If you’re faced with a serious disease, you better hope it’s not a rare one.

After an often tortuous path to diagnosis, people with rare diseases are likely to find that good treatment options don’t exist and none is on the horizon. Many of these conditions are poorly understood, and conducting studies in tiny patient populations can be practically impossible. Most drugs won’t pan out, and those that do will have little demand and financial payoff, no matter how beneficial they are. Drug companies usually direct their attention elsewhere.

Recognizing these challenges, policymakers have worked since the 1980s to encourage rare-disease drug development. They’ve earmarked federal research funding, established dedicated programs at the Food and Drug Administration, extended protection against competition to help secure profits, and awarded lucrative incentives for rare pediatric disease drugs. Still, 95% of rare diseases, which affect an estimated 30 million Americans, lack any approved therapy.

Some blame the FDA. They say the agency is too rigid, imposing impossible requirements and demanding unreasonable proof of effectiveness and safety for rare disease drugs. Some suggest patients would be better off if the FDA just got out of the way — not only for rare disease treatments, but also more broadly.

The current administration now seems determined to do just that, at least for products that fit the “Make America Healthy Again” agenda, like stem cells and psychedelics (never mind recent intrusions on COVID vaccines and abortion drugs).

Large swaths of FDA experts have been DOGE’d or otherwise forced out of the agency. Commissioner Martin Makary has proposed approving drugs based just on their scientific plausibility, while the agency’s chief medical and scientific officer pledged to “take action at the first sign of promise for rare diseases” — potentially making treatments available far sooner, even though many drugs that look promising at the start turn out not to work.

Just last month, the FDA announced approval of a drug the commissioner claimed would help “hundreds of thousands of kids” with autism, not based on a clinical trial but on published case reports of 40 patients with a potentially related condition — alarmingly and unprecedentedly accepting anecdotes as evidence of efficacy.

The call to disarm the FDA is coming from inside the house.

Criticism of the agency’s gatekeeping is certainly not new, but critics are especially vocal now. Banking on expectations that the Trump administration would break through perceived red tape, they are calling on the White House and new FDA leadership to approve rare disease drugs with far less attention to safety and effectiveness than to keeping companies financially interested in developing rare disease treatments and bringing them to market.

It can be reasonable to assess rare disease drugs differently as usual market-driven incentives often fail to yield treatment options. That’s why the FDA has been remarkably flexible about these approvals for decades. Sure, the FDA sometimes says no — but what if the drugs it rejects just weren’t any good?

Ideally, you want to see a drug’s effectiveness replicated in at least two studies to be confident in the results. For drugs approved to treat common diseases (outside of cancer), that replication is typical. But only 13% of approved rare disease drugs (again outside of cancer) relied on more than one robust clinical trial to show they work. Recent FDA policy has made clear that this single-study approach will be the rule for rare disease drugs and potentially for other conditions going forward.

Rare disease drugs are also increasingly granted “accelerated approval,” a pathway that allows drugs for serious diseases to be approved based on predicted rather than proven benefit. Companies must complete required studies after approval, but the FDA has allowed drugs to stay on the market even if these studies fail. This happened for a recent gene therapy for Duchenne muscular dystrophy, a therapy that was later linked to patient deaths.

Even outside accelerated approval, the agency sometimes approves drugs that miss the targets chosen in advance to prove the drug works. A recent study found this happens in 1 of 10 approvals, about half of which were for rare disease.

Despite this flexibility, criticism continues. Rather than taking FDA’s refusal to approve a drug as a critical warning sign, these decisions are often met with the assumption that the FDA must be wrong.

Take the agency’s recent action on elamipretide for Barth syndrome, an ultra-rare, life-threatening genetic disorder characterized by heart, muscle and immune system abnormalities that affects about 300 patients globally.

Given the tiny patient population, Stealth BioTherapeutics, the company developing elamipretide, conducted a trial in just 12 people, which failed to show the drug worked. Some patients continued in an extended version of the trial and appeared to perform better on tests of walking distance and fatigue. However, the FDA reasonably worried this might be because of patients’ awareness that they were receiving the drug, leading to a placebo effect.

After reviewing the evidence, the FDA issued a letter in May 2025 refusing to approve elamipretide. Recognizing the need for flexibility, however, the agency left the door open to approval based on a new, unverified measure of patient benefit: improved muscle strength in the knee. In September, following substantial public criticism from patient advocates and members of Congress, the FDA granted the drug accelerated approval. Stealth will now have to complete another study to see whether the treatment really helps patients — but even if that study fails, the agency may not withdraw approval.

Even if elamipretide fails to pan out, one might wonder what harm lies in just approving it. Maybe it can provide some hope to patients who have nothing else, while encouraging companies not to abandon rare diseases.

The problem is this: Those who criticize the FDA for setting the bar too high shrug off trial data that fail to show benefit, arguing that it doesn’t mean the drug is ineffective. But it is very hard to prove that a drug doesn’t work. If that were the standard, FDA reviewers should just close up shop, leaving a universal “approved” stamp for any drug that appears not to kill patients.

