Democratising clinical research
How a decentralised approach could become the standard for clinical trials
You might think that decentralised trials shot to stardom thanks to the Covid-19 pandemic. But they’ve actually been gaining traction for a while. Way back in 2017, 1170 studies that incorporated some form of digital product to facilitate remote data collection were registered on clinicaltrials.gov.
I’m officially coining the term DeTri.
Let’s dive in…
🤷♂️ Problem
Traditional clinical trials are slow, expensive and inefficient. Less than 5% of eligible patients participate in trials, around one third of enrolled patients drop out and approximately 80% of trials finish late.
They’re also not representative. Real-world patients who receive a specific therapeutic intervention are generally more diverse than trial cohorts when it comes to age, gender, race, ethnicity, disease severity, or co-morbidities.
💡 Solution
Leverage digital health technologies (such as mobile devices, mobile apps, remote monitoring devices, and online social engagement platforms) and integrate them into trial design in order to improve patient recruitment and retention.
📖 Terms
The nomenclature is a bit iffy. A variety of terms have been used to describe trials that incorporate patient-facing and digital health technologies. Virtual trials, decentralised trials, remote trials, direct-to-patient trials, and hybrid trials are often used interchangeably.
Let’s lay down some broad definitions:
Clinical trial. A research study in which patients are prospectively assigned to one or more interventions to evaluate the effects on health-related biomedical or behavioural outcomes.
Decentralised clinical trial (DCT). Integrates modern digital health technologies—such as mobile devices, mobile apps, remote monitoring devices and online social engagement platforms—into clinical trial design.
Contract Research Organisation (CRO). A company that provides support to the pharmaceutical, biotechnology, and medical device industries in the form of research services outsourced on a contract basis. A lot of CROs are innovating in the DCT space.
📚 History
2003. Researchers attempt a prototype double blind randomised placebo controlled trial in people recruited and followed entirely over the internet. The trial investigated the effect of glucosamine on knee pain in patients with osteoarthritis.
2011. The pharmaceutical giant Pfizer receives FDA clearance to conduct a clinical trial entirely online. Every step of the trial, from candidate screening to data reporting, is completed remotely.
2015. The FDA solicit feedback on the use of tele-health technologies to improve efficiency of clinical trial conduct. Major drug companies in Europe and the United States launched feasibility trials using web-based methods.
2016. American biotech Genentech incorporate a videoconferencing and messaging platform into a treatment trial for a rare autoimmune skin condition. Candidates from seven US states are recruited online, where enrolment was more than 20 times faster than for non-remote sites.
2020. In response to the Covid-19 pandemic, the FDA release guidance urging clinical trials to go virtual where possible.
💼 Use cases
Digital technologies might be deployed at one or more stages of a clinical trial:
Patient recruitment
Patient consent
Patient engagement
Data collection
Outcome assessments
Data sharing
👥 Players
The virtual clinical trials market cap is set to reach $11.5B by 2028. Here are just a few of the companies propelling it to those dizzying heights:
IQVIA. An American multinational serving the combined industries of health information technology and clinical research.
Medable. Provides a flexible and modular software platform that enables clinicians to shift from clinic-centric to patient-centric research strategies.
Science37. Uses its system of tele-health programs to remotely screen patients and conduct study visits, removing the need for physical trial sites.
Signant Health. US based company specialising in the delivery of digitally enabled trials.
Parexel. American provider of biopharmaceutical services. Conducts clinical trials on behalf of pharmaceutical clients to expedite the drug approval process.
Covance. Provide a DCT model that provides a more patient-centric approach to study design and execution, ready for COVID-19 and beyond.
PRA Health Sciences. A CRO headquartered in North Carolina. Aims to reduce the burden of research on the patient through the use of mobile technology and connected devices.
Medidata. American technology company that develops and markets software as a service for clinical trials.
Clinical Ink. Enhance clinical trial workflow by reducing manual labor, providing anytime data access, and optimising resources as clinical trials progress.
Decentralized Trial & Research Alliance. Aims to unite industry stakeholders in a mission to make clinical trial participation widely accessible by advancing policies, research practices and new technologies in decentralized clinical research.
🤔 Challenges
Digital literacy. Remote trials may require participants to play an active role in their own data collection. This may not be intuitive for everyone, especially those from a low socioeconomic background, as well as older individuals.
Signal in the noise. Just because you can measure something doesn’t mean you should. Investigators need to ensure that their methodology is hypothesis-driven, rather than by the availability of technology that can collect loads of data.
Data deluge. Continuous patient monitoring through apps and wearables generates a huuuuge amount of data. Appropriate data analysis software will be required to sift through it all.
Dodgy tech. Remote monitoring equipment malfunction could be costly or time-consuming to replace. Lost patient data from equipment issues will delay trial progress.
Patient safety. If patients don’t have direct personal contact with sites or study physicians, there are concerns there may not be enough recognition of serious adverse events or assurances that patients will get the relevant care.
Privacy. Some patient’s won’t feel comfortable sharing sensitive health information over the internet. Secure systems are required.
Dodgy targeting. Trial sponsors might cut out the clinical middlemen (and women) and start recruiting participants based on their Google searches and digital data. Creepy and potentially inappropriate.
🌅 Opportunities
Increased diversity. Virtual trials can recruit patient recruitment from a much wider pool rather than limited to those within a reasonable distance of a participating site. This especially benefits rare disease research, where clinical trials are often competing for the same limited pool of participants.
Recruit and retain. Systematic reviews identifying barriers to clinical trials highlight that one factor in poor recruitment and retention rates are the burdens placed on participants through additional on-site appointments or procedures. Virtual trials could solve that.
Hurry up! ‘AT-HOME-PD’, a fully-virtual extension of two phase III clinical trials, was able to recruit participants within just 6 months of funding. Virtual trials can get out the starting gates much quicker than conventional trials.
Continuous monitoring. Moving into the community and reducing researcher input can facilitate near-continuous data collection complementing specific endpoints normally captured through in-person visits.
Build trust. Some groups in society have suffered historical injustices at the hands of medical research. DCTs could help repair previous damage and ensure patients from all walks of life are adequately and safely represented.
🔮 Predictions
Increased satisfaction. A feasibility study of remote research visits in Parkinson’s Disease patients demonstrated high satisfaction with participation, and increased reported willingness and ability to participate in trials with remote data collection.
Patient-centric. The considerations required to ensure trials can be successfully carried out virtually will help ensure patients are placed at the centre of the clinical trial process.
Cheaper trials. The majority of the pharmaceutical and healthcare industry expects the costs of DCTs to be lower compared with traditional trials.
Speedy drugs. A combination of lower costs, operational efficiencies and higher quality data will reduce the time it takes for effective drugs to reach the market.
Mixed methods. It’s unlikely that all trials will be fully virtual. Most will use a blended approach combining in-person and remote components.
Trial couriers. Trial patients need special medicines and equipments. Delivering these in a safe, controlled and timely manner will require specialist couriers. New business opportunity?
🔗 Links
This paper exploring some of the issues around virtual clinical trials
This short article on the benefits of DCTs
That’s it for this week - catch ya next time 👋