Jessica Hamzelou, MIT Technology Review, August 10, 2023

Max was only a toddler when his parents noticed there was “something different” about the way he moved. He was slower than other kids his age, and he struggled to jump. He couldn’t run.

Blood tests suggested he might have a genetic disease— one that affected a key muscle protein. Max’s dad, Tao Wang, a researcher for a climate philanthropy organization, says he and his wife were initially in denial. It took them a few months to take Max for the genetic test that confirmed their fears: he had Duchenne muscular dystrophy.

Duchenne is a rare disease that tends to affect young boys. It’s progressive—those affected lose muscle function as they get older. There is no cure. Many people with the disorder require wheelchairs well before they reach their 20s. Most do not survive beyond their 30s.

Max’s diagnosis hit Wang and his wife “like a tornado,” he says. But eventually one of his doctors mentioned a clinical trial that he was eligible for. The trial was for an experimental gene therapy designed to replace the missing muscle protein with a shortened, engineered version that might help slow his decline or even reverse it. Enrolling Max in the trial was a no-brainer for Wang. “We were willing to try anything that could change the course [of the disease] and give us some hope,” he says.

That was more than two years ago. Today, Max is an active eight-year-old, says Wang. He runs, jumps, climbs stairs without difficulty, and even enjoys hiking. “He’s a totally different kid,” says Wang.

The gene therapy he received was recently considered for accelerated approval by the US Food and Drug Administration. Such approvals, reserved for therapies targeting serious conditions that lack existing treatments, require less clinical trial data than standard approvals.

While the process can work well, it doesn’t always. And in this case, the data is not particularly compelling. The drug failed a randomized clinical trial—it was found to be no better than a placebo.

Still, many affected by Duchenne are clamoring for access to the treatment. At an FDA advisory committee meeting in May set up to evaluate its merits, multiple parents of children with Duchenne pleaded with the organization to approve the drug immediately—months before the results of another clinical trial were due. On June 22, the FDA granted conditional approval for the drug for four- and five-year-old boys.

Between 2009 and 2022, 48 cancer drugs received accelerated approval to treat 66 conditions—and 15 of those approvals have since been withdrawn.

This drug isn’t the only one to have been approved on weak evidence. There has been a trend toward lowering the bar for new medicines, and it is becoming easier for people to access treatments that might not help them—and could harm them. Anecdotes appear to be overpowering evidence in decisions on drug approval. As a result, we’re ending up with some drugs that don’t work.

[….]

Expanding access

There’s a difficult balance to be reached between protecting people from the unknown effects of a new treatment and enabling access to something potentially life-saving. Trying an experimental drug could cure a person’s disease. It could also end up making no difference, or even doing harm. And if companies struggle to get funding following a bad outcome, it could delay progress in an entire research field—perhaps slowing future drug approvals.

In the US, most experimental treatments are accessed through the FDA. Starting in the 1960s and ’70s, drug manufacturers had to prove to the agency that their products actually worked, and that the benefits of taking them would outweigh any risks. “That really closed the door on patients’ being able to access drugs on a speculative basis,” says Christopher Robertson, a specialist in health law at Boston University.

It makes sense to set a high bar of evidence for new medicines. But the way you weigh risks and benefits can change when you receive a devastating diagnosis. And it wasn’t long before people with terminal illnesses started asking for access to unapproved, experimental drugs.

[….]

Today, there are lots of ways people might access experimental drugs on an individual basis. Perhaps the most obvious way is by taking part in a clinical trial. Early-stage trials typically offer low doses to healthy volunteers to make sure new drugs are safe before they are offered to people with the condition the drugs are ultimately meant to treat. Some trials are “open label,” where everyone knows who is getting what. The gold standard is trials that are randomized, placebo controlled, and blinded: some volunteers get the drug, some get the placebo, and no one—not even the doctors administering the drugs—knows who is getting what until after the results have been collected. These are the kinds of studies you need to do to tell if a drug is really going to help people.