When it’s working well, FDA approval signals to patients (and their doctors and insurers) “this drug has been shown to work” — or at least “this drug has been shown very likely to work.” If FDA approval means anything less, such as “this drug has not been shown not to work,” it fails to serve patients, leaving them no better off than if they were browsing unproven dietary supplements on Amazon. They might even be worse off, if duped into relying on FDA approval as a meaningful indicator of benefit.

Rare disease patients, like all patients, should have drugs that work. The burden must be on companies to prove that their drugs do. Shifting or altering that burden by changing FDA approval standards won’t help, but other changes might. For example, policymakers could improve existing legal approaches that allow patients with serious diseases to try investigational drugs that aren’t yet approved. The federal government could also increase support for the research needed to understand, diagnose and treat rare disease, helping companies focus on the most promising targets and minimizing failures. Unfortunately, the Trump administration’s ongoing decimation of federal health agencies and research instead sets back rare disease science.

Public trust in authorities like the FDA is already depleted. Demanding that the agency greenlight more rare disease drugs, evidence be damned, will make this problem worse — and likely won’t leave rare disease patients better off. Rather than blaming the FDA, the policymakers, companies and patient advocates should be doing all they can to get better drugs in front of the agency’s reviewers.

Holly Fernandez Lynch is a senior fellow at the Leonard Davis Institute of Health Economics at the University of Pennsylvania, where she is also an associate professor of medical ethics and law. Reshma Ramachandran is a family medicine physician and assistant professor at Yale School of Medicine, where she co-directs the Yale Collaboration for Regulatory Rigor, Integrity and Transparency.

Tavares Strachan loves to blur the lines that separate art, science and historical reckoning — as well as past, present and future.

A native of Nassau, Bahamas, he once carved out a 4.5-ton block of ice in the Alaskan Arctic, had it FedEx’ed to the island nation and displayed it in a solar-powered freezer — an extreme commentary on climate change, displacement and interconnectedness.

Strachan became the first Bahamian to visit the North Pole to understand the harsh conditions that in 1909 greeted Matthew Henson — the Black explorer who accompanied Cmdr. Robert Peary on polar expeditions and was likely the first human to ever stand on top of the world. His feat went unacknowledged for years because he was Black.

In another project, Strachan honored America’s first Black astronaut, Robert Henry Lawrence Jr. He died in a plane crash while training a test pilot in 1968 before he could take part in any space mission. So Strachan sent a replica of an artwork inspired by Lawrence into orbit on a SpaceX rocket.

Now a collection of Strachan’s work is on view at the Los Angeles County Museum of Art for his first museum show in the city, titled “The Day Tomorrow Began.”

The multisensory exhibition showcases Strachan’s ability to translate his lifelong passion for scientific inquiry and boundary-pushing adventures into pieces that make you question everything you thought you understood about human progress — all while forcing you to see how Black achievements so easily get written out of the history books.

“The pieces of this particular exhibition that I think may hit people are when they start making connections on their own — the synapses start to fire,” the 45-year-old artist said during a recent tour of his show. “You start to tie in, for example, the relationships between this polar explorer Matthew Henson and Robert Henry Lawrence. And then you start to think about the earlier explorers who left the African continent and this pioneering spirit that is a part of what it means to be African that is oftentimes not articulated or discussed.”

On view until March 29, “The Day Tomorrow Begins” is both whimsical and serious. There is a lot to take in: illustrations and diagrams, displays of traditional African hairstyles, mohair collages made jointly with South African weavers, commemorative ceramics, a field of rice grass whose strawlike scent is meant to pique olfactory memories.

A glassmaker, Strachan covers a wall with two neon signs that spell out quotes by James Baldwin, whose words appear upright, and Mark Twain, whose words appear upside down — in a nod both to the wonders of chemistry and the power of the pen to dissect issues of identity.

In another soaring room, dramatic bronze sculptures flip the script on the triumphs of Western civilization — placing the focus on the oppressed.

One piece depicts the moon, its surface pockmarked by craters. Resting on its north pole is a bust of Henson. Protruding from the moon’s south pole is an upside down bust of Peary.

Fitting for an artist who once put himself through the physical rigors of cosmonaut training, the show feels like a dialogue between opposing forces — boundlessness and constraint, presence and absence.

Strachan, who lives in New York, said his fascination with science and its hidden histories started while growing up as a curious and “very stubborn” kid in Nassau.

He was about 12 when his family bought the first set of encyclopedias that he can remember. But something was off: few entries featured notable figures who looked like him.

Tavares Strachan’s show at LACMA includes a room of whimsical ceramic sculptures from his “Inner Elder” series that are positioned in a field of fragrant rice grass.

(Allen J. Schaben / Los Angeles Times)

“I think this was my first peek into social science,” Strachan said. “Obviously, you can’t collect all of this material without making decisions — you’re deciding what’s seen and what’s unseen. It started to get me going on these questions of visibility and invisibility.”

Strachan started to ponder his place in the ecosystem — and the universe beyond.

Tired of wearing the clothes that his seamstress mother made for him, Strachan raised money for shopping by working on a fishing boat, spending weeks at a time at sea.

