Clinical trials need to keep up with modern science to ensure patients get access to life-changing, life-saving drugs as soon as possible.
That’s according to US regulator, the FDA, which has announced a raft of measures aimed at modernizing clinical trials.
“New research paradigms are needed to break down barriers between real world data and clinical research,” Scott Gottlieb, M.D, noted in his last months as FDA Commissioner.
“For instance, more trials could incorporate data from electronic health records, and adopt electronic informed consent, to enroll more patients in clinical trials closer to where they live and work.”
As more diseases are redefined based on genomic subtype, and precision-guided medicines become increasingly able to demonstrate efficacy early on, the potential to reduce “suffering, death and disability” is growing.
“Efforts to streamline medical product development based on advancing science can be frustrated by legacy business models that discourage collaboration, data sharing, and the adoption of disruptive technologies that make clinical research more effective,” said Gottlieb.
“Without a more agile clinical research enterprise capable of testing more therapies or combinations of therapies against an expanding array of targets more efficiently and at lower total cost, important therapeutic opportunities may be delayed or discarded.”
A new set of FDA guideline documents for industry have been designed to help.
Enrichment Strategies for Clinical Trials to Support Determinations of Effectiveness of Human Drugs and Biological Products outlines a key focus for the FDA, using clinical laboratory tests, genomic or proteomic factors to target treatments to patients within a trial.
This ‘precision medicine’ can make it easier to detect a drug’s effect in people with a particular biomarker and means smaller or shorter clinical trials can be just as robust as larger, longer studies.
Added Gottlieb: “The information gleaned from these approaches can also guide clinical practice, to help doctors get the right treatment to the right patient at the right time.
“The same information used to select patients based on their likelihood of responding positively to a drug in a trial can be used to guide real world care.”
Designing risk-based monitoring
A Risk-Based Approach to Monitoring of Clinical Investigations: Questions and Answers for Industry identifies another key area for the FDA: patient safety.
Currently, there are no clear guidelines on how study participants should be monitored, Gottlieb pointed out.
“Traditional on-site monitoring of each clinical site … is highly resource intensive and may account for up to a third of the total clinical trial cost.
“But traditional on-site monitoring that is overly focused on source data verification doesn’t guarantee data quality,” he added.
This document helps sponsors walk the center line. It guides them through the tailoring of monitoring plans by focusing on those risks with greatest potential to affect study quality and safety.
These publications, Gottlieb explained, are part of an agency-wide drive to modernize clinical trials by streamlining the drug development pathway.
“The agency is committed to developing a regulatory framework for precision medicine that generates robust evidence of product safety and efficacy as efficiently as possible. [This includes] frameworks that are more carefully suited to the kinds of precision technologies that underpin new treatments,” he said.