Despite the public’s embrace of clinician home visits as part of general healthcare, clinical trial sponsors have been on a learning curve regarding implementation of home healthcare in clinical trials due to patient safety and data accuracy concerns. Adoption of decentralized clinical trial (DCT) approaches has risen in the past couple of years as an adjunct or alternative to traditional models, especially in cases where sites were inadequately resourced to keep trials running due to various reasons. Sponsors are increasingly embracing home healthcare due to the benefits, such as reduced burden for clinical trial participants and enhanced patient recruitment, retention, and engagement, and are finding new ways to incorporate it to transform and modernize clinical trials.
Many clinical trial sponsors have explored alternative engagement methods, including patient reimbursement and transportation support, to maintain patient participation throughout the trial period and lessen the negative impact of dropouts on data quality and trial completion. However, these measures alone have proven insufficient in ensuring sustained engagement. As a result, DCTs have emerged as a solution, particularly for chronic disease studies. By leveraging home healthcare solutions and mimicking standard disease management practices, sponsors aim to reduce the burden on patients (e.g., perceived challenges involving geographic location, financial strain, work, and family obligations) and improve participation rates.1
Additional barriers to clinical trial participation include lack of diversity and language or cultural considerations. To overcome these challenges, trial protocols need to be inclusive and consider the needs of diverse populations. Engaging with communities, recruiting clinical trial providers from within those communities, and partnering with relevant organizations can help increase awareness and participation. In addition to reducing patient burden, DCTs offer other advantages, such as sustainability, improved data collection, and a better understanding of patients’ quality of life. Good data management practices are essential to address privacy concerns and ensure access is granted only to necessary research trial staff. Optimizing clinical trial participation and minimizing patient burden requires putting patients at the core of clinical trials, considering their experiences, and designing trials with their needs in mind to offer more options and improved illness management.
An Increasing Patient Retention Problem
Alternative means of increasing patient engagement and retention in clinical trials, including home healthcare options, have long represented an acute need in clinical trial management. This results from patterns of patient behavior, particularly a decline in participation enthusiasm over time. In many cases, participants would begin participation in a trial with a high degree of energy and altruism, wanting to help and committing to frequent site visits. However, after the first few months, the circumstances of daily life would start to interfere, compounded by the burdens of participation, making weekly/frequent site visits difficult. When patients drop out at any point in a trial, all data from their involvement must be removed from the data set, which has a hugely negative impact on trial sponsors’ ability to achieve their goals.2
Trial strategists began exploring different options, like patient reimbursement and hiring taxis for travel to and from trial sites, to ensure patients remained committed throughout the entire trial period. However, those actions were not sufficient to ensure adequate retention or a decrease in the burden of participation, because the issues limiting engagement went well beyond transportation. Many trial participants care for children or elderly parents and must find alternative caretakers to take on those responsibilities, in addition to taking time off from work, when they visit investigator sites for participation in clinical trials.1
A few early adopters began exploring and implementing decentralized and remote clinical trials to optimize trial participation. PPD, the clinical research business of Thermo Fisher Scientific, was among the early advocates for such alternative approaches. In 2016, PPD oversaw a remote global rare disease study with patients visiting trial sites only four times per year for imaging and PI review (representing a 60% reduction in site visits compared with conventional trials). Over time, trial sponsors began aligning with PPD strategies to include more remote solutions within protocols, principally in trials for chronic diseases, such as chronic obstructive pulmonary disease, diabetes, and some cardiovascular diseases. For these therapeutic areas, normal standard of care involves disease management in the home, and mimicking what is done naturally avoids complications or the addition of novel trial-associated burdens for patients.
Additional Barriers to Clinical Trial Participation
Historically, trial protocols have not taken into consideration the needs of people from diverse cultures and backgrounds. Lack of understanding about clinical trials and general mistrust of the healthcare system also serve as barriers for many communities. These are complicated issues that require multifaceted solutions, including listening to and engaging with those communities to understand their concerns and expand awareness. Recruiting clinical trial providers from within those communities, as well as partnering with religious organizations and other sites where diverse populations congregate, dissolves some of the mystery around clinical trials and shines a light on the benefits of participating.
The range of DCT solutions has evolved and PPD has created a DCT ecosystem that consists of leading DCT solutions and, more specifically, home healthcare solutions, both of which play an important role in increasing clinical trial participation among more hesitant groups.
PPD is also working to modernize clinical trials to increase diversity within trial participant populations, as well as taking steps to address various issues among different groups that have made engaging with contract research organizations and trial sites difficult. We are focused on strategies to ensure trial participation and patient materials are more accessible. Additionally, PPD is adding a range of patient solutions to the DCT ecosystem, including mobile sites that offer research near patients’ homes, churches, or other community locations, to relieve patients of the additional burden of culture-related pressure associated with in-home visits.
Initial Concerns of Bringing Trials into the Community
Taking clinical trial activities out of a controlled environment and into unknown and uncontrolled spaces raises considerations regarding how to administer drugs safely outside traditional sterile areas. While enthusiasm among participants is typically easy to achieve, the biggest challenge is gaining trust, not just from sponsors, but from investigator sites as well. In fact, sponsor and site hesitancy has meant that most patients did not know that DCT home healthcare trials were an option.
