The site-less model allows patients to accomplish study visits from the comfort of their own home.
The Food & Drug Administration (FDA) is charged with overseeing the drug development process in order to assure that medicines produced and released to the public by the pharmaceutical industry are both safe and efficacious.
Excessive costs involved in developing and gaining regulatory approval for new drugs,1 however, have resulted in the industry struggling to find ways to reduce expenses and speed timelines, while maintaining this mandate.
Phase III clinical trials are typically responsible for the vast majority of an approved drug’s development costs,2 and study startup (SSU) activities at the outset of trials remain a perpetual bottleneck. This has led to increased scrutiny of the investigative site selection and activation processes for clinical trials.
Patient recruitment and retention are significant contributors to excessive timelines and costs at investigative sites. Estimates have indicated that patient recruitment efforts are potentially responsible for up to 30 percent of the clinical product development lifecycle, while some sites end up not even enrolling a single patient.3
Patient dropout is another issue, with an astounding 85 percent of all clinical trials failing to retain enough subjects to successfully complete a study.4
mHealth Technologies and the Site-less Clinical Trial Model
The emergence of disruptive mobile health (mHealth) technologies like Fitbit, Apple Watch, Telcare, and Scanadu is fueling an industry push towards a more patient-centered approach in clinical trials.
This approach (also known as site-less trials, remote trials and virtual trials) utilizes mHealth devices to allow patients to transmit remote, real-time data (e.g., sleep duration and quality, blood pressure, blood sugar, activity levels, heart rate, etc.) directly to the central study site. Study volunteers communicate with study personnel remotely via technology and receive their medications in the mail.
The site-less model allows patients to accomplish study visits from the comfort of their own homes, while effectively eliminating the need for site selection and activation activities on multiple sites. In the process, study sponsors save significant amounts of time and money, while dramatically reducing trial participation burden on patients.
In addition to mHealth and telehealth technologies, site-less clinical trials may also rely on home visits to patients by skilled nursing resources. This eliminates the burden of a site visit and may improve overall patient retention.
There is significant precedent for home visits in clinical trials, particularly for rare diseases or other serious indications which leave the patients home-bound. Home visits have been leveraged successfully over the last few years to avoid low recruitment or high drop-out rates5.
A recent study on rare disease patients published in Neurology reported that over half (54%) indicated that home-based research visits would increase the likelihood of clinical trial participation6.
With the rise of site-less trials and the confluence of factors contributing to high costs and slow timelines – Are clinical research sites a dying paradigm?
Site-less Technology Contract Research Organizations (CROs)
An interesting development in site-less clinical trials is the concept of the site-less technology contract research organization (CRO). Center Point Clinical Services launched the world’s first CRO specializing in site-less clinical trials in 2016.7
According to CEO Joe Martinez, “The site-less CRO model enables clinical research to be conducted at an unprecedented scale and scope. Study sponsors are able to reach and maintain excellent communication with trial participants, wherever they are, as well as secure and retain reliable data that can be instantly accessed and shared.”10
While Center Point is currently the only CRO focusing exclusively on site-less clinical trials, there is evidence that other CROs are beginning to move in this direction. Earlier this year, Parexel launched a patient sensor solution that will allow study data collection remotely using wearables and sensors.8
Benefits of the Site-less Technology Model
The site-less technology CRO model for clinical trials has a number of benefits including:
Provides a More Diverse and Representative Patient Population. When clinical trial models are built around geographical investigative sites, a large number of potential study volunteers are eliminated simply because they do not have convenient access to these sites – elderly patients with mobility issues, patients who live in rural areas, patients who are very sick, etc. The site-less model opens the trial up to a much wider – and therefore potentially more diverse and representative – patient population.9
Speeds the Recruiting Process. By significantly increasing the number of potential study volunteers, the site-less model can dramatically speed up the trial recruiting process, and ultimately shorten the duration of the clinical trial itself.11
Improves Patient Compliance and Retention. Poor patient medication compliance and retention are a problem in many traditional studies. Frequent communication between study volunteers and study personnel via technology in a site-less trial can improve both patient compliance and retention significantly.
More Cost Effective. The lack of multiple brick and mortar study sites, along with improved patient recruitment and retention, saves sponsors significant amounts of money.
