The role of technology in executing decentralized clinical trials
At the start of the COVID-19 pandemic, the world turned to technology for novel solutions to an at-the-time novel virus. In the clinical trial space, these increasingly open mindsets accelerated the acceptance and adoption of decentralized clinical trial (DCT) strategies and technologies — a silver lining that came at high cost.
The potential benefits offered by DCTs have broad application. For the 4% of the global population diagnosed with one of 7,000 different rare diseases, however, the impact is amplified. Rare and orphan diseases can include genetic, environmental, and infectious illnesses, in addition to rare cancers. The technological advantages to DCTs can reduce barriers to participation, ease hurdles to access, and strengthen retention throughout a study’s duration.
Importantly and fundamentally, DCTs harness technological advancements that help bridge the myriad gaps between patients and researchers, and the treatments aimed at meeting unmet medical needs. This article will explore how technology and DCTs close those gaps in terms of geography, study design, and the particular physical, financial, emotional, and other challenges that make rare disease trials uniquely complex.
How DCT solutions improve access and retention for rare disease patients
Rare diseases can often present a spectrum of neurological, behavioral, and physiological symptoms, all of which can range from mild to severe. This variation may even exist within patient groups of the same rare disease diagnosis. Designing a successful rare disease trial entails planning how patients will be able to participate in the trial, including traveling to the clinical site and suitable accommodations. Some symptoms of rare diseases make it complicated and burdensome for patients to travel, necessitating specialized arrangements and equipment, such as long-distance impulse sensitive ambulances with oxygen tanks.
Pediatric patients or those with developed symptoms usually require a caregiver during the trips to trial sites. The caregiver—who may be a family member, hired nurse, or another individual—should make the necessary arrangements to attend appointments, which may include requesting time off from work or arranging care for other possible dependents. The duties and obligations of a caregiver can present considerable logistical, financial, and emotional burdens. Fortunately, stress and responsibilities related to caregiving can also be reduced with the technological solutions inherent to DCTs.
Instead of requiring patients to undertake the arduous journey to clinical sites, DCTs can implement telemedicine visits for screenings and study visits, thereby reducing a substantial portion of burdensome travel. Two-way, opt-in policies and Informed Consent Form (eConsent) technology enables patients and/or family members to give informed consent via a simple downloadable app. DCTs grant patients and caregivers more freedom throughout the trial. When they can start the screening process in advance from the comfort and convenience of their own homes, patients feel more respected, involved, empowered, and in control.
Virtual sites, telemedicine, and home health systems and solutions
Virtual clinical sites are valuable because they reduce or eliminate the need to open a stand-alone clinical trial site near individual rare disease trial participants, which is often necessary when patients are in rural areas. While not ideal for all cases, virtual sites support patient-facing activities with technology like Remote Patient Monitoring (RPM) solutions and Direct-to-Patient Investigational Product (IP) shipments. Resources that would have otherwise been spent on establishing and operating physical trial sites can be reallocated to improve the overall trial and patient outcomes, especially by incorporating more ways to enhance the experience of patients and family members.
Sponsors can also utilize a variety of telemedicine and home health solutions when designing and conducting rare disease DCTs, ensuring a patient-centric approach that supports family members and caregivers. Telemedicine, whether via computers or mobile devices, enables continuous, close communication and instant data sharing, which are essential to maintain study integrity and achieve successful research results. Sponsors can coordinate cohesive and comprehensive care among the entire medical team (including the investigator and standard of care provider). Additionally, practitioners can schedule and hold appointments with patients, family members, and caregivers, and even obtain samples and biopsies without traveling long distances.
Home health not only reduces travel and financial burdens, but it enables patients to engage with the same healthcare workers throughout the study. This provides patients and family members with an important foundation on which to build a trusting and reliable relationship. A trusted nurse can provide emotional support, counseling, and coaching, in addition to helping navigate trial protocols and gathering health data—all crucial factors in creating a positive trial experience and deeper connection to the study for patients and family members.
Furthermore, patients and caregivers are more likely to be candid about symptoms or side effects with practitioners they trust, resulting in more accurate study data. In turn, healthcare professionals can react quickly and find solutions in case any issues arise, particularly while using real-time RPM technology.
