Virtual Clinical Trials have the potential to drive significant change in the way we conduct clinical research by taking full advantage of digital technologies – for the benefit of patients and the pharma industry as a whole.
John Robert Zibert, LEO Innovation Lab
Zarqa Ali, Department of Dermatology, Bispebjerg Hospital
Simon Francis Thomsen, Department of Biomedical Sciences, University of Copenhagen
Clinical drug development is a time-consuming and complex process that may take up to 15 years. The cost of developing a new drug, from research and development to marketing approval, is an estimated USD 2.6 billion. Approximately 85% of therapies fail through early clinical development, and only half of those reaching phase 3 are approved.
Over two-thirds of the total cost, both money and time, of the discovery and development of a new drug is embedded in the clinical-testing phase.
Virtual Clinical Trials (VCTs), also called remote or decentralized trials, are a relatively new and yet underutilized method of conducting clinical research taking full advantage of technologies such as apps, electronic monitoring devices, and online social engagement platforms.
Making correct diagnoses based on photographs and patient symptomatology has always been part of the dermatologist’s routine. The visual nature of dermatological conditions, the relative ease in evaluating skin diseases remotely, and the fact that skin diseases often are not life threatening and rarely require complex examinations make VCTs very attractive for dermatological research. Thus, VCTs are in many ways made for dermatology.
Patient Recruitment – a Tremendous Expense within Traditional Clinical Trials
Patient recruitment is the single biggest cause of clinical trial delays. Furthermore, 30% of phase 3 study terminations are due to enrollment difficulties. Approximately 80% of trials fail to meet the initial enrollment targets and timelines. These delays can result in up to USD 8 million per day in lost revenue potential for pharmaceutical companies.
VCTs may overcome the challenges faced in conventional clinical trials where recruitment takes time and hence costs a lot of money due to limited reach of suitable participants.
In traditional clinical trials participants are recruited through hospital visits, medical clinics, or using media such as newspaper/radio/television ads. The reach and ability to participate are often limited by patients’ geography.
In VCTs a combination of traditional patient recruitment and digital recruitment is used. The combination of recruitment strategies leads to more rapidly reaching the enrollment target resulting in faster initiation of the study. When happening digitally, patients are targeted directly via web-based platforms, such as search engines and social media. This approach allows for fast recruitment of patients without geographical limitations, reaching eligible trial participants worldwide.
A broader digital recruitment strategy, targeting participants across geographical locations results in a more diverse group of study participants. This more diverse pool of patients would mean a higher degree of representation of the real-world than what can be obtained in traditional clinical trials.
Trial Participation Accessibility leads to Lower Dropout rates
Participation in VCTs is more easily accessible to participants who otherwise would be excluded from or reluctant to participate in a traditional clinical trial.
VCTs are patient centered, meaning that they are based from the patient’s home with the patient’s own smartphone. This approach increases convenience for the participant and allows those with mobility issues i.e. the elderly and disabled can participate in VCTs without the challenges associated with traveling. Accessibility for the working generations is also made easier, since patients do not have to take time off work to participate in a trial because of the remote setup.
When simplifying and making study participation easily accessible, the chances that people comply better with trial instructions increase. In addition, dropout rates are experienced to be lower than in traditional clinical trials, because participation is made easy for the patients.
A Limited Number of Study Sites
A limited number of study sites are involved in VCTs. The aim is ideally to have only a single site, or a concept of one site in each country in global studies, led by a principal investigator. A sub-investigator team will ensure the health and safety of the participants via remote visits where patients contribute with data virtually via their smartphones.
The minimal number of study sites in a VCT stands in contrast to traditional clinical trials that involve many study sites and study teams. The more extended setup in a traditional clinical trial contributes to increased expenses.
In addition, data collection within VCTs is available from multiple mobile sources such as smartphones, apps, watches, electronic patient reported outcomes, and e-diaries. In traditional clinical trials, data collection is limited to the findings made during physical visits.
VCTs may save Pharma Companies Time and Money
Savings are associated with VCTs and hence VCTs may influence the bottom line of pharma companies positively. The time spent conducting a study is reduced due to shorter enrollment periods, faster study initiation, lower dropout rates, and faster data collection.
The cost of managing a VCT study may be lower than conventional studies due to the remote visit setup. Any reduction in the number of operational study sites offers considerable capital savings. In addition, the need to reimburse patients for travel expenses decreases when less physical visits are required.
Limitations of Virtual Clinical Trials
Despite the many benefits in potential cost reduction, online recruitment and site simplification, there are some challenges to conducting VCTs that must be taken into consideration.
Because VCTs are a relatively new way of conducting clinical trials, the regulatory approval framework of VCTs is still in its early phase, and this makes the guidance in the field unclear.
In addition, the data protection aspect of the virtual approach to clinical trials needs to be addressed. Concerns about transferring large amounts of sensitive health data over the internet is a challenge. However, proper implementation of technologies and defense strategies like storing anonymized data on external web servers secured by ID and passwords, using secure emails, and web servers hosted by trusted providers will minimize this risk to an acceptable level.
While in general the reach of digital recruitment is broad, one limiting aspect is that it may not reach the elderly generation as efficiently as the younger generations who use digital technology and social media daily.
For this same target group of seniors, the lack of human interaction in the recruitment process can also be a barrier, because they require the establishment of trust and a personal relationship to become involved in a trial.
It should also be mentioned that some areas of clinical research are not ready for remote monitoring, and the virtual trial design is not advanced enough to attempt phase 1 studies where patients need to be closely observed and located near a clinical site in case there is a reaction.
While VCTs are very suitable for dermatology, they can be less suitable for certain diseases that require sophisticated or in-hospital monitoring. Acute life-threatening diseases, such as strokes, are possibly not appropriate for a fully virtual trial design. Nonetheless, the majority of clinical trials could benefit from incorporating a partly virtual trial design, defined as a hybrid trial design, which enables “the best from both worlds”: increased use of digital data collection as well as physical site visits.
So why are VCTs the Future of Clinical Trials?
VCTs are still a relatively new way of conducting clinical trials, which means that some challenges are still implied. However the positive aspects for both patients and the pharma industry show a huge potential in the simplification and rapid completion of studies. According to Alia, Zibert and Francis’ review article there is a great potential to save time and money while reaching a broader patient pool in VCTs.
VCTs have been used in phase 2–4 trials showing promising results. Studies to date have demonstrated that VCTs are not only operationally feasible, but also successful.
VCTs show high recruitment rates, have better compliance, lower drop-out rates, and are conducted faster than conventional clinical trials. Hence, VCTs meet the goal of the pharma industry in conducting low-risk, high-return trials that result in the ability to bring new treatments to patients faster. Treatments that will improve the conditions of patients across the globe.
We predict that many more of these virtual trials will be seen in the future, particularly within dermatology.