What happened in one judge’s courtroom in Texas could have drastic effects for the United States’ entire drug approval process, experts warn.
US District Judge Matthew Kacsmaryk’s ruling that suspended the US Food and Drug Administration’s approval of the medication abortion drug mifepristone was an unprecedented one, the first time a court has bypassed the federal system set up to determine what drugs should be allowed on the market.
Regardless of whether the ruling – or a part of it – is ultimately allowed to stand, legal scholars, scientists and drugmakers are concerned that the decision could start a trend of drugs being targeted in courts, creating a chilling effect on drug development in the US and hurting patients in the process.
Vaccines, including the Covid-19 shots, antidepressants and psychotropic medicines could be at risk, some said.
“Well, one does not want to be Chicken Little,” former FDA Commissioner Dr. Jane Henney said Wednesday, but “I can’t imagine that it wouldn’t have implications for other products.
“The approval process will be at risk, and it’s not just an approval process that patients rely on and providers rely on, it’s one that has been considered the gold standard, really, for the world,” said Henney, who was the head of the FDA when mifepristone was approved.
Since the dawn of the 20th century, the FDA has had the sole authority in the United States to regulate drugs. In 1906, the federal government created the agency to enforce the Pure Food and Drug Act, which was instituted to ensure that medicine, food and cosmetics were safe.
Over the years, that authority became more defined.
After elixir of sulfanilamide, a drug used to treat streptococcal infections, killed 107 people in 1937, Congress created the Food, Drug, and Cosmetic Act. Signed into law in 1938 by President Franklin D. Roosevelt, it required manufacturers to conduct pharmacological studies to prove that their drugs were safe before they could be sold or advertised. In 1962, drug manufacturers were also required to prove to the FDA that their products were effective.
Modern drug approval in the US is a careful and conscientious process. Before any drug goes to market, there are countless hours of research, the work and expertise of multiple scientists, and several layers of oversight for approval.
Until now, the courts have been deferential to the FDA’s process and have never overturned an FDA decision on the grounds that the agency misjudged the science, said William Schultz, a former deputy commissioner at the FDA and former general counsel for the Department of Health and Human Services.
“Any FDA drug approval involves hundreds of judgments by the agency. And if a court feels free just to kind of take a fresh look at each of those, there’s a chance that a court will find one of those FDA judgments wrong,” Schultz said in an online discussion Monday about the impact of the Texas court’s ruling that was hosted by Protect Our Care, an organization that advocates for equitable and affordable health care.
An ‘assault on science’
Hundreds of well-known biotech and pharmaceutical company leaders, concerned about the effects of Kacsmaryk’s ruling on other drug approvals, signed an open letter Monday in support of the FDA’s authority “to approve and regulate safe, effective medicines for every American.”
The letter also advocated a reversal of the mifepristone decision from a judge with “no scientific training,” saying it “set a precedent for diminishing FDA’s authority over drug approvals, and in so doing, creates uncertainty for the entire biopharma industry.”
In a separate statement, the biotech industry group BIO’s interim president and CEO, Rachel King, emphasized the “dangerous precedent” the decision sets.
“The preliminary ruling by a federal judge in Texas is an assault on science and the FDA’s long-standing role as the authority to make decisions on the safety and efficacy of medicines. For a court to invalidate the approval of a drug that was reviewed and approved more than two decades ago is without precedent. As legal scholars have noted, the courts do not have the medical expertise to make these types of scientific determinations,” King said.
The main lobbying group for the pharmaceutical industry, PhRMA, criticized Kacsmaryk’s ruling as undermining the regulatory process.
“PhRMA has serious concerns with any court substituting its opinion for the FDA’s expert approval decision-making,” said James C. Stansel, the association’s executive vice president, general counsel and corporate secretary. Stansel added that such a decision could have a “chilling effect on the research and development ecosystem.”
The pharmacutical sector is a huge part of the American economy. Of the world’s 25 largest phamacutical companies, 10 are based in the US, and most of the others have a large base of operations in the country.
Often, the US market is the first to get access to new drugs, but that could change if lawsuits undermine the regulatory integrity of the FDA process, said Susan Lee, partner in the law firm Goodwin’s Life Sciences group and Life Sciences Regulatory & Compliance practice, who works with companies to get drugs approved by the FDA.
“If there do tend to be more lawsuits like this, I wonder if there might be a little bit of a tendency to not always look at the US as the first market,” Lee said. “Some manufacturers may say ‘we’d rather go to Europe, where we’re not going to be sued on a jurisdiction-by-jurisdiction basis.’ “
Lee also wonders whether manufacturers will abandon efforts to develop drugs that could be considered unappealing to some, such as those that help women’s health or work to prevent HIV.
“I think there are just certain sectors that are already kind of thinking about whether they might also have a target on their back. I’ve definitely heard that discussed,” Lee said.
What could be next?
The groups at the heart of the Texas case have not disclosed any further plans regarding lawsuits over medications, but experts say they are already hearing concern.
“I’ve already been getting questions from lawmakers and other people about ‘could the Covid vaccine be next?’ or other things that may have stigma around it,” said Dr. Kristyn Brandi, an ob/gyn and abortion provider in New Jersey and a spokesperson for the American College of Obstetricians and Gynecologists.
The Covid-19 vaccines have been thoroughly tested and found to be safe and effective, but they’re the subject of conspiracy theories and misunderstanding about how mRNA vaccines were tested. Beliefs that the vaccines were tested on recently harvested aborted fetal cells made some people decidedly anti-vaccine.
Dr. Lynn R. Goldman, professor and dean of the Milken Institute School of Public Health at George Washington University, is also concerned that mRNA vaccines could be targeted soon.
“There might be people who disagree with some of the technologies that are used by vaccine makers, like the mRNA vaccines, but feeling uncomfortable about a technology is not the same thing as identifying that there is risk,” she said in the Protect Our Care conversation.
Members of the LGBTQ+ community may also be vulnerable, experts say, as activists could target puberty blockers or hormones used in gender-affirming therapy.
“I don’t like to do slippery slope, but I’m also very worried about things like gender-affirming care, since there’s already been so many laws about that recently in other states,” Brandi said.
There is political pressure against other vaccines, antidepressants and psychotropic medicines, among others, former FDA Commissioner Margaret Hamburg and former Principal Deputy Commissioner Joshua Sharfstein wrote in an editorial published Thursday in the journal Science.
“If judges begin to dictate the terms of medication access, then others will seek to use ideology and influence to advance their agendas,” they warn.
A ‘serious moment’ for doctors and patients
Goldman said that any legal decision that could undermine the FDA drug approval process would ultimately hurt the doctors who prescribe them and the people who use them.
Doctors don’t have time to vet all the studies used to prove that a drug is safe and effective, so they rely on the FDA for this work, she said. Court interference could confuse this process.
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“I think that this is, for doctors, an incredibly serious moment, because up to now, we have been able to trust that an approval by the FDA is a science-based decision and that we can say that if the FDA has approved a drug, that it is safe for us to use,” Goldman said.
A lack of confidence in the drug approval process will ultimately hurt people far beyond the most recent decision, Protect Our Care Chair Leslie Dach says.
“Confidence that the FDA can do its work is essential for clinicians and patients who depend on it in its decision-making for matters of life and death,” Dach said.