During the COVID-19 pandemic, there were large numbers of patient referrals and activation of DCT solutions like home healthcare, as it became the only option for keeping trials running while sites were in lockdown. The industry embraced DCT models practically overnight. Interestingly, even after pandemic restrictions eased and investigator sites began easing back into normal operations, many patients elected to stay with the home healthcare approach.
Home Healthcare is Increasingly Important Across All Healthcare
Home healthcare, as a concept, is growing around the world, within the clinical trial space and across healthcare in general. In fact, it is likely that clinical trial sponsors and health professionals will take the lead on bringing care into the home in many areas of the world through DCTs. Once patients become accustomed to a standard of care that involves home health visits, they will expect the same when participating in clinical studies.
Adding Value in the Reduction of Patient Burden
It is essential to assess whether a patient burden exists that home healthcare can alleviate. If patients must go to the hospital or clinic monthly as part of their care regimen, a trial protocol with monthly visits would not be improved with DCT solutions. However, if trial participation required weekly visits, then home visits would eliminate three out of four trips to the hospital and significantly reduce patient burden. Ultimately, it is necessary to examine, consult, and quantify the patient burden that arises from the current standard of care treatment and determine what, if any, additional burden is created by trial participation. PPD has implemented a tool, StudyGage, which quantifies patient burden following any of the DCT Ecosystem solutions being applied, inclusive of home healthcare.
Additional Benefits of DCTs
Reduced patient burden, while an important goal, is not the only benefit of DCTs. DCT designs confer net financial benefits to the sponsor, including shorter development cycle times, lower clinical trial screen failure rates, and fewer protocol amendments.3 DCT designs also create more of a “green,” sustainable trial application due to the reduction in patient travel and paper when technology is utilized.
Furthermore, DCTs also enable improved collection of data, specifically quality-of-life measures. Rather than having to remember changes over the past month when filling out a questionnaire, patients can keep daily diaries, and that information, when coupled with telemedicine or home healthcare visits, is timelier and more accurate. The recent FDA guidance on DCTs aligns with this and encourages investigators to consider DCT solutions when an in-person meeting is not required.4
In addition, seeing patients in their home environment provides a better picture of how their quality of life is impacted by the trial. This is especially important for patients who suffer from white-coat syndrome and generally present more severe symptoms when visiting an investigator site or hospital.
Good Data Practices are Essential for Home Healthcare
Management of personal health information (PHI) continues to be a focus area with the introduction of new privacy laws and retrospective amendments of existing systems.5 One of the biggest challenges is justifying the collection and storage of PHI with respect to who should have access and how access is granted or revoked. Some PHI is needed in advance of home visits, but how long should those data be stored? Should it be kept for patients that are no longer involved in a study?
Good data management practices are critical for solving these questions. The key is ensuring that only necessary data is made available to only those who truly need the information. Now that regulators have seen successfully executed DCTs, they understand these models better. As a result, regulators are clarifying their position through robust guidelines, and this will positively impact global adoption of DCTs.4,5
Conclusion
The adoption of home healthcare solutions within DCTs has emerged as a promising strategy to enhance patient engagement and participation. DCTs leverage home healthcare to reduce the burden on patients, improve participation rates, and address patient retention challenges. Furthermore, DCTs offer advantages, such as sustainability, improved data collection, and a better understanding of patients’ quality of life. Overcoming barriers to clinical trial participation, including lack of diversity and cultural considerations, requires inclusive trial protocols that consider the needs of diverse populations. Deeper engagement with communities and relevant organizations can increase awareness and participation.
The COVID-19 pandemic accelerated the adoption of DCT models and secured trust from sponsors and sites while assuaging fears around safe drug administration and data security. DCTs not only reduce patient burden, but also offer net financial benefits to sponsors, including increased efficiency and improved data collection. The growing concept of home healthcare is becoming increasingly important across all healthcare sectors, including clinical trials, and, as patients become accustomed to receiving care at home, patients increasingly favor the reduced burden associated with DCTs. Regulators are also clarifying their position on DCTs, which will likely contribute to their global adoption.
Overall, home healthcare solutions play a crucial role in enhancing patient engagement, improving trial participation rates, and modernizing clinical trials to meet the needs of diverse populations while maintaining data quality and safety.
References
- “Quantifying DEI in clinical trials.” Accessed 18 May 2023.
- "The True Cost Of Patient Drop-outs In Clinical Trials.” mdGroup. 1 Oct. 2020.
- Tenaerts, Pamela. “Financial modeling from Tufts Center for the Study of Drug Development demonstrates substantial net benefits to sponsors who use decentralized clinical trials (DCTs) technology.” Tufts Center for the Study of Drug Development. 13 Jan. 2022.
- Decentralized Clinical Trials for Drugs, Biological Products, and Devices: Guidance for Industry, Investigators, and Other Stakeholders. S. Food and Drug Administration. May 2023.
- Recommendation Paper on Decentralised Elements in Clinical Trials. European Medicines Agency. 13 Dec. 2022.