January 17, 2024 PAO-12-23-CL-08
One of the foundational mottos at the PPD clinical research business of Thermo Fisher Scientific, is that — with respect to clinical trials — one size does not fit all. This applies to all types of studies, including decentralized clinical trials (DCTs), which present a new spectrum of possibilities requiring careful consideration to align the study design with the needs of the investigator and the patient population. Consequently, the business has amassed a tremendous depth and breadth of solutions within its PPD DCT Ecosystem to ensure the optimal fit for the needs of any study, including an array of virtual technologies that can suit various indications, designs and combinations of principal investigators and mobile sites to accommodate the diversity and cultural preferences of different patient groups.
The development of this robust ecosystem of solutions was enabled by our dedication to proactively amassing knowledge and understanding of patient, caregiver and trial site needs, while learning from opportunities made evident during the early implementation of DCTs, when data were scarce and best practices had yet to be developed. Throughout our early exploration of DCTs and their impact, we placed considerable emphasis on identifying what solutions were effective and what could and should be done differently, and then accelerating those changes where appropriate while continuing to evaluate their impacts.
Maintaining a focus on patient centricity without losing sight of the importance of the investigator is central to the PPD® DCT Ecosystem. Although the traditional clinical trial model provides a familiar pathway to achieve results, there is a crucial need to evolve beyond it for broader inclusivity. Our commitment to this evolution is evidenced through the tailored solutions within the PPD DCT Ecosystem, which offer patients the flexibility of participating from home and receiving digital alerts as a reminder to complete important trial assessments of electronic documents that can then be accessed in real time by the investigator. With the introduction of PPD Home Trial Services, such as blood draws and vital signs monitoring, patients can remain at home or work, reducing travel and associated complexities of trial participation. This comprehensive approach not only ensures a more diverse representation in trials but also significantly reduces challenges faced by patients and caregivers, bringing trials closer to communities and making participation more feasible and less daunting.
Breaking down barriers to participation and enhancing the patient and caregiver experience are paramount. The approaches to a decentralized design take into consideration the digital footprint of where important clinical input is being acquired. This could be at an investigator’s site, the patient’s home or work, or other areas where the patient may spend time. Not only does this align with our mission to foster an improved clinical trial experience, but it also benefits sponsors by streamlining the acquisition of vital trial data coming from various collection points. It is also important to note that these data streams are critically secure and aggregated for real-time review plus future analysis. These data are instrumental in driving the creation of superior healthcare products and pushing the boundaries of what is possible in the clinical trial sector.
We can envision a future where clinical trials are accessible and well understood by a broader segment of the global population. Currently, as numerous drug candidates advance through preclinical phases, the success of their transition into human trials heavily depends on effective patient enrollment. It is imperative to understand the reasons that might dissuade potential participants from joining trials. By addressing these issues, we enhance the likelihood of these groundbreaking drug candidates reaching their full potential, ensuring that innovations are not hindered by the inability to recruit and maintain participants.
Offering appropriate services in different geographies is also important. Not everyone can easily get to an investigator site. Only so many patients are within a two-hour drive of brick-and-mortar hospitals and clinics where the experts running clinical trials are located, particularly for trials investigating rare diseases. It should not be acceptable that patients dealing with life-threatening illnesses for which clinical studies are available cannot participate because the impact would be too great on their families or for other reasons related to the distance needed to travel to a site.
Finally, increasing the diversity of trial participants ensures that the drug — when fully evaluated — will be effective for a wide array of patients with different ethnic, cultural and other backgrounds. Conventional approaches to clinical studies have fallen short of achieving the optimal levels of diversity and representation to advance the industry’s goals with regard to health equity. The PPD DCT Ecosystem is designed to tackle this issue head on to ensure that the data obtained in trials are maximally representative of the disease population and that the needs of all patients are considered.
The transition from the traditional clinical trial paradigm to a DCT approach, though beneficial in many ways, comes with associated costs. These expenses include acquiring new technologies, training staff to proficiently use them and orchestrating comprehensive change management strategies. Ongoing work is needed to establish concrete proof points and to validate the spectrum of available DCT solutions.
The initial step in this transformative journey is ensuring active patient participation, which necessitates a profound understanding of the diseases with which patients grapple and the burdens they impose on individuals and their families. To participate in a clinical trial, patients must integrate trial participation into their already busy lives, which typically requires a complex balancing act. For trials targeting pediatric and geriatric patients, particularly those investigating neurological diseases, the impact of trial participation extends well beyond the patient to include their caregivers. Additionally, studies investigating rare diseases with geographically dispersed patient populations face participation difficulties rooted in the constraints of geography.
