analyzed longitudinally through daily patient surveys using an in-home medication-dispensing engagement platform. The FDA Draft Guidance on “Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments” emphasizes the importance of optimizing signal-to-noise ratio when measuring COAs to ensure adequate sensitivity to detect treatment-related change. Using an in-home smart hub enables the collection of either Patient Reported Outcomes (PROs) or other COAs (e.g., from Bluetooth-connected peripheral devices) right in the patient’s home at the same time of day, which will provide the greatest consistency and is a valid strategy to maximize the signal-to-noise ratio. Furthermore, the FDA has an ongoing initiative to promote Clinical Trial Diversity, including the “Enhancing the Diversity of Clinical Trial Populations – Eligibility Criteria, Enrollment Practices, and Trial Designs” Guidance. This guidance highlights the need to make clinical trials less burdensome and more accessible to reduce participation barriers for people in certain genders, ages, or racial or ethnic groups. Multifunctional in-home technology that enables specific types of study virtualization, like vital sign collection and virtual visits via a telehealth connection, is a valuable tool to bring clinical trials directly to patients’ homes.
Conclusion
Long-term patient retention strategies are critical to reducing drug development cost and timeline by relieving the need to replace difficult-to-enroll patients and can be accomplished by both reducing the burden of clinical trial participation and consistently engaging patients every day in their home environments. Considering that mobile apps are at best inconsistent, or at worst biased and ineffective, for engaging patients long-term, innovations in in-home technology that keep patients retained and engaged by encouraging repeated interactions between patient and technology provide a valuable future approach. One example of this type of technology is the in-home medication-dispensing smart hub that promotes daily interactions such as removal of study medications, responding to survey or ePRO questions, measuring vital signs using Bluetooth-enabled peripheral devices, or interacting with study personnel using a telehealth connection. These repeated interactions yield durable patient retention over years (manuscript in preparation). Incorporating this type of technology into clinical trials can provide drug developers additional tools to maintain consistency with current regulatory trends, including the FDA’s initiatives on patient-focused drug development and diversity and inclusion in clinical trials.