By Rodika Tollefson
Clinical trials are an integral part of getting a new pharmaceutical drug on the market. But research and development (R&D) is a slow, expensive process, and the challenges are many patients, from recruiting and managing the trial sites to maintain data integrity.
Total cost estimates for bringing a drug to market vary widely, with some research placing it just south of $2.9 billion. More recent data brings the average closer to $1.3 billion and the median to $985 million. Regardless of how the numbers pencil out exactly, clinical trials take up a large share of the R&D costs—and the high financial cost and the lengthy duration historically have posed the biggest barriers to clinical research, according to a report by the US Department of Human Services.
According to Neill Barron, part of the problem is that clinical trials have grown more complex, but the underlying processes have remained the same. He saw this first-hand during his 26 years in the pharmaceutical industry.
“We’re now in the age of big data, but the pharmaceutical industry hasn’t moved with the times,” says Barron, director of clinical data analytics at ClinTex. “We’ve moved from paper processes to electronic, but we’re still doing the same things we did in 1995. We have more tools now, and the trials are becoming more inefficient because of all the complexity.”
ClinTex, a London-based technology startup, wants to solve some of those complexities with blockchain and predictive analytics. The company says its newly launched Clinical Trials Intelligence (CTI) platform—which combines blockchain, machine learning (ML) and data analytics technologies—can help keep clinical trials on track by allowing various stakeholders to visualize operational data and prevent or address problems that can derail the timelines.
Why the blockchain?
Some of the most significant challenges in clinical trials stem from data, such as data management, quality, integrity, analytics and reporting. Data falsification or misrepresentation is among the problems, with various fraud cases surfacing in recent years. Because blockchain decentralizes various tasks and validates transactions, it provides cryptographic proof of the data lineage, or an immutable ledger; researchers or clinicians can’t make any changes to the records as they could with a traditional program.
“Data needs to have a high level of integrity and quality to be valid, and it can’t be tampered with—that’s very difficult to achieve,” says Sam Dhesi, MD, who previously served as the head of Emerging Technology for the UK’s Department of Health and Social Care. “Having data stored on the blockchain enables trust that it hasn’t been tampered with.”
Although blockchain could solve a variety of problems that drive up costs, ClinTex started with what Barron sees as a fundamental, underlying problem across the industry: data silos and lack of information sharing.
Data needs to have a high level of integrity and quality to be valid, and it can’t be tampered with—that’s very difficult to achieve. Having data stored on the blockchain enables trust that it hasn’t been tampered with.
—Sam Dhesi, former head of Emerging Technology, UK’s Department of Health and Social Care
“At any one time, there are tens of thousands of ongoing trials, but they don’t share any lessons learned or reuse any information, and that makes those trials inefficient,” he says. “We feel that data is the way to get that collaboration going.”
The industry has acknowledged this problem, and some initiatives have attempted to change the status quo. ClinTex hopes its technology can encourage this much-needed collaboration.
“There are data silos even within a pharma company itself, not to mention across the industry,” Barron says. “Data sharing, data quality, collaboration around data and poor usage of that data to drive decisions—having a common platform can allow these issues to be addressed.”
The ClinTex CTI platform extracts the raw clinical trials data into a data model that anonymizes and summarizes it, protecting intellectual property tied with the source. It doesn’t divulge information such as drug efficacy, for example. However, it does help develop metrics that drive standard key performance indicators (KPIs).
With an analytics layer on top of these KPIs, and a visualization layer on top of that, the ClinTex CTI technology allows trial managers, data managers and remote staff to quickly assess if the trial is progressing as planned or identify and troubleshoot problems.
Data sharing, data quality, collaboration around data and poor usage of that data to drive decisions—having a common platform can allow these issues to be addressed.
—Neill Barron, director of clinical data analytics, ClinTex
One example is missing data. This often requires a lot of back-and-forth communication between the pharmaceutical company and the clinical investigator, slowing down reporting.
“Part of what our tool does is identify where the issues are happening so the sites can make sure they’re achieving the data accurately,” Barron says.
Solving multiple issues at once
The ClinTex platform is in its first iteration, and the company plans to add six more modules (including two planned for launch in 2022). The collection of modules will add capabilities for tasks such as visualizing clinical data, managing patient recruitment and predicting operational problems that can be addressed proactively.
In the case of patient participation, for instance, early trial dropouts are common. While physicians are paid for each patient they recruit into the study, the patients aren’t incentivized. Factors that may lead to early withdrawal range from age to adverse medication interaction.
Patient recruitment accounts for as much as 30% of a clinical trial’s timeline, and early patient withdrawal can further slow down the process. Using ML algorithms to analyze different data sets, ClinTex could identify which patients are at risk of dropping out.
“The investigator could proactively investigate potential root causes and intervene,” Barron says. “And they could also look at it afterward retrospectively and use that intelligence to drive better trial design.”
Dhesi, who became familiar with ClinTex during his time working as a doctor and medical researcher in the National Health Service, believes the modular, comprehensive platform the company is developing is unique in the industry. These kinds of projects typically focus narrowly on problems like storing clinical data on the blockchain or recruiting patients. ClinTex, on the other hand, is tackling a range of cost drivers.
“It appears that ClinTex is developing the operating system for trials of the future,” he says.
Another aspect that’s different for ClinTex is tokenization. The company has developed its own cryptocurrency token, on the Ethereum blockchain, that is publicly traded on several cryptocurrency exchanges.
The token is currently used for payments to clinical investigators and access to the platform via its licensing model. The use of a crypto token does have some disadvantages, such as exposure to fluctuations in the price of blockchain network transfer fees. But it has allowed ClinTex to form its own micro-economy where stakeholders can be rewarded through the token directly, keeping clinical trial costs down.
Barron acknowledges that the biggest obstacle—the lack of industry collaboration—is not an easy one to remove. Some of the problem is cultural, he believes, because this is “such a closed industry” that needs to protect intellectual property and other data.
Dhesi agrees, noting that pharmaceutical companies are historically slow-moving organizations, and it takes time to overcome distrust in an idea such as decentralization. But another cultural barrier is distrust in blockchain technology itself.
“Any cutting-edge technologies are at a certain place on the hypo curve. There was a lot of hype around blockchain a few years ago, but the use cases were poorly understood,” he says.
The hype has come down, and there’s more clarity around the use cases, Dhesi adds, but “we’re still not quite there culturally in terms of completely trusting the technology.”
There are, of course, other challenges, such as the energy footprint impact and internet connectivity in remote areas.
Regardless, the idea of decentralization is taking hold. For instance, researchers are looking at how blockchain could impact the growing need for virtual clinical trials to cut costs and increase patient engagement. Even the IEEE (which sets standards for a range of technologies) is exploring how to conduct blockchain can “be tapped to better and accelerate clinical trials that will innovation in healthcare.”
ClinTex is only in the beginning stages of taking its solution to the market. And Brendan Mannion, a technology engineer who’s the company’s director of operations, says the company is still “breaking things” and trying to understand the best way to use blockchain. The full potential of the technology is yet to be realized.
“We have to be flexible in our scope and how we use blockchain and cryptocurrency,” he says. “The whole space is moving so fast and innovating so fast. We have a plan—but we’re going to keep pivoting a bit in the future.”
Lead photo courtesy of Getty