Last Updated on January 11, 2023 by Raoul Patel
Data Could be the Key to Improving Drug Trials and Dropping Placebos
Clinical trials often involve two sets of patients, one that receives a new drug and another which receives a placebo. Some argue that placebo using trials are unfair and unethical. Better data gathering and availability could be the solution to replace placebos and result in more effective clinical drugs trials.
Placebo trials date back to the 1800s and have been used in medicine for over 150 years. But often trial patients take the risk of getting an ineffective treatment and never know if they were given a drug that could work, or not. As per Wired, the flip side of this is that clinical trials are not designed to treat patients that are involved, but instead “future patients,” as per OpenNotes Keane Scholar Charlotte Blease who confirms the purpose “of a clinical trial is not to treat the individual who participates.” But Blease adds that “most patients are not debriefed after clinical trials,” calling the practice of not revealing if a patient received an actual drug or a placebo a “terrible way to treat clinical participants.”
Digitisation of health records could replace placebo trials
Digital transformation is offering a potential solution to placebo trials. Better availability of health records such as doctors’ records and pathology reports could replace the need for a group taking a placebo. Instead real-world patients and their records could be used as a comparison with drug trial participants. Hence control group results would be generated instead from patient records.
In 2018 pharmaceutical company Roche bought oncology company Flatiron health for $1.9 billion. The smaller company had been working with the US Food and Drug Administration (FDA) since 2016 to understand the potential of using real world evidence as a control mechanism in trials.
In 2019, the FDA approved the first drug based on tests that used real world data. A partnership between Pfizer and Flatiron Health enabled the FDA to use health records in order to approve a breast cancer drug, Ibrance, to also be used as a treatment for men.
David Harland, medical head of personalised healthcare at Roche UK, says we’re “at a tipping point,” when it comes to systematically collecting real world data, adding:David Harland, medical head of personalised healthcare at Roche UK
“We’re now at the place where we can start to collate and curate that data to a very high regulatory grade.”
Other pharmaceutical and drug development companies are also investigating the use of real-world data. Harland says the pace of innovation in the oncology sector has led him to predict that in five years’ time more than half of submissions for drug approval will include a synthetic control arm, or real world data. Harland adds:
“What you are theoretically able to see is how your intervention compares with how patients are performing in the real world as opposed to an artificially good response that you might see in a clinical trial because of the placebo effect.”
Real world patient data, and its collection and validation, is now potentially robust and trustworthy enough to begin to replace placebo trials and their ethical implications.