Opposition to Texas mifepristone ruling unites biotechs
Biotech Sisters leading industry response
A virtual who’s who of the biotech industry has signed onto a brief urging a federal appeals court to reverse a district court order that revoked FDA’s approval of the abortion drug mifepristone. A group of 240 Democratic members of Congress have signed a separate petition that made the same demand.
The briefs, filed Tuesday night with the Fifth Circuit Court of Appeals, seek to both temporarily stay the ruling by Judge Matthew Kacsmaryk of the U.S. District Court for the Northern District of Texas before it goes into effect on Friday, and to have it overturned.
An informal group of women biotech CEOs, the Biotech Sisterhood, has played a leading role in crafting and organizing the industry’s response.
The perception that the Texas ruling poses a threat to the life sciences industry as a whole, and the hope that its views will influence the courts, has led BIO to take a strong stance. The trade association, which has had vigorous internal debates about taking positions on controversial topics, is unified in its call for reversal of the district court ruling, BIO interim CEO Rachel King told BioCentury.
Other groups and individuals told BioCentury they are drafting briefs that will be submitted if, as is very likely, the case is appealed to the Supreme Court.
“Far from being limited to one drug, the logic of the district court’s order overturns the long-settled legal basis of FDA’s drug-approval process.”
The biopharma industry and congressional briefs argued that Kacsmaryk fundamentally misinterpreted the laws and regulations governing drug regulation and cherry-picked and misstated scientific and medical data. If it stands, his ruling would not only bar access to a safe and effective drug, but it would also destabilize the biopharmaceutical industry by creating a pathway for judicial challenges to FDA’s decisions about scientific and medical issues, putting decades of progress at risk, according to the briefs.
The industry brief echoed comments former FDA attorneys made to BioCentury over the weekend. Drafted by Eva Temkin and colleagues at King & Spalding, it asserted that Kacsmaryk’s ruling is a threat to the entire industry. Tempkin served in policy and legal positions at FDA, including as associate chief counsel during 2013-17.
“Far from being limited to one drug, the logic of the district court’s order overturns the long-settled legal basis of FDA’s drug-approval process,” according to the industry brief. “Unless stayed, the district court’s lawless opinion will empower any plaintiff to grind drug approvals to a halt, disrupting patients’ access to critical medicines.”
Some former agency lawyers, however, including Daniel Troy, who served as FDA chief counsel in 2001-04, told BioCentury that concerns about impacts of the Texas district court ruling beyond mifepristone are overblown.
In a motion filed Wednesday requesting a stay of the Texas district court decision, the Department of Justice argued both that Kacsmaryk was wrong to conclude that FDA erred in approving mifepristone and that there is no legal basis for him to second-guess FDA. “There is no basis in science or fact for plaintiffs’ repeated claims that mifepristone is unsafe when used in the manner approved by FDA. Nor is there any basis in administrative law for the district court’s unprecedented overriding of FDA’s considered scientific judgment.”
Wednesday, 69 Republican members of Congress who describe themselves as “pro-life” filed a brief supporting the Texas district court’s ruling. Their brief argued that abortion drugs are inherently unsafe and that by approving any drug in the class FDA has exceeded its authority.
Role of the sisterhood
Biopharma company executives, attorneys and investors along with current and former FDA officials spent much of the past weekend analyzing the Texas ruling and developing responses.
There is a broad consensus among biotech leaders that the Texas ruling poses a threat to the industry’s ability to develop medicines, BIO’s King told BioCentury. She issued a statement Saturday afternoon, calling the ruling “an assault on science and the FDA’s long-standing role as the authority to make decisions on the safety and efficacy of medicines.”
The ruling “sets a dangerous precedent for undermining the FDA and has consequences that extend well beyond a single drug,” King told BioCentury Wednesday.
BIO’s Executive Committee voted unanimously on Tuesday to sign onto the industry brief, King told BioCentury. It focuses on concerns about the impacts of Kacsmaryk’s ruling on drug development and regulation and does not express a position on access to abortion, she noted.
The Biotech Sisterhood, a group of women CEOs with over 100 members, catalyzed the industry’s actions.
Two of its members, Amanda Banks, an adviser to early cancer detection company Harbinger Health and former president and CEO of Blackfynn Inc., and Shehnaaz Suliman, CEO of ReCode Therapeutics Inc., wrote the first draft and helped galvanize support for an open letter that sounded the alarm, asserting that Kacsmaryk’s ruling set a “precedent for diminishing FDA’s authority over drug approvals, and in so doing, creates uncertainty for the entire biopharma industry.”