But clinical trials aren’t an option for everyone who might want to try an unproven treatment. Trials tend to have strict criteria about who is eligible depending on their age and health status, for example. Geography and timing matter, too—a person who wants to try a certain drug might live too far from where the trial is being conducted, or might have missed the enrollment window.

Instead, such people can apply to the FDA under the organization’s expanded access program, also known as “compassionate use.” The FDA approves almost all such requests. It then comes down to the drug manufacturer to decide whether to sell the person the drug at cost (it is not allowed to make a profit), offer it for free, or deny the request altogether.

Another option is to make a request under the Right to Try Act. The law, passed in 2018, establishes a new route for people with life-threatening conditions to access experimental drugs—one that bypasses the FDA. Its introduction was viewed by many as a political stunt, given that the FDA has rarely been the barrier to getting hold of such medicines. Under Right to Try, companies still have the choice of whether or not to provide the drug to a patient.

When a patient is denied access through one of these pathways, it can make headlines. “It’s almost always the same story,” says Alison Bateman-House, an ethicist who researches access to investigational medical products at New York University’s Grossman School of Medicine. In this story, someone is fighting for access to a drug and being denied it by “cold and heartless” pharma or the FDA, she says. The story is always about “patients valiantly struggling for something that would undoubtedly help them if they could just get to it.”

But in reality, things aren’t quite so simple. When companies decide not to offer someone a drug, you can’t really blame them for making that decision, says Bateman-House. After all, the people making such requests are usually incredibly ill. If someone were to die after taking that drug, not only would it look bad, but it could also put off investors from funding further development. “If you have a case in the media where somebody gets compassionate use and then something bad happens to them, investors run away,” says Bateman-House. “It’s a business risk.”

FDA approval of a drug means it can be sold and prescribed—crucially, it’s no longer experimental. Which is why many see approval as the best way to get hold of a promising new treatment.

As part of a standard approval process, which should take 10 months or less, the FDA will ask to see clinical trial evidence that the drug is both safe and effective. Collecting this kind of evidence can be a long and expensive process. But there are shortcuts for desperate situations, such as the outbreak of covid-19 or rare and fatal diseases—and for serious diseases with few treatment options, like Duchenne.

Anecdotes vs. evidence 

Max accessed his drug through a clinical trial. The treatment, then called SRP-9001, was developed by the pharmaceutical company Sarepta and is designed to replace dystrophin, the protein missing in children with Duchenne muscular dystrophy. The protein is thought to protect muscle cells from damage when the muscles contract. Without it, muscles become damaged and start to degenerate.

The dystrophin protein has a huge genetic sequence—it’s too long for the entire thing to fit into a virus, the usual means of delivering new genetic material into a person’s body. So the team at Sarepta designed a shorter version, which they call micro-dystrophin. The code for the protein is delivered by means of a single intravenous infusion.

The company planned to develop the therapy to treat patients with Duchenne who could still walk. And it had a way to potentially fast-track the approval process.

Usually, before a drug can be approved, it will go through several clinical trials. But accelerated approval offers a shortcut for companies that can show that their drug is desperately needed, safe, and supported by compelling preliminary evidence.

For this kind of approval, drug companies don’t need to show that a treatment has improved anyone’s health—they just need to show improvement in some biomarker related to the disease (in Sarepta’s case, the levels of the micro-dystrophin protein in people’s muscle).

There’s an important proviso: the company must promise to continue studying the drug, and to provide “confirmatory trial evidence.”

This process can work well. But in recent years, it has been a “disaster,” says Diana Zuckerman, president of the National Center for Health Research, a nonprofit that assesses research on health issues. Zuckerman believes the bar of evidence for accelerated approval has been dropping. 

Many drugs approved via this process are later found ineffective. Some have even been shown to leave people worse off. For example, between 2009 and 2022, 48 cancer drugs received accelerated approval to treat 66 conditions—and 15 of those approvals have since been withdrawn.

