Fixing Clinical Trials Through Blockchain

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In my recent conversation with Health:Further CEO Marcus Whitney, he highlighted blockchain as a technology that has not yet reached the promise and full maturity of the hype that surrounds it. “It’s not overhyped,” said Whitney, but it’s still a young technology. “It’s the World Wide Web was in 1994.” (Nicolas Cole makes a similar comparison in his blog post “2018 will be the year blockchain technology goes mainstream.”)

So if blockchain is young and full of technological potential, what will the future look like, and what does it mean for healthcare? There’s of course a lot of conversation around blockchain as one of the potential solutions to issues surrounding data ownership, data transparency, personal health information and EHRs. The applications go even deeper into areas of health information technology, though. One area that doesn’t seem to be getting as much above-the-fold coverage is the realm of research and clinical studies. Because the future of our health and healthcare system is dependent on a robust pipeline of new ideas, technologies and treatments, this is a possibly under-appreciated but still important use of blockchain that we wanted to highlight.


The Problems With Clinical Trials

A survey of clinical research professionals from 2016 found that the most common challenge cited by respondents was “trial design.” Here’s the summary:

Clinical trials have been growing increasingly complex for years, and those running them are feeling the pressure to design trials ‘that give the right answers, in the most simple and unobtrusive way for patients, that are acceptable to regulators and payers’.

Respondents from study sites are particularly concerned with meeting the ‘challenge of focussing on the best study design’ for ‘very complex modern clinical trials’. Such concerns are borne out in the ‘high rate of failure to meet primary endpoints due to poor or complex design’.

The second biggest challenge was regulations, leading “many respondents [to] feel constrained by the complexity of guidelines they follow.” I have also heard anecdotally from members of the clinical trial community that simply getting enough patients into a study is often a massive headache. There may be too few people with the disease being treated, people who could qualify may not be aware of the study, or the information presented may not be sufficient to attract participants. With all of these challenges, it’s easy to see how mistakes could creep in, and the clinical trial community, along with regulatory bodies like the FDA, work hard to ensure that they’re mitigated.

Unfortunately, the problems with clinical trials don’t stop at procedural or statistical error. As is the case with any high-stakes issue, there are instances of fraud and manipulation. Here’s how Stephen George and Marc Buyse put it in a 2015 research paper on the subject:

There is no reliable evidence that data fraud, the deliberate fabrication or falsification of data, is a common occurrence in clinical trials. Moreover, in multicenter clinical trials, fraud perpetrated by a single investigator or at a single site is very unlikely to affect the scientific conclusions of the trial. However, whatever the true incidence of data fraud in clinical trials, high-profile cases provide sobering evidence that it does occur regularly. When fraud is detected after the results have been announced, the negative impact on the perception of the results of the trial in question as well as on the public perception of the clinical trial enterprise itself can be profound. 

Of course, the FDA is aware of the issue and continually works to prevent it. To take a random example: FDA collaborating with RBM software provider to enhance trial oversight.

I don’t want to overstate the problem, nor do I want to seem as though I’m taking shots at the clinical trial community. So I’ll simply say that in a world where the results of a study are potentially life and death for patients, ANY level of error needs to be minimized and fraud MUST be pursued and shut down. Obvious statement, right?


Blockchain As a Solution?

This is where blockchain can play a role.

It all comes down to caring for patients responsibly, whether that’s in a clinical setting or during a trial. We often talk about data ownership and security in the context of personal health information and electronic medical records. The same issues apply to clinical trials.

According to a 2017 article by Benchoufi and colleagues, almost 10% of trials [the FDA] monitored have issues with consent collection. These problems include “failure to re-consent when new information becomes available [and] failure to obtain written informed consent,” among others. In other words, these patients aren’t necessarily aware of changes made during the course of a trial and/or their consent is not documented.

“consent is a dynamic process that involves a complex circuit of data and interacting actors,” say the authors. A statement which, to blockchain proponents, sounds like a perfect opening. A blockchain system can provide unambiguous proof of consent. Timestamping each ledger entry, a hallmark of blockchain, “enabl[es] clinical research stakeholders, such as sponsors, investigators and IRBs […] to share consent and re-consent related data in real time, and archive and historicize consent sets […]”

With that in mind, the authors developed a method for “unfalsifiable time-stamping information” using the Bitcoin network. Through relatively simple dashboard, study participants were able to consent and, if necessary, re-consent. The authors were able to track these changes, as well as their interactions (such as emails) with participants, in a single secure document.

Along those same lines, the decentralization provided by blockchain means that patients have far more control over their clinical trial data. This is exactly the same benefit we see when talking about PHI such as EHRs and genomic information. According to Benchoufi et al, blockchain “gives to the patient, or more widely to patient communities, control over their consent and its revocation. The end-to-end connectivity creates a network that empowers patients and researchers as peers” (Emphasis added).

Improving the reliability and reproducibility of scientific studies is a continuous process, with obvious importance for potentially life-and-death projects like clinical trials. And, of course, trust in both specific studies and the scientific endeavor as a whole is undermined when problems occur.

A 2017 commentary, also written by Benchoufi along with Philippe Ravaud, suggests that the simple ability to track data in a robust and (theoretically) unfalsifiable way will improve the world of clinical trials.

[Blockchain] involves a decentralised secure tracking system for any data interactions that could occur in the context of clinical trials, with a peer-to-peer inclusive network that enables data sharing on the research side and ensures all the needed transparency and care for privacy concerns on the patient community side.

In turn, this system can lead to more trust in clinical research, whose credibility has been considerably undermined with repeated scandals in recent years. Blockchain technology can be considered a basis for improved clinical research methodology and a step toward better transparency to improve trust within research communities and between research and patient communities.

As the authors note, blockchain is built around “privacy-by-design.” It’s the equivalent of having to opt-in to an email list rather than be added by default and have to opt-out. As a result, it gives patients far more confidence that their information will be - must be, by default - respected and used wisely. Ultimately, they say, people will be more likely to share their personal health information for research purposes because they are confident it won’t be abused.

This is the future of healthcare we want to see realized. One in which patients, providers, and other stakeholders such as clinical trial managers have a relationship built on trust and scientific rigor. One in which patients have control of their own information and work collaboratively with their medical team to use that information effectively.

For one more article on blockchain’s potential use in improving clinical trial methodology and reducing fraud, be sure to check out Improving data transparency in clinical trials using blockchain smart contracts.