Clinical trials are the gold standard research methodology for developing new medical knowledge and for bringing new drugs, devices, and treatments to market. And because that market is about improving the lives of humans, they are quite rightly conducted under strict methodological and regulatory frameworks to ensure safety and efficacy. But current, centralized, approaches to clinical trials also make them slow and costly, which is hardly a course of treatment to applaud. Adopting decentralized approaches, including emerging and transformational technologies, will address current shortcomings and result in the accelerated delivery of better therapeutics and vaccines, and at less cost.
While the COVID-19 pandemic has made clinical trials a well known term, they are not at all new. The earliest recorded history of clinical research dates back to approximately 500 BC and involved a comparison between two dietary regimens in an uncontrolled fashion. In one group the subject’s diet consisted primarily of vegetables and water and in the other group, the diet consisted mainly of meat and wine. That research study lasted just 10 days and the vegetable group appeared more nourished than the meat and wine group.
Today, it takes approximately 10 to 12 years for a new drug to come to market and typically only 10% of drugs tested become approved for use. While costs vary depending on the specific clinical trial, it generally costs in the region of 1.5 to more than 2.5 billion US dollars to take a drug from discovery of a therapeutic to delivery of a marketable approved drug.
Many factors have been identified that impact the success of clinical trials reaching their anticipated outcomes, including failure to demonstrate efficacy or safety. However, a major issue is the failure to recruit the requisite number of trial participants that meet eligibility criteria.
Ultimately, the long timeline for bringing potentially life-saving drugs to patients that need them or to patients that have failed other treatments, has major implications for morbidity and mortality. Of the trials that are completed, more than 30% of the costs are due to patient recruitment and retention.
Significantly, privacy and data security are both paramount in performing clinical trials and are often cited as a concern and a reason why patients may not be willing to participate in research, which exacerbate the recruitment challenges..
Clinical trials historically are very centralized around academic medical centers. Likewise, the data necessary for identifying potential research subjects within those hospital systems’ electronic medical records (EMR) are centralized and often insufficient to accurately match subjects to the inclusion criteria. Additionally, social determinants of health (SDoH) are not typically found in EMR system data sets, which limits the ability to identify many patients who may be at the greatest risk for the medical condition being studied. Underserved populations in clinical trials are typically less than 10% of the study population and represent a significant problem in generalizing the clinical research to broader populations. For example, 40% of the US population is composed of underrepresented minority racial and ethnic groups but 75% of clinical trial participants of drugs approved in 2020 were white.
Some of the inefficiencies and constraints of traditional clinical trials can be diminished significantly using a decentralized clinical trial framework. Decentralized clinical trials leverage technologies such as telemedicine, mobile, and local health care providers, and other digital technologies, to eliminate the geographic limitations that often negatively impact traditional trials.
In decentralized clinical trials, dropouts due to patients having to travel long distances to the clinical trial site are decreased. Likewise, enrollment of patients at distant locations significantly improves when decentralized trial technologies are used. Decentralized clinical trials enable trial participants to take part in clinical research from literally anywhere and patients can better integrate their participation into their normal daily lives than in traditional trials. Additionally, as sensor technologies evolve, new forms of digital biomarkers can be generated to better understand interventions and provide new types of datasets not previously available in the analog clinical trial world. Likewise, existing sensor technologies can enable remote collection of a variety of data and open the door for more timely and effective analytic capabilities. Decentralized trials and advanced analytics also makes it more efficient to design and conduct adaptive clinical trials which enable the modification of trials using ongoing, real-time accumulating trial data without altering the validity and integrity of the trial.
Transforming from a model where health data is centralized in enterprise EMRs, and is bought and sold without the data subject benefitting, and where health-relevant/SDOH data is scattered all over the metaverse, to a new paradigm will have dramatic effects on both health care delivery as well as clinical trial execution.
In this new paradigm, patients will be empowered by having ownership, access and control of all their health and health-related data. Utilizing state-of-the-art encryption methodologies, blockchain distributed ledger technology combined with artificial intelligence and machine learning utilizing Federated approaches, will revolutionize the healthcare and life sciences industry.
Blockchain technology can improve identity management, security, data integrity and privacy, as well as provide an immutable audit trail for a variety of workflows. Next-generation artificial intelligence with the ability to perform analytics on mobile device data at the edge in a federated manner, without having to move data to analytic models in a privacy preserving manner, will enable patients to participate in research in ways that have not previously been possible. Additionally, these Web 3.0 technologies will enable patients to monetize their personal data estates in ways not previously envisioned, thus creating new Health 3.0 data economies.
Likewise, pharma companies can engage with networks of empowered patients using blockchain-enabled fine-grained consents for clinical trial matching. Using federated analytics on much more robust datasets from multiple healthcare systems, in addition to inclusion of health-relevant SDoH data, will allow the pharma world to conduct trials that align better to real world scenarios and result in more effective drug development.
While decentralized clinical trials are still a new model that is developing in a traditionally slow moving and technology averse industry, the vital signs of this emerging approach are positive.
In 2020, the Decentralized Trials & Research Alliance (DTRA) was formed with a goal of enabling the collaboration of stakeholders to accelerate the adoption of patient-focused decentralized clinical trials and research within life sciences and healthcare. Now with some 125 world-class global members, the DTRA’s vision is to make research participation accessible to everyone, enabled by the consistent, widespread adoption of appropriate decentralized research methods and cutting edge technologies. Watch this space but stay focused. It’s going to move very quickly.