Balancing the demands of everyday life is challenging — and considerably more so for individuals living with chronic diseases. Adding the commitments of a clinical trial to their regimen can amplify these challenges. Recognizing this, the PPD DCT Ecosystem is meticulously crafted to support and simplify patient and caregiver journeys. Through user-friendly mobile apps, patients can more easily incorporate trial-related tasks into their daily routines, such as filling out digital questionnaires/eDiaries at home or work, or having home healthcare further ease the process, offering onsite blood draws and vital signs monitoring, eliminating frequent travel to trial sites. The integration of telehealth services and wearable technologies enables remote interaction with the investigator and continuous data collection. This enables patients to participate without disrupting their routine while also assisting in completing the trial on time. With these patient-centric tools and services, we ensure individuals can embrace clinical trials with minimal disruption, maximizing the therapeutic benefits while maintaining their life's equilibrium.
Trials implemented within the comprehensive PPD DCT Ecosystem benefit from the synergies that result from integrating an array of best-in-class digital solutions and clinical services with thoughtfully crafted, patient-centric protocol designs. The critical importance is providing patients with options for participation; the when is already decided, so giving them some freedom to choose can improve the recruitment and retention of patients within the trial, ultimately keeping trials on track for completion.
The availability of home healthcare — including blood draws, vital signs monitoring, eDiary reviews, adverse event assessments, etc. — significantly elevates the efficiency of DCTs. Incorporation of digital or clinical functions into the clinical trial framework eliminates the need for participants to physically visit a trial site and reduces the level of effort required to participate. Crucially, these measures not only provide convenient alternatives for participants, they also safeguard the integrity of the trial and the fidelity of the data collected, enabling the trial to remain robust and reliable.
In any clinical trial, solid data management is paramount, as the data generated drive the conclusions of the study. DCTs create new challenges with regard to effective data management, as a substantial portion of data must be collected outside of traditional investigator sites.
Compliance with best practices and regulations is essential. This involves implementing a critical cybersecurity framework, establishing an appropriate infrastructure backbone and providing thorough oversight of the cloud technology that hosts the relevant data lakes. Ensuring the security and anonymity of patient data is crucial, and stringent de-identification protocols must be implemented to shield patient/trial information while preserving the utility of the data.
As data collection methods in clinical trials continue to evolve, the approaches used to manage those data must adapt accordingly. Data can take various forms — from handwritten notes transcribed and scanned into digital systems, to real-time clinician entries or patient inputs via tablets, smartphones or PCs. All these data must be captured and translated to ultimately inform sponsors' decisions concerning the safety and efficacy of candidate drugs. This underscores the critical nature of efficient data flow, capture and exchange mechanisms. Furthermore, the advent of novel data capture technologies, such as wearables, requires further innovations to ensure the security and anonymity of the data collected. We are also proactively addressing challenges posed by data silos within healthcare, which can significantly impact the qualification and quantification of who can participate in what type of study.
In response to the COVID-19 pandemic, global regulatory agencies more readily embraced DCTs, acknowledging their potential to transform the clinical trial landscape. Recent guidance documents issued by the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) reflect this shift in perspective, offering further support for the adoption of DCT solutions and supporting new solutions that can increase participation and diversity.
Our business has adopted a holistic approach to effectively navigate this evolving terrain. Considerations span from the point of patient entry into a clinical trial and the consent process, whether electronic or paper-based, to the intricacies of global and state-level regulatory requirements regarding home healthcare and mobile units. Each protocol is carefully tailored to align with the patient population, indication and study goals.
DCTs can change both the frequency and nature of visits to investigator sites relative to conventional trial models. In some cases, these changes may be perceived negatively by investigators, owing to concerns about potential revenue loss. As such, through our DCT Network, we support principal investigators (PIs) and sites on their DCT change management journey and remain engaged in ongoing dialogue to provide comprehensive communication and education to enable investigators to adapt to the evolving landscape, capitalizing on the reduced visit frequency for individual trials. Ultimately, our goal is to mitigate these concerns by enabling investigators to participate in a more extensive array of trials, which could lead to an increase to the individual investigator’s revenue.
To enhance transparency and understanding, it is essential to quantitatively and qualitatively assess the influence of DCTs on all aspects of clinical trial management. This comprehensive evaluation encompasses the assessment of specific tools and solutions, as well as the effect of DCTs on investigator site visit frequency, type, and associated expenditures. We have developed a patient burden assessment tool, StudyGage, which aids in quantifying this metric for a given study while evaluating the impacts of specific changes. We are actively pursuing the development of similar tools to assessing considerations like costs and diversity to obtain both a high-level overview of the value that DCT tools offer and a granular understanding of their critical contributions to individual trials, both presently and in the future.
