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FDA Forming Consortium to Study Gene Biomarkers Linked to Serious Adverse Events

NEW YORK (GenomeWeb News) — The US Food and Drug Administration is creating a consortium with members of the pharmaceutical industry and academia that aims to observe how genetic biomarkers contribute to serious adverse events, according to an NIH report and people familiar with the project.
The FDA’s decision to form the initiative, called the Serious Adverse Events Consortium, comes at a time when a chorus of critics increasingly blames the agency for not doing enough to ensure the safety of marketed drugs.
But at least one official from a big pharma company believes the consortium will have little effect on tracking and avoiding SAEs, and said his company decided not to join the group because its goal “is not only daunting but unlikely.”
It is not immediately clear when the agency plans to unveil the consortium, which would be part of the Office of Critical Path Programs, or what industry or academic members will participate in it. According to Nadine Cohen, head of pharmacogenetics at Johnson & Johnson Pharmaceutical Researchand Development, the initiative is in a “very preliminary” stage.
Cohen said she is familiar with the consortium but would not say whether her company plans to join it. However, Johnson & Johnson could be on the short list because it believes genetic biomarkers can play a role in identifying and mitigating adverse events.
In drug discovery, we are … constantly looking for markers of efficacy and safety as part of our duediligence research and development," Cohen was quoted as saying in a British Medical Journal article in March.
At least one pharma official isn’t convinced that the consortium will succeed. “The notion is that we will find a genomic marker that will predict” who will suffer an SAE, according to the official, who asked to remain anonymous because he is not part of the group. “The problem is that it will take thousands and thousands of patients to screen in order to validate a particular marker.”
This official said SAEs, which include hepatotoxicity, rabdomyalysis, and QT prolongation, among others, typically occur in less than one in 1,000 patients and are “inherently unpredictable either by pre-clinical or clinical development. “And because of the rarity of the event, the prospect of doing that is not only daunting but unlikely,” the official said. “People just haven’t done the math.”
This official said his company decided not to participate in the consortium for these reasons.
According to the official, the FDA is attracted to the consortium because “they need to be more aggressive in signal detection, in getting these [SAE] reports in and figuring out what’s happening” so they can determine what steps to take.
The official said the agency is becoming “more and more conservative” to the point where “if you have one, two, three cases of severe hepatotoxicity out of millions who have been treated, [then] you’re getting a Black Box warning.”
The official said hepatotoxicity has been “the single-most common reason to pull a drug off the market.”
The Serious Adverse Events Consortium isn’t the only federal initiative aimed at improving drug safety. The Critical Path is also linking the Association of Clinical Research Organizations with the Clinical Data Interchange Standards Consortium to form the Clinical Data Acquisition Standards Harmonization project. This new group would be charged with “developing sample case report forms for reporting adverse events,” according to a National Institutes of Health summary of a Roadmap steering committee meeting that took place last December.
According to the summary, the Office of Critical Path Programs, which will oversee the Serious Adverse Events Consortium, directs other similar initiatives. The summary said the office is a “cross-cutting coordination and harmonization [center] of all the centers within the FDA. Areas of focus in this effort are bioinformatics and data standards, biomarkers, establishing public-private partnerships, and developing guidance and regulations.”
Rachel Behrman, director of the Office of Critical Path Programs, spoke at the steering committee meeting. According to the meeting summary, she said other alliances under its aegis include the ECG Warehouse, which is a partnership between the FDA and electrocardiography product maker Mortara Instruments; the Oncology Biomarker Qualification Initiative, which pairs the FDA with the National Cancer Institute and the Centers for Medicaid and Medicare Services; the Biomarker Consortium, which brings together the FDA, the NIH, the Pharmaceutical Research and Manufacturers of America; and a Memorandum of Understanding between the FDA and NCI to create the Federal Investigator Registry of Biomedical Information Research Data, or FIREBIRD, “which will enable clinical investigators, NCI, FDA, and industry entities sponsoring clinical trials of investigational drugs to manage clinical investigator information electronically in a fully secure manner," according to the MOU.
Behrman did not respond to multiple telephone calls seeking comment for this article. An FDA spokesperson was not immediately able to respond.

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