Melfulfen was one of these. The drug was granted accelerated approval for multiple myeloma in February 2021. Just five months later, the FDA issued an alert following the release of trial results suggesting that people taking the drug had a higher risk of death. In October 2021, the company that made the drug announced it was to be taken off the market.

There are other examples. Take Makena, a treatment meant to reduce the risk of preterm birth. The drug was granted accelerated approval in 2011 on the basis of results from a small trial. Larger, later studies suggested it didn’t work after all. Earlier this year, the FDA withdrew approval for the drug. But it had already been prescribed to hundreds of thousands of people—nearly 310,000 women were given the drug between 2011 and 2020 alone.

And then there’s Aduhelm. The drug was developed as a treatment for Alzheimer’s disease. When trial data was presented to an FDA advisory committee, 10 of 11 panel members voted against approval. The 11th was uncertain. There was no convincing evidence that the drug slowed cognitive decline, the majority of the members found. “There was not any real evidence that this drug was going to help patients,” says Zuckerman.

Despite that, the FDA gave Aduhelm accelerated approval in 2021. The drug went on the market at a price of $56,000 a year. Three of the committee members resigned in response to the FDA’s approval. And in April 2022, the Centers for Medicare & Medicaid Services announced that Medicare would only cover treatment that was administered as part of a clinical trial. The case demonstrates that accelerated approval is no guarantee a drug will become easier to access.

The other important issue is cost. Before a drug is approved, people might be able to get it through expanded access—usually for free. But once the drug is approved, many people who want it will have to pay. And new treatments—especially gene therapies—don’t tend to be cheap. We’re talking hundreds of thousands, or even millions, of dollars. “No patient or families should have to pay for a drug that’s not proven to work,” says Zuckerman.

What about SRP-9001? On May 12, the FDA held an advisory committee meeting to assess whether the data supported accelerated approval. During the nine-hour virtual meeting, scientists, doctors, statisticians, ethicists, and patient advocates presented the data collected so far, and shared their opinions.

Sarepta had results from three clinical trials of the drug in boys with Duchenne. Only one of the three—involving 41 volunteers aged four to seven—was randomized, blinded, and placebo controlled.

Scientists will tell you that’s the only study you can draw conclusions from. And unfortunately, that trial did not go particularly well—by the end of 48 weeks, the children who got the drug were not doing any better than those who got a placebo.

But videos presented by parents whose children had taken the drug told a different story.

[….]

But the difference is not statistically significant for the results the trial was designed to collect. And there are some safety concerns. While most of the boys developed only “mild” side effects, like vomiting, nausea, and fever, a few experienced more serious, although temporary, problems. There were a total of nine serious complications among the 85 volunteers. One boy had heart inflammation. Another developed an immune disease that damages muscle fibers.

On top of all that, as things currently stand, receiving one gene therapy limits future gene therapy options. That’s because the virus used to deliver the therapy causes the body to mount an immune response. Many gene therapies rely on a type called adeno-associated virus, or AAV. If a more effective gene therapy that uses the same virus comes along in the coming years, those who have taken this drug won’t be able to take the newer treatment.

Despite all this, the committee voted 8–6 in favor of granting the drug an accelerated approval. Many committee members highlighted the impact of the stories and videos shared by parents like Brent Furbee.

“Now, I don’t know whether those boys got placebo or whether they got the drug, but I suspect that they got the drug,” a neurologist named Anthony Amato told the audience.

“Those videos, anecdotal as they are … are substantial evidence of effectiveness,” said committee member Donald B. Kohn, a stem-cell biologist.

The drugs don’t work?

Powerful as they are, individual experiences are just that. “If you look at the evidentiary hierarchy, anecdote is considered the lowest level of evidence,” says Bateman-House. “It’s certainly nowhere near clinical-trial-level evidence.”

