Studies conducted by Oregon State University indicate that the U.S. Food and Drug Administration (FDA) is increasingly greenlighting new pharmaceutical drugs on the basis of singular clinical trials, while also reducing the level of public information about these trials. This raises questions about the rigor and openness of the drug approval mechanism.
While medical experts acknowledge the imperative of swiftly bringing potentially life-saving medications to market, particularly for severe conditions like cancer, they argue that these findings underscore the necessity for enhanced transparency in drug approval processes.
According to Veronica Irvin, a study co-author and associate professor in Oregon State University’s College of Health, pharmaceutical firms, for drugs subjected to multiple clinical trials, are typically mandated to disclose only the outcomes of two selected trials. This leaves unanswered questions regarding the rationale for selecting those particular two trials and the outcomes of any remaining trials.
Irvin clarified, “The intent is not to insist on an excess of studies for cancer drugs; rather, the complete set of results or trials that have been concluded should be disclosed. While this wouldn’t necessarily impede their approval, it would provide a fuller understanding.”
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Impact of the 21st Century Cures Act
The researchers focused on developments following the enactment of the 21st Century Cures Act in 2016, which was designed to hasten the approval of new medicines, thereby providing patients more prompt access to potentially life-saving treatments. Under this legislation, the FDA has lessened certain standards, allowing for the quicker approval of drugs intended for priority health conditions, such as cancer. Additionally, the FDA has shifted its emphasis away from randomized clinical trials, permitting the use of surrogate markers in specific instances as an alternative to clinical outcomes.
Veronica Irvin provided an example, noting that while it might take extensive time to establish through long-term clinical trials whether a medication lowers heart attack risks, the approval process can be accelerated by examining surrogate markers like blood pressure. Nonetheless, she emphasized that a reduction in blood pressure is not a guaranteed indicator of decreased mortality risk from heart ailments.
Comparative Study of Drug Approvals
The studies, which were published in peer-reviewed journals, evaluated FDA approvals of new medications in 2017 and 2022 to assess the number of trials employed for each drug’s evaluation prior to approval. Researchers also examined the extent to which results of drug trials were made available on ClinicalTrials.gov, a publicly accessible database administered by the National Institutes of Health.
In 2022, 24 of the 37 approved drugs (approximately 65%) received approval based on a single study. In contrast, prior to the enactment of the Cures Act in 2016, only four of 20 new drugs (20%) were approved on the basis of a single trial.
Public Availability of Clinical Trial Data
Veronica Irvin pointed out that the absence of publicly posted results does not necessarily imply that the FDA is not privy to the complete set of results. However, these results are not made available to the general public until they are publicly posted.
Irvin emphasized the intended transparency of the FDA’s process, stating that the objective of ClinicalTrials.gov is to offer a platform for the lay public to access both the trials and their results in an understandable format.
She concluded, “It is essential for both physicians and patients to have access to the complete set of data.”
References
The lead authors of the studies are Robert Kaplan from Stanford University and co-author Amanda Koong, a medical student at the McGovern School of Medicine in Texas. The studies are published in the Journal of the American Medical Association Network Open and Health Affairs Scholar.
Frequently Asked Questions (FAQs) about FDA Drug Approval Process
What is the main concern regarding the FDA’s drug approval process?
The primary concern is that the U.S. Food and Drug Administration (FDA) is increasingly approving new pharmaceutical drugs based on just one clinical trial. This has led to reduced transparency and questions about the thoroughness of the drug approval process.
How has the 21st Century Cures Act affected drug approvals?
The 21st Century Cures Act, enacted in 2016, was designed to accelerate the approval of new medicines. As a result, the FDA relaxed some of its standards, permitting faster approvals for drugs aimed at treating priority health conditions such as cancer. It also allowed the use of surrogate markers in specific instances instead of relying solely on clinical outcomes.
What do experts say about the need for transparency in drug approvals?
Experts argue that while fast-tracking potentially life-saving medications is crucial, there is also a need for greater transparency in how these drugs are approved. They advocate for the disclosure of complete sets of results or trials that have been concluded to provide a fuller understanding of a drug’s efficacy and safety.
What percentage of drugs was approved based on a single study in 2022?
In 2022, approximately 65% of the 37 approved drugs were greenlighted by the FDA based on a single clinical trial.
What was the situation before the 21st Century Cures Act?
Before the enactment of the 21st Century Cures Act in 2016, only 20% of 20 new drugs were approved based on a single trial. This suggests that the Act has significantly impacted the rate of single-trial-based approvals.
Is the FDA the only entity that has access to full trial results?
No, the FDA is not necessarily the only entity with access to full trial results. While not all results are publicly disclosed, they are made available to the FDA. The issue lies in the fact that these results are not always made public, thereby reducing transparency.
What is the role of ClinicalTrials.gov?
ClinicalTrials.gov is a publicly accessible database maintained by the National Institutes of Health. It is intended to provide a platform for the general public to access information about clinical trials and their results, enhancing transparency and public understanding.
What do researchers recommend for improving the drug approval process?
Researchers recommend a more transparent approach, where pharmaceutical companies disclose all completed trials and their results. This would provide physicians and patients with a more complete picture, aiding in more informed healthcare decisions.
More about FDA Drug Approval Process
- Journal of the American Medical Association Network Open
- Health Affairs Scholar
- FDA’s Drug Approval Process
- 21st Century Cures Act
- ClinicalTrials.gov
- National Institutes of Health
- Oregon State University’s College of Health
- McGovern School of Medicine in Texas
- Stanford University School of Medicine
8 comments
Wow, this is kinda concerning. Didnt know the FDA was approving drugs with so little testing. What’s the point of trials if they’re gonna fast track everything?
After reading this, the 21st Century Cures Act seems more like a double-edged sword. Good in intent but maybe not in execution?
65% based on one study? thats nuts. Especially when we’re talking about medications that could have long-term side effects.
Never trusted the FDA much and this just adds fuel to the fire. you’d think with something as serious as medication, there’d be full transparency.
I’m all for getting life-saving drugs out there, but shouldn’t we know all the details? Feels like they’re cutting corners and it’s us who might suffer.
Interesting article. If pharmaceutical companies are conducting multiple studies, why only show one or two? Makes you wonder what they’re hiding.
ClinicalTrials.gov should be everyones go-to before even thinking of taking a new medication. Clearly, we can’t rely on the FDA to tell us everything.
Transparency is key in healthcare. The more we know the better we can make decisions about our own health. This is eye-opening.