Decentralized and Virtual Clinical Trials
Josipa Ljubicic, Chief Executive Officer, Proqlea Ltd
Decentralized and virtual clinical trials, with pharmacies as collaborators, mark a significant transformation in medical research methodologies, utilizing digital tools for remote trial management. They overcome traditional obstacles such as geographical limitations and promote patient engagement through remote monitoring and telemedicine, with pharmacies managing medication dispensing and adherence. By dispersing trial operations, these trials streamline procedures, cut costs, and boost efficiency, ultimately expediting medical breakthroughs and broadening access to experimental therapies. Integration of telemedicine improves patient access and participation, ultimately accelerating medical innovation and the development of experimental therapies.
Introduction
According to the National Centre for Biotechnology Information, 30% of participants drop out during clinical trials, causing harm to the study. This can be prevented by solutions involving virtual clinical trials. [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3684189/]
Drop out can be prevented by implementation of decentralized and virtual clinical trials.
The traditional approach to clinical trials requires a clinical study site, which is usually a hospital, where participants are recruited, experimental drugs are administered, interviews with patients are conducted, data is managed, and documents are signed. Some (or all) of these processes can be done remotely using technology. Such clinical trials are called decentralized or virtual clinical trials.
Imagine this situation:
An elderly patient volunteer lives several hours' drive away from the study site. Instead of spending time and energy traveling there, they sign up for a virtual telemedicine system and talk to their doctor online about their experience with the new medicine. They monitor some symptoms themselves with devices they have on hand, then fill out forms and surveys by themselves, without waiting in a long line. The medication is available in their local pharmacy, where it has been delivered from the site.
The patient has now saved time, money, and labour of travel, while the study site has gained all information needed, using minimal staff.
Virtual and decentralized clinical trials have the potential to contribute to clinical research in several ways: overcoming geographical limitations, increasing patient engagement, streamlining procedures, cutting costs, saving money... and ultimately boosting efficiency and aiding in reaching medical breakthroughs, all with the help of technological innovations, good network connectivity, and a more accepting attitude towards technology.
Challenges of Traditional Clinical Trials
For a clinical trial to be of high quality, diversity in patients are needed. Potential patients are unlikely to be located in the same area, and as such CT’s face geographical limitations. Patients who must travel several hours to the clinical site, have to arrive at a specific time, and then have to spend several hours there as well, may not join the Clinical Trial due to stress or burden of travel, they may drop out of it, or they may not be as compliant due to dissatisfaction. Since clinical trials should be conducted for the patient, i.e., the patient should be the focus, this creates difficulties in the quality of research.
In CT’s, patient engagement issues are typically lack of awareness, limited access to information and resources, patient burden and inconvenience and lack of diversity and inclusion. Potential patients will never participate in clinical trials if they are not aware of them. Some patients are not sufficiently informed about the trial process and their rights, so they are not interested. Furthermore, if patients want to inform themselves, they may not have access to disease and medication data, and current study status due to legal regulations or data privacy regulations. If patients agree to participate in the study, they bear a certain burden, such as the waste of time and burden of travel mentioned earlier, and complex treatment procedures. Finally, individuals from marginalized communities often do not participate in clinical trials, so the drug is not tested for all groups because it is often not easily accessible to them (e.g., patients over 65 years old, patients with disabilities, people of colour).
Clinical trials sometimes face operational inefficiencies. Sometimes processes within clinical trials are not optimized for productivity, cost reduction, time efficiency, and quality of collected data. Common challenges include participation retention, when patients do not follow up after the trial, resulting in a lack of staff and resources, and problems in data management when multiple incompatible data storage systems are used.
Overview and benefits of decentralized and virtual Clinical Trials
The concept of decentralized or virtual clinical trials is not new, but it was developed further after the global COVID-19 pandemic. Decentralized and Virtual Clinical Trials involve the use of digital health technology to enable remote participation by patients. Decentralized systems do use a central site, which can be confusing due to the name, but not all processes are conducted at the same site; they may occur at other local smaller sites or remotely from home. A virtual clinical trial would be a type of decentralized clinical trial that is fully remote and online. In practice, most clinical trials operate using a hybrid model, incorporating remote technology for some aspects of the trials
Role of digital tools in remote trial management
The key word here is technology. Various software and hardware solutions can replace or enhance some processes of clinical trials, showcasing benefits of decentralized and virtual clinical trials.