This is not the way we should be approving drugs, says Zuckerman. And it’s not the first time Sarepta has had a drug approved on the basis of weak evidence, either. 

The company has already received FDA approval to sell three other drugs for Duchenne, all of them designed to skip over faulty exons—bits of DNA that code for a protein. Such drugs should allow cells to make a longer form of a protein that more closely resembles dystrophin.

The first of these “exon-skipping” drugs, Exondys 51, was granted accelerated approval in 2016—despite the fact that the clinical trial was not placebo controlled and included only 12 boys. “I’ve never seen anything like it,” says Zuckerman. She points out that the study was far too small to be able to prove the drug worked. In her view, 2016 was “a turning point” for FDA approvals based on low-quality evidence—“It was so extreme,” she says.

[….]

But for many in the scientific community, that data still needs to be confirmed. “The clinical benefit still has not been confirmed for any of the four,” Mike Singer, a clinical reviewer in the FDA’s Office of Therapeutic Products, told the advisory committee in May.

“All of them are wanted by the families, but none of them have ever been proven to work,” says Zuckerman.  

[….]

Selling hope

On June 22, just over a month after the committee meeting, the FDA approved SRP-9001, now called Elevidys. It will cost $3.2 million for the one-off treatment, before any potential discounts. For the time being, the approval is restricted to four- and five-year-olds. It was granted with a reminder to the company to complete the ongoing trials and report back on the results.

[….]

Doctors may end up agreeing that a drug—even one that is unlikely to work—is better than nothing. “In the American psyche, that is the approach that [doctors and] patients are pushed toward,” says Holly Fernandez Lynch, a bioethicist at the University of Pennsylvania. “We have all this language that you’re ‘fighting against the disease,’ and that you should try everything.”

“I can’t tell you how many FDA advisory committee meetings I’ve been to where the public-comment patients are saying something like ‘This is giving me hope,’” says Zuckerman. “Sometimes hope helps people do better. It certainly helps them feel better. And we all want hope. But in medicine, isn’t it better to have hope based on evidence rather than hope based on hype?”

A desperate decision

A drug approved on weak data might offer nothing more than false hope at a high price, Zuckerman says: “It is not fair for patients and their families to [potentially] have to go into bankruptcy for a drug that isn’t even proven to work.” 

The best way for people to access experimental treatments is still through clinical trials, says Bateman-House. Robertson, the health law expert, agrees, and adds that trials should be “bigger, faster, and more inclusive.” If a drug looks as if it’s working, perhaps companies could allow more volunteers to join the trial, for example.

Their reasoning is that people affected by devastating diseases should be protected from ineffective and possibly harmful treatments—even if they want them. Review boards assess how ethical clinical trials are before signing off on them. Participants can’t be charged for drugs they take in clinical trials. And they are carefully monitored by medical professionals during their participation.

That doesn’t mean people who are desperate for treatments are incapable of making good decisions. “They are stuck with bad choices,” says Fernandez Lynch.

To read the entire article, see here 

Ariel Wittenberg, E&E News: July 8, 2022

When Diana Zuckerman was growing up, there was dirt underneath the slides. Her children played on playgrounds placed atop sand and mulch. But, today, when she drives around her Bethesda, Md., neighborhood, Zuckerman sees [many] rubber playgrounds.

The sight concerns Zuckerman, because as president for the National Center for Health Research she has testified in front of multiple municipal and even state governments about the toxic chemicals that can lurk in rubber playground surfaces.

The chemicals can include neurotoxins like lead and other heavy metals, as well as carcinogenic chemicals like polyaromatic hydrocarbons, endocrine-disrupting chemicals like phthalates, and chemicals linked to other organ damage like volatile organic compounds and PFAS, or per- and polyfluoroalkyl substances.

“All these rubber surfaces, they can be very pretty, and when you know nothing, you think it’s great because if my child fell on it, it is so spongy and pretty and safe,” she said. “But we actually don’t believe that it’s safe from the chemical exposure perspective.”

