Data Quality Issues Continue to Delay Drugs Development

But an exciting blockchain-based data analytics solution is finally on the horizon

In yet another case highlighting the failure of legacy data management systems in clinical trials, US-based pharma Zogenix’s application for the release of a drug to treat seizures associated with Dravet syndrome, a form of epilepsy, was denied last month by the Food and Drug Administration (FDA). Zogenix completed phase III trials of the drug, called Fintepla (ZX008, fenfluramine oral solution), in September of last year and had expected FDA approval by early 2020. However, the FDA was forced to delay approval due to a need for “additional data.” This latest delay comes on the heels of another delay for the same drug. In April 2019, according to this release from Zogenix, the FDA rejected Zogenix’s Feb 2019 application on the basis that it “contained an incorrect version of a clinical dataset.” The persistent delays, both of which are related to failures of data management, mean a critical treatment for sufferers of Dravet syndrome, a form of epilepsy that cannot be effectively treated with existing anti-epilepsy medicines, remains unavailable.

Only one in 10 drugs that starts trials gets approved by the FDA, and it now takes on average more than 10 years from concept to approval. Both the time to approval and the cost of development, which now sits at a colossal $2.558 billion per treatment, have ballooned over the last 20 years. While there are numerous contributing factors at play, poor data management — as highlighted by Zogenix’s failure — has emerged as a frustratingly difficult problem to overcome. In the digital age, where our power to capture and analyze data has never been so great, how can it be that inadequate data management holds back the development of important medical treatments?

As our ability to capture and share data has improved with digital technologies, the challenges related to the secure management of that data have likewise increased. Amidst high-profile data breaches like the Equifax disaster in which personal financial information for close to 150 million people was leaked, data security has become a high priority in all industries. When it comes to the development of medical treatments, which involves the collection of sensitive data on patients, the need to manage intellectual property, and the necessity to meet the stringent requirements of regulators, data security is of paramount importance.

Blockchain-based IT solutions have emerged in recent years as the best way to improve data security in healthcare more generally. A number of startups, like UK-based Medicalchain and US-based BustIQ, are now offering viable solutions for managing and sharing electronic health records, while a number of national health agencies globally have conducted pilots to explore blockchain-based healthcare data management.

Blockchain-based solutions are also being developed specifically for applications in clinical trials. The EU-backed public-private consortium PharmaLedger, in fact, identifies clinical trials — along with supply chain and health data — as one of the three most promising applications for blockchain in healthcare.

While maintaining the security of the data collected in clinical trials for the development of new medicines is essential, it’s also critical to have the capacity to analyze that data. ClinTex’s Clinical Trials Intelligence platform (ClinTex CTi), currently under development by a UK-based team of clinical trials professionals and blockchain/software experts, creates a complete solution for the gathering, management, and analysis of data in clinical trials.

“The volume and variety of clinical data sources to support new medicines development is growing. This clearly presents data protection challenges, for which blockchain is an ideal solution. Less thought of, however, is how we derive insights and intelligence from this data to drive collaboration across researchers in academia, biotech and big pharma. CTi is leveraging blockchain to drive this collaboration and deliver medicines to patients faster and cheaper”

-Brendan O’Mainnin, Co-Founder, ClinTex

Blockchain technology ensures data security and integrity by distributing data across participants while providing permissioned and granular read-write access using public-private key pair cryptography. That mouthful of industry jargon can be translated as follows: all stakeholders in a clinical trial — from researchers, to vendors, regulators, and patients — can be provided with a secure view of the data collected in the trials that is specific to their position. For example, regulators can be given a universal view but with patient information anonymized. At the same time, certain stakeholders can be given specific write permissions. A patient, for example, could be given permission to contribute information to a specific data set that is critical for the trial, such as heartrate data gathered from a wearable and personal reports added from a mobile device. Finally, the pharma or clinical research organization managing the trial can be given a secure but universal view of the data in real-time as it’s collected.

These features, unlocked by the new technology, make it possible to eliminate, once and for all, the frustrating data management and analysis-related failures that are so common in clinical trials today — and that lead to delays in introducing important medical treatments.

ClinTex CTi deploys a suite of applications that, together, unlock the power of digitalization in clinical trials medical research. One application in particular, the Clinical Data Visualisation Application (CTi-CDV), is relevant to the data management failures that have been holding back the approval of important drugs like Zogenix’s Fintepla. CTi-CDV provides enhanced clinical data review through the use of data visualisations to identify issues that may impact on the trial integrity and safety of the patient. CTi-CDV can facilitate the flagging of potential issues like data quality and disease exacerbation that may impact a clinical trial’s success. Importantly, CTi-CDV can offer, in real-time, the corrective actions that need to be recorded and monitored in order to protect the integrity of the trial while upholding patient safety.

“In my 20 years’ experience working on and around clinical trials, I’ve seen too many costly and life impacting delays caused by issues that, frankly, should be trivial to solve in the digital age. Finally, with emergence of these new technologies, we’re able to develop a next generation clinical trial intelligence platform that will, at last, enable streamlined and safe development of important medical treatments, without delay.”

• Ethan Diamant, Director of Clinical Data Services, ClinTex.