1) Overcoming geographical limitations
In theory, a patient should only need to visit the clinical site once to meet with the doctor and understand the procedure. After that, other obligations, such as receiving medication, completing questionnaires, or consulting with the doctor, can be done remotely from their home, at a local site much closer to them, with the help of nearby pharmacies.
2) Enhancing patient engagement through remote monitoring and telemedicine
Today, remote monitoring and telemedicine encompass much more than just talking to patients via webcam. Modern systems are used not only for communication but also for collecting data. They are adaptable for both patients and professionals and can independently measure symptoms such as high blood pressure, diabetes, weight loss or gain, heart conditions, chronic obstructive pulmonary disease, sleep apnea, and asthma. Example of technology used for remote monitoring is weight scales, pulse oximeters, blood glucose meters, and blood pressure monitors. With today's global connectivity, widespread use of technology, and falling prices, technology is more accessible and affordable than ever before.
3) Improving patient access and participation
Patients can independently educate themselves about their rights and the trial process using a digital platform containing all necessary information and resources. After being informed, the investigator verifies whether the patient agrees with everything. This allows the patient time and agency to make decisions independently, without external influence, and frees up the investigator's time for other processes.
All this leads to streamlining of procedures and cutting costs, boosting efficiency and expediting medical breakthroughs.
Importance of pharmacies in the process
Pharmacies can recruit diverse and local patients by reaching out to them directly. They hold real-world evidence data which are useful. Some companies have the finance to use advanced machine learning algorithms to design betters’ studies.
For patients who are in the process of clinical research, pharmacies can offer direct-to-patient services, with shipments of drugs or medical devices in smart packaging directly to patients.
Instead of the patient traveling to the clinical centre where the study is conducted, the medication is delivered to a local pharmacy where everything is familiar to the patient and they can pick up the medication without traveling and waiting for a long time.
Case Studies or Examples
Here are some real-world examples of successful decentralized and virtual clinical trials
The Michael J. Fox Foundation Virtual Study
This study enrolled individuals with and without Parkinson's disease using the Fox Trial Finder, a clinical trial matching tool. More than 160 participants from 39 sites across the country took part in the study. Since Parkinson's disease is typically diagnosed visually, the virtual platform allowed investigators to remotely examine participants via videoconferencing, eliminating the need for travel. This approach provided wide geographic representation and included individuals who otherwise wouldn't have been able to participate. A follow-up evaluation showed high participant satisfaction, with 90% expressing satisfaction with the trial, 80% indicating a willingness to join a similar future trial, and 85% stating they would be more able to participate if it could be done remotely.
AT-HOME PD
This study will conduct follow-ups with participants from two large, multicenter Phase 3 Parkinson's disease studies (Steady PD III and Sure PD3) via annual virtual visits using web-based video conferencing. Participants in the AT-HOME PD study will also provide self-reported outcomes quarterly through Fox Insight, an online clinical study platform, and submit monthly assessment data on tremor, gait, voice, and balance collected via a smartphone.
23andMe LRRK2 Study
This study aims to investigate the linkage between the leucine-rich repeated kinase (LRRK2) genetic mutation and Parkinson's disease by recruiting a national cohort of carriers. Due to the rarity of the LRRK2 mutation, a traditional study would necessitate multiple sites worldwide. Instead, leveraging the Fox Insight online platform, the study successfully recruited 300 participants, including 50 with Parkinson's disease. Participants will be followed remotely with annual virtual Parkinson's disease examinations.
Future Implications and Conclusion
Virtual trials present several advantages over traditional studies, notably in their participant-centered approach. The geographic reach of virtual trials is not constrained by the location of participants but by their internet access. These trials offer additional benefits such as comfort, convenience, and confidentiality for participants. They also streamline the initiation process, enable more frequent participant assessments, and simplify the management of multiple Institutional Review Boards (IRBs).
Case studies indicate that virtual trials could significantly reduce costs in the long term. Despite the availability of tools to conduct virtual trials, the primary barriers to their widespread adoption appear to be a lack of creativity and determination within the industry. By overcoming these challenges, virtual trials have the potential to revolutionize the field of clinical research.
Author Bio
Josipa has over 20 years of experience in the pharmaceutical industry and is an expert in QA (Quality Assurance). She has helped more than 150 companies establish effective QMS and has hosted over 320 audits and regulatory inspections. Her expertise extends to clinical trials, focusing on innovative products for rare diseases. Josipa has advised leading pharmaceutical companies throughout the clinical trial process. Under her leadership, Proqlea became a full-service CRO, promoting high-quality products. She is currently working on establishing the first clinical trial hub for the EU and Balkan region.