Concerns about modern playground surfaces were first raised decades ago, when recycled tire crumb rubber became a popular playground surface. The recycled tires often contained lead and other heavy metals, and public health experts were especially concerned that using the bite-sized material on playgrounds could unnecessarily expose small children, particularly toddlers, who are prone to putting objects in their mouths.

Nowadays, many municipalities searching for new playground surfaces know enough to stay away from tire crumb rubber.

But many continue to install what’s called “pour in place” rubber, flat, spongy rubber surfaces that can contain other chemicals of concern, like polyaromatic hydrocarbons, phthalates, and volatile organic compounds. Those more solid rubber surfaces can also contain additives and binders that could harm the health of children, though the exact chemicals vary by manufacturer.

Artificial turf, long-popular on athletic fields, has also been marketed as a playground surface. Some PFAS are used to manufacture the plastic grass blades (Greenwire, Dec. 8, 2021).

Sarah Evans, an assistant professor of environmental medicine and public health at Mount Sinai Hospital in New York City, says those poured in place rubber playgrounds still pose exposure risks to kids. Children could pick up chemicals on their hands as they play and then accidentally ingest them when they eat meals or snacks at the playground.

“We are very concerned about that,” she said. “And we don’t have a good handle of what is in those products.”

Indeed, the exact risks rubberized playgrounds pose to children are unknown, in part because the federal government’s efforts to investigate the issue have been stalled by both the Trump administration and the coronavirus pandemic.

In 2016 EPA completed a report looking specifically at tire crumb rubber playgrounds meant only to explain the best ways to test such surfaces for chemicals. A second report meant to characterize potential human exposure to tire crumb rubber has never been released.

[….]

In the absence of federal action, industry has continued to say that all playground surfacing options are safe.

[….]

Evans, at Mount Sinai, said she also hears from parents who are concerned that their cities and towns have already installed rubber playgrounds.

Playing outside, even on a rubber playground, can be very important for kids’ development. So, rather than eschew the playground altogether, Evans recommends that parents be extra vigilant if their children play on rubberized surfaces and make sure hands are washed before children eat or as soon as they get home. Kids who play on crumb rubber should shake out their clothes and change when they come home.

 To read the entire article, click here.

Emily Alpert Reyes, Los Angeles Times: June 23, 2022

In Rochester, N.Y., Diane Coleman has relied on a machine to help her stay alive, but she worries that it might be slowly undermining her health.

Her ventilator was among millions of breathing devices that Philips Respironics recalled last summer over safety concerns about numerous models of its ventilators, BiPAP and CPAP machines.

The reason: Polyester-based polyurethane foam used to muffle noise in those machines could degrade, giving off chemical gases and bits of black debris that could be swallowed or inhaled.

The possible risks: headaches, dizziness, nausea, irritated eyes and airways, and “toxic or cancer-causing effects,” according to federal regulators. The Food and Drug Administration put the recall in its most serious category, involving “a reasonable probability” that a product “will cause serious adverse health consequences or death.”

Yet a year later, many patients are still awaiting replacements — and some are using the recalled machines despite those possible risks.

Coleman said her machine underwent some initial repairs, but she is seeking a new one after federal regulators sought more safety testing of the replacement foam used for such fixes. The 68-year-old, who is president and chief executive of the disability rights group Not Dead Yet, has a form of muscular dystrophy and uses her ventilator roughly 22 hours each day.

She is nervous about how it could affect her, but “it’s not like I can stop using it.”

[….]

The devices are commonly used to treat sleep apnea, a disorder in which breathing is repeatedly interrupted during sleep, which can increase the risk of heart problems and leave people dangerously drowsy during the day. The FDA has advised patients who use the recalled CPAP or BiPAP machines to talk to their doctors about whether to stop.