Over 400 biopharma leaders, including Albert Bourla, chairman and CEO of Pfizer Inc. (NYSE:PFE), have signed the letter since it was posted on Monday.
Two other members of the Biotech Sisterhood, Grace Colón, executive chairman of ProterixBio Inc. and Elizabeth Jeffords, president and CEO Iolyx Therapeutics and a member of BIO’s Board of Directors, worked with King & Spalding on drafting the industry amicus brief.
“This feels like a direct attack on patients.”
The issues raised by the Texas decision stand out for biotech CEOs because they jeopardize the industry’s ability to create medicines, Jeffords told BioCentury. “We spend our whole lives studying and training and growing into these roles, and we do it because we care about patients. This feels like a direct attack on patients.”
The concerns would be same if the decision had been about a drug for a condition unrelated to women’s reproductive health, Jeffords said. “We could be talking about any drug — it could have been minoxidil or methotrexate instead of mifepristone — because at the end of the day, FDA is responsible for evaluating if a drug is safe and effective. If FDA isn’t the single arbiter of what is safe and effective, we don’t know how to develop drugs and get them out to patients.”
Julia Owens, a co-founder of the Biotech Sisterhood, said that there was a broader representation of female CEOs on the letter posted Monday than on previous open letters on public policy issues. She noted that the Sisterhood has a Slack channel with about 70 female CEOs and a very active WhatsApp group.
Female biotech leaders will be taking a more active role in future public policy debates, Owens, president and CEO of Ananke Therapeutics Inc., told BioCentury. “We recognize that as a collective we have power, and we want to use that power for good, to speak out on judicial overreach that could erode FDA’s independence and put the entire industry at risk, to shine a light on neglected areas of women’s healthcare that haven’t gotten adequate funding, and so much more.”
Pfizer is the only large pharmaceutical company to sign the brief.
PhRMA has not submitted a brief; in a short blog posted Wednesday, the trade association expressed “serious concerns with any court substituting its opinion for the FDA’s expert approval decision-making.”
Other organizations that signed the brief include Gilead Sciences Inc. (NASDAQ:GILD), Nkarta Inc. (NASDAQ:NKTX) and RA Capital Management.
Individual signers include CEOs of over 100 biotech companies, along with prominent biopharma investors.
Allowing the Texas ruling to stand, they said, “would chill crucial research and development, undermine the viability of investments in this important sector, and wreak havoc on drug development and approval generally, causing widespread harm to patients, providers, and the entire pharmaceutical industry.”
Calling for deference to FDA expertise
The industry brief accused Kacsmaryk of “substituting personal conclusions—drawn from anecdotes and cherry-picked publications—for FDA’s rigorous, data-driven scientific analysis.”
It pointed out inconsistencies between assertions in his ruling and the ways in which FDA and drug developers have operated for decades, including his holding that “FDA must provide a special justification for any differences between a drug’s labeling and the conditions that existed in the drug’s clinical trials,” as well as his opinion that “head-to-head studies are necessary to demonstrate meaningful therapeutic benefit.”
In addition, the brief accused Kacsmaryk of establishing an inappropriate and impossible standard for the collection of adverse event data and “adopting an improperly narrow interpretation of what constitutes a serious or life-threatening illness and ignoring intervening amendments to the FDCA.”
The biopharma industry and congressional Democrats stressed the importance of deferring to FDA’s expertise.
“Congress intended FDA, not the courts, to serve as the expert arbiter of the safety and effectiveness of Drugs,” the industry brief said.
In their brief, congressional Democrats stated: “While Congress permitted some judicial review of FDA’s approval decisions, it did not invite federal courts to substitute their judgment for the expert conclusions of FDA’s scientists.”
The two briefs did not assert that the courts have no authority over FDA approval decisions, but they argued that the courts have and should continue to defer FDA’s expertise on scientific and medical issues.
This is an argument that could resonate with Supreme Court justices, especially Brett Kavanaugh, former FDA attorneys told BioCentury.
The Democratic members of Congress, like the biopharma industry leaders, are concerned about what they view as the broad effects of the Texas district court ruling.
“The perils of this unwarranted judicial intervention into science-based determinations can hardly be overstated,” the lawmakers’ brief stated. “Researchers, health care providers, and patients suffering from a range of medical conditions rely on the integrity and stability of the rigorous science-based drug approval process. The specter of precipitous judicial meddling therefore threatens access to life-improving and lifesaving drugs.”