Digital clinical trials – pharma’s next secret sauce?

Clinical trials are slow, expensive and fail most of the time. While digital technologies have transformed preclinical studies, pharma companies have been reluctant to digitise clinical trials due to concerns around regulatory acceptance. However, some, unfazed, are seizing opportunities presented by Electronic Health Records (EHRs), the Internet of Things and AI and making big bets on digital.

The potential opportunity is huge – clinical trials account for half of the $2bn it takes to bring a drug to market and high costs are increasingly unsustainable.

A pioneer in digital clinical trials

Pfizer has been an early adopter of digital in clinical trials. It has been conducting small-scale pilots, scaling up successes and examining failures. A decade ago, it launched a pilot programme, the first ‘virtual’ clinical trial, where patients could participate entirely from home. The trial was investigating a hypertonic bladder medication and patients were to be screened online, manage their own trial activity and report results online to a trial investigator. While the trial had to be abandoned due to recruitment challenges, Pfizer has since successfully implemented the first regulatory grade decentralized trial, expecting it will improve retention and yield high-quality real-world evidence.

Digitising clinical trials has been a key component of Pfizer’s R&D turnaround strategy. Pfizer set itself the goal to reduce its clinical trials by 2.5 years by the end of 2021 and claims to be making great progress. Automation has been the focus for achieving this goal – clinical trials are human-intensive and require many manual processes and transfers of data. Pfizer is creating an environment where data can flow directly from a patient into an
electronic database after anonymisation and populate clinical trial submission documents,
without having touched human hands.

Pfizer and BioNTech’s pivotal Phase 3 Covid-19 vaccine trial is an example of how disruptive technology can streamline trials – over 44,000 patients were recruited and a submission was made within 248 days, an extraordinary feat. ICON CRO, who ran the trial for Pfizer, credited digital innovations enabling remote monitoring as one of the key reasons for trial efficiency. Approximately 90% of the trial monitoring was remote. This enabled operational efficiencies such as using a team in Japan to cover American sites to maximise the value of different time zones.

Digital technology can’t solve all the challenges of clinical trials. Pfizer has recognised digital solutions should be evidence-based and alleviate a defined issue, implementing digital as part of its wider R&D strategy through determining best use cases and propagating successes. It is also identifying use cases in creative ways, last year challenging AI companies in a hackathon to predict data discrepancies in clinical trial datasets.

The future of digital in clinical trial design

To date, digital solutions have mostly been for replacing standards – tablets instead of paper – but the truly disruptive nature of digital may be realised in trial design. Adaptive designs will be supported by digital biomarkers that indicate when pre-specified changes to trial design can occur and when patients will drop out. Advanced analytics including AI and big data will support platform trials, for instance, they will recognise similarities in basket trials to borrow across baskets and reduce the number of patients needed.

Covid-19 has prompted a ‘golden age within clinical development landscape’ according to Steve Cutler, CEO of ICON CRO, where capital and developments in science and technology are enabling the transformation of clinical trials. With regulators more receptive to the idea of digital clinical trials and evidence, taking the opportunity to demonstrate validity and advantages of approaches is sure to pay off.