Ramaswamy argues that the FDA should err on the side of approving promising therapies faster and then monitoring their effects after doctors start prescribing them. The agency should place “greater emphasis on post-approval surveillance for safety issues to protect patients, rather than adding time and cost to innovative development,” he said in a statement. He highlighted the FDA’s standard requirement of two trials for approval and suggested a single trial — which would generally save biotech firms time and money — would be adequate.

Ramaswamy, with billionaire Trump adviser Elon Musk, is spearheading the “Department of Government Efficiency,” an outside-government project to streamline bureaucracy and carry out “mass head-count reductions,” the two wrote in a Wednesday op-ed. Ramaswamy left Roivant’s board in February 2023 when he launched his presidential campaign but is still one of its largest shareholders.

“It’s an obvious conflict of interest,” said Diana Zuckerman, president of the nonpartisan think tank National Center for Health Research, who points out that the FDA has already lowered its standards considerably to speed novel drugs to market. “Some people might think, ‘He’s a knowledgeable person.’ He’s a knowledgeable person with a vested financial interest in what he’s saying.”

In an interview, FDA Commissioner Robert Califf said he knows and respects Ramaswamy but pushed back on some of the entrepreneur’s harshest criticisms, such as once questioning the need for the agency. “We tried that,” he said, pointing as an example to the drug thalidomide that caused birth defects in babies in the 1960s, “which is why the FDA exists in its current form.”

“Almost every additional authority at FDA has been because of a catastrophic public health event that harms people,” he said.

Califf defended the two-trial standard, particularly for new drugs when there are already effective therapies available, while emphasizing that the FDA frequently accepts less evidence for potentially life-changing therapies. As for Ramaswamy’s financial stake in biotech, Califf said, “at FDA, you’re not allowed to make decisions about industries in which you have a vested interest.” He added, “I would just say, the amount of money he has invested and what’s at stake speaks for itself.”

[….]

Of all novel drugs approved in 2020, more than half relied on a single such trial, one study found.

On Saturday, Ramaswamy praised Trump’s choices to lead the FDA and other health agencies, saying he had met with them and “it’s clear they’re serious about reducing cost & they understand innovation is a key part of the solution.” Marty Makary, whom Trump tapped to lead the FDA late Friday, has emerged as a forceful critic of the agency’s culture, faulting it in a 2021 op-ed for a “counterproductive rigidity and a refusal to adapt.”

Ramaswamy made his name as a wunderkind biotech analyst on Wall Street while earning a law degree at Yale, before striking out on his own as an entrepreneur. He told Forbes for a 2015 cover story that Roivant would be the “Berkshire Hathaway of drug development.”

[….]

Ramaswamy pulled off what was at the time, in 2015, the largest public offering in biotech, raising $360 million with the listing of Axovant, a Roivant subsidiary developing an Alzheimer’s drug. Controversy followed as a clinical trial failed to show a benefit and the stock value collapsed.

Roivant has gone on to develop several drugs that won FDA approval, but it has made a bigger splash for its savvy dealmaking.

[….]

Roivant executives have told investors its pipeline of experimental drugs could someday be worth $10 billion a year in sales. Ramaswamy holds a roughly 7 percent stake in the company, in addition to stock options to purchase millions more shares.

Trial results for two drug candidates — one to treat an autoimmune condition that causes a skin rash and muscle weakness, another for an eye inflammation disease — are expected in the second half of 2025, executives have said. The trial for the autoimmune condition is designed to support FDA approval without a second one, the company has said.

Roivant declined to comment.

Ramaswamy criticized the FDA during his failed bid for the GOP presidential nomination, a campaign where he sought to distinguish himself with a provocative style on an array of subjects. He called the agency “corrupt” and its actions “hypocritical, harmful & unconstitutional” in July 2023.

After he endorsed Trump, he continued to slam the agency. “It’s not at all obvious that we’re better off for having an FDA at all,” he said in May, dedicating an episode of his podcast to the issue. He suggested that the private market could be a better judge of what patients should trust, similar to how customers rely on J.D. Power ratings to buy cars. “The FDA has actually crowded out the rise of those alternative intermediary institutions,” he said.

[….]

Several academic scholars interviewed for this story said they disagree with Ramaswamy’s contention that conducting two major clinical trials to replicate results is unnecessary.

Sanket Dhruva, a professor at the University of California at San Francisco School of Medicine, acknowledged that clinical trials are expensive and add to the cost of developing new medicines. Still, he said, “What about costs that are passed on to the health-care system using tests and treatments that are relatively unproven?” He argued that approving less-studied drugs can give patients false hope or cause them harm and add to higher insurance premiums.

[….]