Improves Ability to Achieve Regulatory and Commercialization Goals. Use of mHealth technologies with continuous monitoring increases data volume, while improved patient compliance leads to more reliable data. Additionally, the centralization of the study site in site-less trials leads to a reduction in record-keeping inaccuracies and omissions, and adverse events and safety reporting failure. Ultimately, the improvement in data reliability and record-keeping helps sponsors to reduce cost and better achieve regulatory and commercialization goals.12
Challenges of the Site-less Technology CRO Model
There are a number of challenges inherent in the site-less CRO model:
May not work for Complex Trials. The site-less model is more difficult to implement for trials that require complex study visit procedures, or those that can only be conducted by expert medical specialists using technical equipment.
Data Security. Given that large volumes of sensitive patient data will be transmitted over the internet, data security is a concern that must be addressed. Patient recruitment and retention can also be affected by data security concerns.10
Difficulty Attracting Older Patients. The site-less model is best suited to younger patients who are familiar and comfortable with remote communication and technology, while patients over 60 may be reluctant to participate due to a lack of in-person support in the trial. Additionally, patient recruitment through social media will likely not be very effective for people over 60.11
The site-less clinical trial model provides a number of significant benefits to study volunteers that serve to improve patient recruitment and retention. Site-less trials have the potential to dramatically reduce clinical timelines and reduce costs for sponsors. That said, site-less trials are currently not the best choice for complex or specialized trials, and may be challenging to implement with older populations.
Nevertheless, site-less trials will likely continue to grow in popularity as the industry becomes more familiar with the benefits of patient-centricity. Hybrid studies, where patients have a choice as to how they interact with study personnel – either attending in-person appointments at a study site or participating remotely using mHealth technologies – will likely become more common.
Ultimately, the greatest beneficiary of the site-less model may be patients, as this approach makes participation in clinical trials more convenient, while speeding the commercialization of life-saving medicines.
1 Tufts CSDD Press Release, March 10, 2016. Available at: https://csdd.tufts.edu/news/complete_story/tufts_csdd_rd_cost_study_now_published
2 “Stifling New Cures: The True Cost of Lengthy Clinical Drug Trials,” Manhattan Institute for Policy Research, Avik S. A. Roy. March 2012. Available at: https://www.manhattan-institute.org/pdf/fda_05.pdf
3 “Bring Down the Cost of Clinical Trials with Improved Site Selection,” Clinical Leader, December 19th, 2013. Ed Miseta. Available at: https://www.clinicalleader.com/doc/bring-down-the-cost-of-clinical-trials-with-improved-site-selection-0001
4 “Considerations For Improving Patient Recruitment Into Clinical Trials.” The Clinical Leader. Available at: https://www.clinicalleader.com/doc/considerations-for-improving-patient-0001
5 “Home Clinical Trial Visits – A Patient Centric Solution.” https://www.clinicalleader.com/doc/home-clinical-trial-visits-a-patient-centric-solution-0001
6 “Rare Disease Clinical Research: Caregivers’ Perspectives on Barriers and Solutions for Clinical Research Participation.” Neurology April 5, 2016 vol. 86 no. 16 Supplement I8.001
7 “Center Point Clinical Services launches site-less CRO,” CenterWatch News Online, December 8th, 2016. Available at: https://www.centerwatch.com/news-online/2016/12/08/center-point-clinical-services-launches-site-less-cro/
8 “PAREXEL Launches Patient Sensor Solution to Transform Clinical Trial Data Collection,” Business Wire, March 30th, 2017. Available at: https://www.businesswire.com/news/home/20170330005102/en/PAREXEL-Launches-Patient-Sensor-Solution-Transform-Clinical
9 “Patient Recruitment via Social Media: Lessons Learned,” Pharmaceutical Executive, Marylyn Donahue. Feb. 13th, 2012. Available at: https://www.pharmexec.com/patient-recruitment-social-media-lessons-learned
10 “Pfizer Director Defends Virtual Trial after Recruitment Struggle,” Outsourcing-Pharma.com, Nick Taylor. March 6th, 2012. Available at: https://www.outsourcing-pharma.com/Clinical-Development/Pfizer-director-defends-virtual-trial-after-recruitment-struggle
11 “How to Make the Most of Your Online Patient Recruitment,” Forte Research Systems, April Schultz. January 10th, 2017. Available at: https://forteresearch.com/news/make-online-patient-recruitment/
12 “Pharma Sees Great RFID Opportunities for Clinical Trials,” Outsourcing-Pharma.com, Emilie Reymond. May 2nd, 2007. Available at: https://www.outsourcing-pharma.com/Clinical-Development/Pharma-sees-great-RFID-opportunities-for-clinical-trials
Filed Under: Drug Discovery