Real-time data collection and care coordination in clinical trials
RPM solutions, such as Electric Patient Reported Outcome (ePRO), eDiaries, and wearable technology help gather important health data at more optimal intervals, more efficiently, and with greater accuracy compared to traditional methods. Chosen technology assists rare disease DCT sponsors in maintaining the exacting data recording and monitoring process. The data gathered and stored can shine new light on factors and situations in a rare disease patient’s life which illustrate more authentic real-world scenarios and associated evidence.
ePROs and eDiaries offer an effective alternative to the traditional paper questionnaires and booklets previously employed to gather data from patients and caregivers during trials. If paper questionnaires are damaged or misplaced over the course of a study, significant data could be excluded from the analysis, possibly skewing the results, implications, and future developments. As an alternative, patients with rare diseases who are part of DCTs and their caregivers can use their personal devices (via downloadable software) and/or sponsor-provided devices to record important data (e.g., received treatments, vital signs, physical examinations, sleep patterns, and emotional and mental well-being). Some software or hardware can also remind patients and caregivers to administer scheduled treatments as well as input the proper data in a timely fashion.
To further support patients with rare diseases and their care team during DCTs, sponsors are strongly encouraged to consider incorporating sensors and wearable technology in clinical studies. Solutions that automatically measure and record the relevant data reduce the burden on patients and caregivers since they are not required to manually assess and record health data. The health data can be directly transferred to a clinical site or team in near real-time and will be more accurate due to minimized human error.
Since the data can be received and read almost immediately, the medical team can react quickly to concerning incidents and determine appropriate solutions. Additionally, anomalies (such as a recorded high heart rate due to exercise or an exciting event) can also be noted and disregarded in the data pool, ensuring a robust fact-based data analysis with minimized outliers.
With RPM, telemedicine, and more, study plan designs must factor in the different types of biometrics, imaging, software, hardware, and other technology that will be integrated into the trial. Sponsors must remain mindful during the study planning process that all these components operate in sync; for example, home health and RPM, as intended within the trial protocol and ICF. Because DCT technology and traditional clinical research methods are increasingly being used together in hybrid trial models, it is advantageous for sponsors who are interested in implementing DCT strategies to consult with experienced CROs and vendor partners.
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Flexibility from decentralized trials drives patient trust and diversity
A final factor is not a technology itself, but it can be fortified by technology and the ways in which technology is activated in DCTs. That factor is trust: It is essential to any trial and without it, clinical development would certainly come to a standstill.
Rare diseases are present globally, therefore the cultures and healthcare systems that patients and practitioners experience—and that sponsors must plan for—can vary greatly. Healthcare systems may be more centralized or decentralized, with actors such as social security and insurance organizations also taking on different roles according to the region. Some healthcare systems are extremely complicated, and DCT designs can simplify orchestrating and conducting clinical research, increasing accessibility, and making it easier to carry out rare disease trials on a global scale. Such simplifications can inspire patient trust.
Regardless of the culture and the healthcare system, there must be a foundation of trust among patients, their family members, and healthcare practitioners (such as home health nurses or sponsors). Building this foundation hinges on understanding and accounting for the needs, expectations, and resources of everyone involved.
For rare disease patients and their family members, feeling that they’re heard and adequately involved in the trial is a fundamental component to establishing trust. In addition to being acknowledged, patients and their loved ones may consider it important that certain courses of action are taken as well. The more flexible a study is, the easier it is for healthcare providers to accommodate the patients’ and family members’ wishes. Rare disease studies designed based on DCT principles supply the flexibility needed to solve challenges, reduce burdens, and cultivate important long-term relationships based on trust.
Conclusion
Decentralized trial designs are well suited to rare disease clinical development and can reduce the numerous burdens rare disease patients, family members, and caregivers face. DCT strategies provide substantial benefits in the form of flexible hybrid model study protocols, which are crucial to achieving patient enrollment diversity goals. Solutions such as telemedicine, home health, RPM, and wearable technology enable clinical researchers to include people with rare disease regardless of their location.
The pandemic brought many of these technologies to the forefront of clinical development and bolstered the creation of innovative, market-shifting solutions. DCT strategies and technology are now firmly established in the clinical development sphere and will continue to enable and advance rare disease research.
Every study is unique. If you have questions about decentralized trial strategies, you can contact us, or you can learn more about Precision’s approach to decentralize clinical trials.