Read the full article in The Washington Post here.

From repealing climate policies to overturning guidance on the safe development of artificial intelligence (AI), Republican Donald Trump made plenty of promises during his presidential campaign that could affect scientists and science policy. But fulfilling all of his pledges won’t be easy.

Trump, now the US president-elect for a second time, will have some advantages as he re-enters the White House in January. The first time he took office, in 2017, his victory was a surprise, and many government watchers who spoke to Nature say he didn’t have a solid plan. By contrast, the Trump administration that enters office next year will be better prepared, and Trump himself is likely to face fewer checks on his power now that he has consolidated control over the Republican establishment, says Matt Dallek, a political historian at George Washington University in Washington DC who studies the evolution of the modern conservative movement.

But that doesn’t mean he will be able to do as he pleases, Dallek adds. “There’s a kind of revolutionary sweep to a lot of Trump’s promises that may collide with the messy reality of implementation.”

Here Nature talks to policy and other specialists about what might be in store on a range of science issues during a second Trump administration.

[….]

In the weeks leading up to the US election, Trump teamed up with political figure Robert F. Kennedy Jr on a platform promising to “make America healthy again” by tackling the root causes of chronic diseases, removing toxic substances from the environment and combating corporate corruption. Trump has said that he will let Kennedy, who has questioned the effectiveness of vaccines, “go wild on” health, unnerving public-health and health-policy researchers.

It remains to be seen whether Trump will appoint Kennedy to a position such as director of US Health and Human Services (HHS) — or whether the US Senate would approve such a move — but it’s clear that Kennedy will have Trump’s ear on health issues.

Georges Benjamin, the executive director of the American Public Health Association in Washington DC, worries about Kennedy’s role in the new administration because he has long cast doubt on the vaccine-approval process, threatening to undermine confidence in the jabs and cause a resurgence in illnesses such as measles. “People will get sick and die because of the confusion around vaccines, if [Kennedy and Trump] implement some of the things they verbalize,” he says.

Considering Trump’s isolationalist approach and his past comments criticizing the World Health Organization, support for global health is also likely to be “greatly scaled back” during Trump’s second term, says Ezekiel Emanuel, a bioethicist and long-time observer of the US biomedical funding landscape at the University of Pennsylvania in Philadelphia. The United States is “the key player” in the funding of global-health initiatives, says Emanuel. This includes, for instance, a programme that aims to end the global AIDS epidemic. So it’s “hard to be optimistic” about the future, he adds.

During Trump’s first term, his administration barred people from half a dozen countries that it said were “compromised by terrorism” from entering the United States and implemented an anti-espionage programme called the China Initiative that led to the arrests of a number of scientists of Chinese heritage. Although the Biden administration overturned the travel ban and ended the China Initiative, federal officials have continued efforts to guard against foreign interference in US research.

Like the first Trump administration, the new one will probably clamp down on granting visas to foreign researchers and students from some countries, says Jennifer Steele, an education-policy researcher at American University in Washington DC. Policies that make it harder for international and US researchers to meet would also make it harder for new scientific collaborations to arise, says Caroline Wagner, a specialist in science, technology and international affairs at the Ohio State University in Columbus. That’s because such partnerships are fuelled by face-to-face contact. “Collaborations don’t begin with people just e-mailing each other across the miles,” she says.

But there might be one bright spot on the collaboration front, at least for US–China partnerships. Denis Simon, a non-resident fellow at the Quincy Institute for Responsible Statecraft, a foreign-policy think tank in Washington DC, thinks that a crucial pact governing US–China scientific cooperation that has been expired for the past year is likely to be signed by the Biden administration before Trump’s second inauguration in January. Although a renewed agreement would probably be more limited in scope owing to increased US–China tensions, its existence would show that “both governments give their blessing” to collaborations, Simon says.

Read the full article in Nature here.

Michelle Tarver, the new leader of the Food and Drug Administration’s medical device center, faces significant challenges ahead.

Tarver is taking over as director of the Center for Devices and Radiological Health as the regulator grapples with questions around artificial intelligence in medical devices, racial bias in pulse oximeters, concerns about the recall process after Philips pulled millions of respiratory machines, and scrutiny of its former leader’s potential conflicts of interest.

Patient advocates, who have called for stronger device standards and faster, more transparent recalls, see an opportunity for change with a new director. Meanwhile, medtech companies hope to build on predecessor Jeff Shuren’s legacy of making the U.S. the go-to market for new medical devices.

“She is in a position to show a commitment to public health and safety, and possibly build that trust and confidence in the public for medical devices,” said Maria Gmitro, founder and president of the Breast Implant Safety Alliance, a nonprofit advocacy group.

