Clinical trials becoming more flexible and targeted due to new technologies, says expert

Life sciences specialist Helen Cline of Pinsent Masons, the law firm behind Out-Law.com, said that 'big data' can be used to focus clinical trials more effectively and to "improve understanding of how a treatment will work in practice".

"There’s a big opportunity here," she said. "If life science companies can get enough insight early in a drug's development, resources can be prioritised for the most promising therapies. Data analytics and technology are already changing how trials are conducted and can increase the value of the data and insights that come out of these trials."

Cline was commenting after Martin Kohn, chief medical scientist at Sentrian and former chief medical scientist of IBM, said that big data-based clinical trials (6-page / 45KB PDF) should replace the "flawed" existing clinical trials model.

"Our ultimate goal is prevention," Kohn said, according to a report by Wired magazine. "If we want prevention to be effective both medically and economically then we need big data."

Cline said there are already examples of clinical trials where data is helping to inform the studies being undertaken. She highlighted Cancer Research UK’s recently launched 'National Lung Cancer Matrix' trial, which is an adaptive approach to clinical trials that aims to advance lung cancer treatment.

"As proposed, the trial model recognises that every patient’s cancer is unique and adopts a more personalised approach, targeting particular genetic abnormalities in each patient’s tumour," Cline said.

"With the advances that are being made in stratified medicine and health information technology, including the use of real world data, the randomised, double-blind, placebo-controlled model may no longer be the best approach in all cases," Cline said. "Data generated in randomised clinical trials do not typically demonstrate how medicines perform in a normal clinical setting; this can make it difficult for decision makers to determine accurately a medicine’s possible value and may subsequently restrict patient access."

"Digital technologies, along with advancements in data analytics, genomic and diagnostic technologies, along with access to real-world patient data, are making more flexible clinical trial models, that improve understanding of how a treatment will work in practice, possible," she said.

"Connecting the growing volume of information from fitness trackers, connected medical devices, implantables and sensors with traditional health information, medical records, clinical research data and genomic data has the capability to optimise medicines development but there are challenges; data accuracy; data security; privacy concerns and regulatory oversight," Cline said. "As companies strive to turn this data into actionable insights and set up a secure collaborative environment we are likely to see more partnerships between traditional life sciences companies and information technology specialists."

She highlighted IBM's recent partnerships with Apple, Johnston & Johnston and medical technology company Medtronics on health data as an example of the arrangements we are likely to see more of.

The Salford Lung Study is another "innovative on-going project" that demonstrates the potential of collaborations across the wider health care system, Cline said. The study is exploring the real-world benefits of a late-stage GSK investigational respiratory medicine alongside currently available treatments and involves new drugs being tested before they have been fully licensed by regulators (7-page / 847KB PDF).

"If these new trial models were embedded in the EMA’s adaptive pathway to drug licensing, the approval and reimbursement status of new medicines may be more certain and it may be possible to secure market access for drugs that provide a benefit for subgroups of patients but do not pass the traditional evidentiary thresholds for approval and reimbursement," Cline said.