Tom Wilson, who administers a Facebook support group for CPAP users affected by the recall, said he has read comments from group members who say they haven’t had any communication with Philips despite registering their devices with the company as much as a year ago. Some have paid out of pocket to get other devices.

[….]

“They’ve botched the whole thing,” said Dena Young, senior counsel at Berger Montague, who said that most of the people represented by her firm had not gotten a replacement or repair. As they wait, “some of them are still using the Philips because they don’t have a choice.”

Federal investigators have also taken interest: In April, Philips said the U.S. Department of Justice had subpoenaed the company in regards to events leading to the recall.

Consumer safety advocates argue that the halting process underscores the shortcomings of the recall system, which relies heavily on private companies to inform consumers and take action.

“It isn’t actually easy for the FDA to take products off the market,” said Diana Zuckerman, president of the National Center for Health Research, a nonprofit research center that has raised concerns about the safety of medical products. “It should be a lot easier than it is.”

But the actions that the FDA has taken so far in the Philips recall also show that the agency “has more power in recalls than they usually use,” Zuckerman said.

The FDA’s Center for Devices and Radiological Health told Philips last month that it was seeking to order the company to turn in a plan that could include not only repairing and replacing the recalled devices, but also providing refunds. In a November report, an FDA investigator found that Philips had failed to start taking appropriate action years earlier when the company first became aware the foam could be breaking down. Emails showed that the company was aware of “foam degradation issues” as early as October 2015, the FDA investigator found.

Within three years, more emails indicated that Philips had gotten more complaints about crumbling foam in ventilators and said that testing had confirmed that it broke down in high heat and high humidity, but the firm “made the decision not to change the design,” according to the FDA report in November. The FDA investigator noted that dating to 2008, Philips had gotten more than 222,000 consumer complaints that included keywords such as “contaminants, particles, foam, debris, airway, particulate, airpath and black.”

[….]

This year, the FDA found that Philips’ efforts to alert patients were insufficient, concluding that many patients were probably still unaware of the health risks nine months after the recall had begun. In March, it ordered the company to notify health professionals, device distributors and users of the recalled machines after estimating that only 50% of patients and consumers who had gotten recalled CPAPs and BiPAPs within the last five years had registered with the company for a replacement.

[….]

In Philadelphia, Meghann Luczkowski likewise worried about what would happen if Miles, her 8-year-old son, were switched to a different ventilator. Miles has a rare form of dwarfism that causes a “floppy airway” that needs to be reopened with mechanical ventilation. He had briefly been put on a different ventilator in the past but couldn’t maintain safe levels of oxygen and suffered “blue spells” in which his skin changed color.

His Philips machine has been “his lifeline,” allowing him to live at home with his family. But Luczkowski said they had begun noticing black buildup when they changed a filter in the machine.

“It’s a very scary thing to hear that the machine that keeps your child alive could suddenly be the thing that’s harming them,” Luczkowski said.

[….]

Concerns have continued to mount. From April 2021 through April 2022, federal regulators have gotten more than 21,000 reports about medical issues potentially tied to the recalled devices — or malfunctions likely to cause injuries if they recurred — including 124 reports linking them to deaths. Cancer has been a stated concern: Since 2020, more than 1,100 such reports about Philips CPAP or BiPAP machines have included the words “cancer,” “tumor” or “tumour,” said Madris Kinard, chief executive of Device Events, which gathers data to track problems with medical devices.

Those medical device reports, which can be submitted by health professionals and patients as well as manufacturers, do not require verification that the device caused the injury or death; Philips stressed that submitting such reports “is not evidence that the device caused or contributed to the adverse outcome or event.”

However, Zuckerman said “it’s assumed that a lot of deaths and other serious injuries don’t get reported at all.”

In La Quinta, Matthew P. Stone counts himself as relatively lucky. The 61-year-old, who had been using a Philips CPAP machine for sleep apnea, had been able to fall back on an old device from another manufacturer.