The road ahead offers an ever-growing array of opportunities to further enhance the impact of DCTs, but much work remains to be done, particularly to increase patient awareness of these innovative approaches. The success of clinical studies overall hinges on patient participation, and without their awareness, engagement will remain elusive. We are addressing this challenge with a keen focus on non-commercial approaches that prioritize the needs of people living with specific diseases. That includes harnessing the power of existing data lakes, breaking down data silos, democratizing access to information and applying advanced data-mining techniques to identify individuals who align with specific trials and stand to gain the most from participation.
Looking to the future, we anticipate several key trends that will drive the further evolution of DCTs. Telehealth is poised to play an increasingly substantial role, expanding its footprint in these trials. The integration of digital biomarkers through smartwatches and wearables is expected to surge, offering increasingly robust and convenient data collection methods. Moreover, we predict that simplified and easily deployable devices for appropriate clinical monitoring in therapeutic areas like cardiovascular disease will soon be made available for use in clinical trials. Near-patient solutions are evolving with a focus not just on home healthcare but also on participation in health clinics, mobile research sites and direct-to-patient services. These services can provide devices that patients or caregivers can use to extract microsamples of blood, which can then be shipped to labs for analysis in trials.
Further efforts will aim to leverage electronic devices and data to streamline interactions between physicians and patients, in part to eliminate the need for extensive retraining each time individuals participate in new trials or transition between trial phases. Additionally, simplifying data entry for home collection of eSource data while ensuring precise data management and facilitating data sharing between healthcare and clinical trial systems will become increasingly important. Lastly, continuous education of investigators and their teams in effectively navigating the clinical trial landscape using DCT solutions will remain an essential aspect of our commitment to advancing increasingly patient-centric trials.
With a decade of experience running DCTs and the deeper understanding gained over the past four to five years — coupled with our integration into Thermo Fisher Scientific's expansive resources and capabilities — the PPD® clinical research business is uniquely positioned to drive progress at an accelerated pace, offering innovative solutions that overcome challenges. Notably, we have devised solutions aimed at enhancing medication adherence and launched sustainability initiatives.
The PPD DCT Ecosystem is further fortified by our exceptional employees and carefully selected partners, who form the foundation of our clinical trial operations. We are committed to continuously enhancing our capabilities, by establishing PPD Home Trial Services, for example, which ensures that we maintain complete oversight and management control, encompassing the hiring of healthcare providers and project and country-level coordination.
We will continue our commitment to investing in partnerships and external solutions, maintaining a focus on dismantling barriers to participation and simplifying the clinical trial journey for patients, caregivers and families. Our partnerships are grounded in trusted relationships and a shared understanding of our mission to seamlessly deliver technical solutions and clinical services. Our business maintains vigilant oversight of these partnerships to ensure consistent operations and the realization of goals, spearheaded by its decentralized solutions director. In tandem, our digital implementation managers oversee each step in the integration of DCT solutions within clinical trials — whether that involves electronic consent, electronic clinical outcome assessments, telehealth or wearable technologies.
As clinical research continues to evolve and modernize, the PPD DCT Ecosystem was thoughtfully developed to fit not only the present landscape of DCT models but the developments to come. The term “ecosystem” reflects not only its complexity but its dynamics; rather than a static framework, the DCT Ecosystem is a living, breathing entity that thrives on innovation and adaptability.
The PPD clinical research business is fully committed to this dynamism, which is central to driving further advances in clinical studies. We will continuously introduce novel tools and services while refining our existing capabilities. Importantly, we want to continue to develop innovations to furnish additional metrics that assess study performance and gauge our impact on reducing patient burdens, optimizing cost-efficiency and fostering diversity and inclusivity within clinical trials.
Our aspiration is not just to make incremental changes but to propel transformative changes on behalf of sponsors, investigators, patients and the communities we serve. We are driven by a vision of a clinical trial landscape that transcends convention, placing patients firmly at the forefront of progress and forging a brighter, more inclusive future for all.
The PPD DCT Ecosystem offers comprehensive solutions covering most facets of clinical trial management, from initial recruitment to trial closeout. By mitigating the most pressing barriers to clinical trial participation, we accelerate the enrollment process and enhance participant retention rates, which further translates into expedited drug development, shortened time to market, greater patient impact and superior investment outcomes.
Richie Pfeiffer is the Senior Director of DCT Strategy and Innovation within the Digital and Decentralized Solutions team at PPD, the clinical research business of Thermo Fisher Scientific. He has over 25 years of experience in healthcare, data/technology and life sciences. He is a board-certified nuclear medicine technologist with an advanced degree and experience in health informatics. He also has experience with phase I-IV clinical trials focused on DCTs as well as standard and novel clinical and digital solutions and services. Notably in his career, he brought a beta amyloid radiopharmaceutical to market globally.
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