In closing arguments, Peter Stein, MD, director of the Office of New Drugs in the Food and Drug Administration’s Center for Drug Evaluation and Research, acknowledged clinicians’ arguments about the need for an effective drug to reduce the incidence of preterm birth — a major cause of infant death in the United States. United State. He said the agency agrees with doctors who testified during three days of hearings about the urgency of such a drug, but only if the data and science back it up — which is not the case for McCain.
“Hope is a reason to continue to search for effective options, whether we find them here or elsewhere,” he said. “Hope is not a reason to take a drug that has not been proven effective. Or keep it on the market.”
The recommendations of the panel of independent advisors are non-binding, although the agency usually follows its advice. Withdrawing a drug from the market is a very unusual move.
The three-day hearing was emotional for both members of the audience, as well as members of the Obstetric, Reproductive and Urological Drugs Advisory Committee.
Several health groups have supported keeping Makena on the market while further studies are conducted, fearing that its withdrawal will deepen health disparities. Martha Nolan, senior policy advisor at Healthy Women, a nonprofit health group focused on helping women make informed decisions about their care, said.
Members of the panel, made up of maternal health experts, neonatologists, statisticians and other experts, spoke of the difficulty of making their decision.
“I am so disappointed… I wish we weren’t sitting here today,” said one of the members. Another expressed his “deep sadness” at McKenna’s massive trial, which turned out to be of no benefit.
Reproductive endocrinologist Esther Eisenberg supported the drug’s withdrawal, “but I’m in a lot of struggle. That’s a very difficult question.”
Cassandra Henderson, a maternal-fetal medicine specialist in New York City who was the only panelist who argued that the large clinical trial showed promise for some patient subgroups who voted to keep the drug on the market, said she was concerned about the low representation. of minority women on trial, because “we know that race is a kind of alternative to racism and all structural inequalities.”
Drug maker Covis Pharma and its backers argued that the study may have lost its benefits in the high-risk population of the United States because the participants were largely Eastern European and only 7 percent were black. In a filing with the US Food and Drug Administration, the drug company described the latest trial as “flawed,” not only because of its racial demographics, but also because the population was low-risk and women had access to national health care systems that are vastly different from the complex micro-system in United State.
Rajav Shari, Covis’ chief innovation officer, testified that the company was willing to work with the agency to limit the use of Makena to only a “high-risk target group” and would also agree to stop active promotion of the drug.
He has termed this a “practical approach” that would enable individual clinicians, in consultation with their patients, to make decisions about whether or not the use of the drug might be beneficial.
Shari said Covis is committed to conducting additional studies to address questions about the drug’s potential risks and benefits, emphasizing that reducing preterm birth is a public health priority and an area of unmet need in drug development.
“We are not suggesting that race biologically differentiates between patients,” he said on Wednesday. “At the same time, it is well-documented that preterm birth disproportionately affects black and other minority women in the United States. These and other social determinants of risk are factors in identifying populations at greater risk where McKenna is likely to be effective.”
But Joseph Alukal, MD, a urologist and director of men’s health at Columbia/New York Presbyterian, suggested the racial inequality argument “suggests the drug is effective and indicates the drug is safe” when we don’t actually have an answer for it.
He’s “sensitive to the disparity issues that have been raised,” said Mark Hodak, a neonatologist at the University of Florida College of Medicine. However, he said allowing Makena to remain in the market was inappropriate and would lead to “complete regulatory chaos”.
Makena was approved by the Food and Drug Administration in 2011 under the Expedited Approval Program for Drugs for Serious Conditions for which there are no cures. The drugmakers are then required to conduct studies confirming the drug’s benefits to continue selling the drug. But the controversy over the effectiveness of Makena’s drug more than a decade after its approval highlights the complexities of that program, highlighting how the agency could take years to pull the drug off the market even if officials believe it is ineffective.
In McKenna’s case, the FDA’s Center for Drug Evaluation and Research proposed its removal from the market in October 2020 — a move that followed 9-7 votes by an expert advisory panel a year earlier to remove it from the market based on disappointing results from a large confirmatory study. But regulatory requirements, as well as the pandemic, have slowed the process.
Stein of the Food and Drug Administration argued that leaving Makena on the market for narrow use would “overturn the intent of the accelerated course.” He argued, “In the absence of evidence of efficacy, we are only left with the risk. The balance between benefits and risks for Makena is not appropriate.”
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