Tarver’s leadership

Tarver was named permanent director last week after spending more than 15 years with CDRH. Some of her accomplishments include helping build the first patient engagement advisory committee at the FDA, developing patient-reported outcome measures, and conducting trials and surveys to capture patient preferences.

Both patient groups and device companies commended Tarver’s experience and focus on patients, describing her as thoughtful and approachable.

“She let it be known that she cared very much about patients understanding the risks and benefits of medical products, and including patients and consumer advocates in the process,” said Diana Zuckerman, president of the National Center for Health Research, a nonprofit think tank.

Madris Kinard, CEO of Device Events, a company that makes software to track adverse event reports and recalls, said Tarver has been receptive to suggestions in public meetings. For example, Tarver recently helped make certain demographic data available in the FDA’s adverse events database, which can be helpful in learning about off-label use in pediatrics.

Andrew Fish, CEO of the Medical Device Innovation Consortium, a public-private group founded in 2012, said Tarver is a respected leader in the agency and has been involved in efforts around patient input, patient engagement and clinical trial diversity.

[….]

House Democrats have called for an investigation into Shuren’s tenure after a New York Times report found Shuren’s wife, Allison Shuren, worked as legal counsel for medtech companies while he led CDRH. The FDA told MedTech Dive in August it found no evidence that Shuren violated the criminal conflict of interest statute or that regulatory decisions were affected by Allison Shuren’s employment.

One of the concerns raised in the report was that Allison Shuren’s firm worked on an acquisition of Allergan as the FDA asked the pharmaceutical company to recall certain models of breast implants for the risk of developing a type of lymphoma.

“It’s concerning reading about the timeline of the Allergan breast implant recall and Dr. Shuren’s wife being a part of the law group that was involved in the sale of Allergan to Abbvie,” Gmitro said. “It doesn’t build a lot of trust.”

The Department of Health and Human Services’ Office of Inspector General said it does not have an update to share publicly on whether an inquiry will be opened into Shuren’s tenure.

Michael Abrams, senior health researcher at Public Citizen, said an investigation is warranted to “advise and alert” Tarver to existing and future conflicts.

Future priorities

Tarver outlined some of CDRH’s priorities during a keynote at Advamed’s The Medtech Conference in mid-October. She spoke about the importance of listening to patients, based on her experience as an ophthalmologist.

“It ties directly into the vision of our center,” said Tarver, who still works with patients. “We put people in the U.S. first, and we want them to have access to high quality, safe and effective medical devices of public health importance first in the world.”

She called out three strategic priorities for the center: promoting a modern and diverse workforce, making the organization more agile and resilient to be prepared for future challenges, and advancing health equity.

Tarver will also lead CDRH through the next round of user fee discussions in 2027. The fees made up more than a third of the agency’s budget in 2022, according to HHS.

[….]

Patient groups, on the other hand, see the user fee process as giving industry too much of a voice in the FDA’s activities. Abrams would like the FDA to get more funding from Congress and less from industry.

Advocates have also called for more input into the negotiations between the FDA and industry leading up to the agreement. Those discussions are not open to the public and have been criticized for a lack of transparency. The FDA is required to hold meetings with patient groups and outside experts during user fee negotiations, but the discussions are separate from talks with industry.

“None of the rest of us are allowed in. … No patient groups, no consumer groups, no academic researchers, nobody else,” Zuckerman said. “It’s all behind closed doors, and we’re all shut out of the process.”

[….]

Focus on device safety

Patient advocates also called for stronger premarket review of devices. The majority currently go through the FDA’s 510(k) pathway, a less rigorous process where devices need to demonstrate they’re substantially equivalent to predicate devices, or products already on the market. The FDA has proposed changes in draft guidance, such as recommending devices not be based on a predicate that was recalled for design reasons.

Those changes are not enough, Zuckerman said. Higher-risk devices that go through more scrutiny under the FDA’s premarket review process often still submit data from single-arm trials without a control group, Zuckerman said.

Zuckerman added that it doesn’t make sense for the highest-risk medical devices to have lower evidence standards than low-risk prescription drugs. 

“Something for tummy aches shouldn’t be going through a more rigorous process than a cardiac implant,” Zuckerman said. 

Patient groups also called for stronger oversight after devices are on the market, as well as faster, more transparent recalls. They also called for broader adoption of unique device identifiers to ensure recalls are efficient and for less reliance on voluntary recalls.

“It’s great if we’re getting innovative devices to market faster. I understand people wanting to try new devices, especially if there are no other alternatives,” said Device Events’ Kinard. “But the FDA needs to be just as quick to act and recall a device when they find a problem.”

Read the full article on MedTech Dive here.