Clinical trials of the future

Back-to-back Covid-19 mRNA vaccines suggesting 95 percent efficacy are a shot in the arm for biotech’s role in helping to solve some of humanity’s health challenges. Behind the headlines, another frontier technology – virtual clinical trials – came of age came of age supporting the research triumph.

Virtual trials rely upon digital platforms to address chronic issues in drug discovery, such as assembling sufficient trial cohorts, enabling remote research collaboration in real time, and unifying relevant data sets. They support the quality, standardization, reproducibility, and efficiency of clinical research while maintaining regulatory compliance. Pandemic lockdowns can further exacerbate clinical trial challenges by cutting access to clinical trial participants. 

Even as the COVID pandemic shut down drug trials elsewhere, a leading US biotech firm was able to continue testing its breakthrough coronavirus vaccine on a virtual trial platform designed by Medidata. It was proof-of-concept for the role virtual trials may play in enabling solutions to humanity’s most significant health challenges, from cancer to heart disease.

“Medidata’s unified platform is helping us put patients at the center of our efforts to develop a safe and effective vaccine against Covid-19,” said an executive at the drug discovery firm.

A patient-centric approach to humanity’s health challenges

At the heart of virtual clinical trials is patient centricity. This means putting participant safety, convenience, health, and emotional needs first by leveraging digital technologies to enable human connection.

Physicians and their patients who leverage virtual tools can enroll and submit consent forms electronically over their mobile devices. They participate from home – sending in results and communicating with doctors – without regular in-person clinic visits. Concerns can be addressed in real-time over video-conferencing with clinical trial professionals.

In a recent Investigational New Drug Phase 3 trial to treat Wagner Grade 3-4 Diabetic Foot Ulcers (DFUs), trial participants were able to obtain important wound images during a virtual visit. These images were later integrated into the study database. TissueTech, Inc. an emerging biotechnology company, was then able to proceed with their late-phase clinical trial thanks to this technology, utilizing the Medidata platform to complete remote monitoring activities.

“The technology,” says Nick McCoy, Vice President, Clinical Operations at TissueTech, “allowed us to quickly pivot when the pandemic hit. We were able to continue to meet the needs of both trial participants and investigators at multiple sites.”

Resolving a host of mounting drug discovery challenges

Even before Covid-19 hit, pressures on clinical trials were already mounting. Participant scarcity, soaring costs, scattered data sets, paper document filing, and manual spreadsheet analyses had bogged down treatments and cures to critical health challenges for years.

The pandemic focused attention on these issues, as well as clinical trial virtualization as a potential solution. “Among the wake-up calls this novel coronavirus has sounded the need to transform the way we conduct clinical trials and utilize the resulting data,” says Glen de Vries, co-founder and co-CEO of Medidata, a Dassault Systèmes company.

“The pandemic has exposed limitations in the clinical trial process that have had repercussions not only in the development of drugs but also in how we might have been better prepared to face this pandemic head-on,” de Vries added.

According to a Tufts Center for the Study of Drug Development study1, the cost of developing a new prescription medicine that gains marketing approval has risen 145 percent over a decade to $2.6 billion – even as success rates have plunged by nearly half to only 12 percent.

Meanwhile, a total of 90 percent of all trials worldwide are unable to find and enroll participants within their target timeframes, and 76 percent of studies miss enrollment targets altogether2.

Virtualized clinical trials promise to overcome such problems. One of those groundbreaking advantages is reducing the need for large patient enrollment. Artificial intelligence (AI) tools and significant data assets (collected in one digital library) can combine current patient data with historical data from earlier trials to enable robust testing with a limited cohort.

Even as they decrease the need for large patient numbers, virtual clinical trials open the door to far greater enrollment by allowing remote testing. This is particularly critical in the case of rare diseases.

Machine learning tools, meanwhile, match patient profiles to trials, translating into more robust, better-designed trials.

Not least, a unified digital platform yields dramatic cost savings through operational efficiencies. With Medidata, researchers administer studies in one digital environment, seamlessly moving between every platform capability, from access control to key risk indicators. The dashboard gives intuitive readouts of clinical outcomes, eliminating unwieldy spreadsheets.

A future full of unlimited possibility

Ultimately the mission, says de Vries, is to deliver more accurate, timely, and insightful clinical outcomes that lead to transformative healthcare solutions for humanity. Patient-centric methodologies and evolving machine learning capabilities will combine to enable more accurate and insightful analysis of data outcomes.

“Moving toward virtual trials enables the process to proceed without external interruption, but it also allows for better data aggregation and analysis,” says de Vries.

“With advancements in digitization and connectivity, particularly as we increasingly virtualize clinical trials, we can do a better job in everything from rare diseases to diabetes to oncology and beyond.”