Under the new leadership of Marti Makri, the FDA should pay more attention to the important role Real world clinical data – Such as health insurance database – In the ongoing evaluation of drug safety and efficacy, beyond the original clinical trials presented for FDA approval.
Issues with drug safety and efficacy can be remembered in the clinical testing phase, assured after a drug goes into the market. The FDA has already started recognizing this problem, for example, Deployment FDA Sentinel initiative, which is advanced In a main component of the agency’s developed security monitoring system.
The clinical trials presented to the FDA during the drug approval process include methodically rigid, usually limited participant variety, short follow -up periods and controlled settings with small sample sizes. Real world clinical data provides supplementary insight into regular clinical practice by capturing data from more diverse population.
For example, pregnant women, patients with cum-free medical conditions, low groups (eg, elderly and minority), or other drugs are often excluded from clinical trials to separate specific variables. But this feature does not have this feature in real -world clinical medicine: doctors treat patients under all their complexity and dirty conditions.
What exactly does it matter how a new drug performs tightly in controlled experimental conditions, but how the drug is used and how it performs in the practice of everyday medicine. Real world data can detect rare adverse events or long -term security issues that were not clear in original tests.
The health insurance database, with large -scale longitudinal data, may be particularly valuable to identify signs of drug -related risks. The FDA’s regulatory activity does not end with the drug approval process, but involves the ongoing vigilance to assess problems that initially missed the clinical testing phase.
Critics will argue that double-blind placebo-controlled tests provide more reliable information than the real world clinical data, especially about the question of cause, whether effectiveness or unfavorable effects. But the realities on the ground are more complex, which is why we should use all available evidence in assessing drug safety and efficacy. Each study design has its own strength and weaknesses, including random controlled tests.
Reply in many ways in research studies design to control potentially confused factors is only one, and it only works when the relevant results end with a large number of subjects in hand.
Only the fact that random controlled tests presented for FDA approval are operated and funded by pharmaceutical companies seeking approval for their product, they do not automatically invaluate their results, but of course we should consume the possibility of functioning and reporting prejudice. While the real -world clinical data also requires probe, and it comes with its set of limitations, advanced statistical methods such as professional scores matching and instrumental variable analysis can confuse and reduce prejudice, and can improve the ability to conclude the cause from such data.
To refer to just an example of a real-world clinical data study, which corrected the incorrect findings of the Clinical Testing Data presented to FDA, my colleagues Jamie Bryan-Hall and Ryan Anderson at morality and public policy policy center. Safety study Mifepristone’s pill used in chemical abortion. It represents the biggest study of the abortion pills so far, which uses data from an all-Pear Insurance Claim Database that included 865,727 prescribed mifepristone abortion from 2017 to 2023.
The study found that about 11 percent of women experienced sepsis, infection, bleeding, or another serious adverse event, which after mifepristone within 45 days. In this study, the clinical trials reported on the real -world rate drug label of severe adverse incidents after mifepristone miscarriage were at least 22 times the summary figure of “less than 0.5 percent” and presented to FDA.
This is not the first time that serious adverse events were discovered only after approved for the use of a drug. The discovery of the drug Vioxx (Rofecoxib) explicit adverse effects-especially its association’s heart attack and stroke-vertical world’s clinical data highlighted the important role of post-mortling monitoring in identifying the safety signs left out in clinical trials. Vioxx was approved by the FDA for pain management and inflammation in 1999, but in 2004 it was withdrawn from the market due to serious side effects in original clinical trials, nominated for relatively small, controlled populations, which were healthy, small, and less comorbidities in comparison to vioxx post-upering. In addition, short periods of testing stopped assessment of long -term adverse effects such as heart events.
It was a proper study in which electronic health records were used (for example, Kaiser Permanent And Treatment) And adverse event reporting systems of FDA such as pharmacovigilance systems allowed researchers to analyze results in millions of patients taking Vioxx. Thousands of people would have died unnecessarily from heart attacks and strokes, as a result of these studies, and as a result of removing Vioxx from the market.
Similarly, the real -world clinical figures informed the FDA’s decision to add a black box warning to SSRI antidepressants for adolescents and young adults.
Physicians practice therapy in the complex and dirty real world, not in artificially pure conditions of a controlled clinical testing. While the preliminary studies presented for FDA approval have their qualifications, they also have underlying limitations. These should be complemented – and where necessary, correct – by real world clinical data. To fulfill its mandate, the FDA cannot ignore the importance of the ongoing security and efficacy testing after a drug goes to the market, even after the early work of drug approval is over. Life depends on this work of ongoing vigilance.
Aaron Kheriyati, MD, is a companion and director of the Biothics and American Democracy Program at the Ethics and Public Policy Center.