But Stone, like others, has been galled by the way the recall has played out. At one point, Stone said, he tried to lodge a complaint with federal regulators and was referred to a Southern California number that kept cutting out before he could leave a message.

“I am so incredibly disappointed,” he said, “at the lack of advocacy by anybody involved.”

To read the entire article, click here.

Matthew Perrone, Fox13: February 08, 2022

WASHINGTON — (AP) — President Joe Biden’s pick to lead the Food and Drug Administration has stalled in the narrowly divided Senate, an unexpected setback that could delay decisions on electronic cigarettes and a raft of other high-profile health issues pending at the agency.

Biden nominated Dr. Robert Califf for the job in November after a 10-month search that critics complained left a leadership vacuum at the powerful regulatory agency, which has played a central role in the COVID-19 response effort.

Califf, a cardiologist who was an FDA commissioner under President Barack Obama, was viewed as a safe choice who could easily clear the Senate, given his 2016 confirmation by an overwhelming vote, 89-4.

But his latest Senate bid has been snared by political controversies on both the left and right that threaten to sink his nomination and leave the FDA in limbo for months — possibly even until a new Congress convenes next year.

No vote has been set on Califf’s nomination as Senate Democrats, the White House and other administration officials make a full-court press to lock up the votes needed to pass the 50-50 chamber. Former FDA officials warn that failure to move on Califf’s nomination will make it even harder to find and confirm future nominees.

“If he can’t get confirmed it bodes poorly for almost anyone else who could be nominated,” said Dr. Stephen Ostroff, who twice served as acting FDA commissioner. “What you’re seeing here is a lot of extraneous issues inserting themselves into the confirmation process and the same thing would happen to virtually anyone else nominated.”

Five Senate Democrats are opposing Califf due to his consulting work for drugmakers and the FDA’s track record of overseeing addictive painkillers that contributed to the U.S. opioid epidemic. That group includes Sens. Joe Manchin of West Virginia and Maggie Hassan of New Hampshire, both from Republican-controlled states ravaged by the epidemic.

With Sen. Ben Ray Luján of New Mexico absent and recovering from a stroke, Democrats need the support of six Republicans to confirm Califf.

[….]

The White House long assumed enough Republicans would support Califf to easily overcome any Democratic defections, given his strong support from the pharmaceutical lobby. Indeed, Califf seemed to be cruising toward confirmation after a cordial hearing before the Senate’s health committee in December, which included friendly exchanges with most of its Republican members.

But two days after his appearance the FDA eased longstanding restrictions on abortion pills that allowed women to order them through the mail. Although Califf had no role in that decision, dozens of anti-abortion groups lobbied Republicans to vote against him based on earlier changes impacting the medications while he was at the FDA.

[….]

“It is troubling to see Dr. Califf judged on issues that are a very small part of the FDA’s responsibilities,” said Steven Grossman of the Alliance for a Stronger FDA, which represents industry, patient and consumer groups that interact with the agency. “This narrow focus increases the likelihood there will be more and longer periods when FDA is without permanent leadership.”

The White House is unlikely to send another FDA nominee to Capitol Hill if Califf can’t clinch 50 votes, noted Grossman, a former HHS and Senate staffer.

In that scenario, the current acting FDA chief, Dr. Janet Woodcock, could continue leading the agency for months to come — potentially into next year. She can serve as acting commissioner as long as Califf’s nomination is pending, followed by another 210 days after it is withdrawn or expires, under federal law.

[….]

Last week Sen. Elizabeth Warren, D-Mass., announced her support for Califf after he agreed to not work for any pharmaceutical company for at least four years after leaving the FDA. Califf has recently served as a board director or adviser to more than a half-dozen drug and biotech companies, according to his ethics disclosure form.

“I think all this publicity that ‘maybe Califf isn’t going to make it’ is going to get people more focused on why they want Califf there,” said Diana Zuckerman, of the nonprofit National Center for Health Research. “I think he still has a very good chance.”

To read the entire article, click here.