At night, far from shore, more stars flickered than he’d ever noticed, and he was awestruck by the way phosphorescent creatures set the waves aglow.

Strachan learned to navigate by tracking the movements of celestial bodies and hunt fish by reading the currents, building on the ancient knowledge his elders handed down.

Up until then, his 700-island archipelago had felt like the center of the world. Now his curiosity was a universe of possibilities.

But while his mind raced across the heavens, Strachan couldn’t stop thinking about the voids that made written accounts of pioneering feats and extraordinary voyages on Earth seem incomplete.

Charles Darwin is a household name, but how many people know that the world’s most famous naturalist learned taxidermy from John Edmonstone, a formerly enslaved Black Briton who owned a bird-stuffing shop in Edinburgh, Scotland?

Strachan was in his 20s when he stumbled upon Henson’s achievement.

“Science — that’s the place where knowledge is produced, and meaning,” exhibition curator Diana Nawi said. “How do we know what we know? I think that’s fundamentally what [Strachan]’s asking.”

Nawi said Strachan’s projects resist the “calcification of history,” which she describes as the process in which a dominant group reinforces narratives that glorify themselves while ignoring or actively erasing the contributions of others.

“Histories are tools of power,” Nawi said. “How do you take that power for different people and different ideas, but also, how do you undo the singular concept of that power?”

In Tavares Strachan’s LACMA show, stories of racial uplift and groundbreaking achievements take center stage. This illuminated piece uses glass, argon and electrodes to evoke the spirit of America’s first Black astronaut, Robert Henry Lawrence Jr., who died in a plane crash before he could take part in a space mission.

(Allen J. Schaben / Los Angeles Times)

“In 2020 … it was the toppling of monuments, for instance,” Nawi said, referring to the decommissioning of statues that celebrate the Confederacy in the wake of George Floyd’s murder.

“History is ripe for the retelling and the retaking,” Nawi said.

That sentiment has many meanings in the current moment.

Some of those removed monuments have been transformed into new artworks that will be on view in the show “Monuments,” at MOCA Geffen and the Brick from Oct. 23 to May 3. At the same time, the Trump administration has attacked public displays of factual but less-flattering aspects of U.S. history as too “woke.”

Strachan’s work also suggests there is power in acts of poetic justice.

Inside one darkened room in the show, a life-size, glass “portrait” of the astronaut Lawrence appears to be levitating as if free from gravitational forces. His frame is lit from within, head-to-toe, by argon trapped in electrified tubes shaped to resemble the human circulatory system, making his soul visible.

America’s first Black astronaut never got to transcend Earth’s atmosphere.

Tavares Strachan poses with his installation “Six Thousand Years,” which is made up of 2,000 panels from his “Encyclopedia of Invisibility.” The leather-bound tome contains 17,000 entries that the artist wrote to bring attention to little-known facts and Black trailblazers.

(Allen J. Schaben / Los Angeles Times)

By fashioning this portrait of Lawrence to capture his spirit, and by shooting a satellite honoring Lawrence into orbit, Strachan wanted to metaphorically help him achieve that goal.

“For me it’s important to ensure that when someone has done something incredible, that the level of storytelling is aligned with the nature of the act, hence the audacity of putting an object into space and trying to get his energy back into the cosmos,” Strachan said.

“But also, it’s about having a 10-year-old walk in and be just amazed by the technical feat of creating this glass object.”

A third case of a potentially more severe strain of mpox was confirmed in Los Angeles County on Friday, leading officials to investigate the possibility it is starting to spread locally.

The trio of cases, all reported publicly this week, represents the first time this particular type of mpox, known as “Clade I,” has been found in the United States among people who had no history of traveling overseas to high-risk areas.

The first case, reported publicly on Tuesday, involved a resident of Long Beach. The second and third cases, reported Thursday and Friday, occurred among other Los Angeles County residents. All three patients were hospitalized but are now recovering at home.

“At this time, no clear link has been identified between the cases,” the L.A. County Department of Public Health said.

Nonetheless, “the confirmation of a third case with no travel history raises concerns about possible local spread in Los Angeles County,” Dr. Muntu Davis, the L.A. County health officer, said in a statement. “We’re working closely with our partners to identify potential sources and understand how this potentially more serious type of the mpox virus may be spreading.”

“While the overall risk of … exposure to the public remains low, we are taking this very seriously,” Long Beach Mayor Rex Richardson said in a statement. “This underscores the importance of continued surveillance, early response and vaccination.”

This type of mpox is different from the one that spawned a global outbreak in 2022, which is known as “Clade II.”

Clade I is potentially even more concerning, however, because it may cause more severe illness and spread more easily, “including through close personal contact,” such as massage or cuddling, in addition to sex, the L.A. County Department of Public Health said.

The California Department of Public Health said last year that Clade I has historically caused more severe illness than Clade II, but added that “recent infections from Clade I mpox may not be as clinically severe as in previous outbreaks, especially when cases have access to quality medical care.”

Mpox, formerly known as monkeypox, is primarily spread through close, intimate contact, such as through body fluids, sores, shared bedding or shared clothing, as well as kissing, coughing and sneezing, health officials say.

Tell-tale symptoms “include rash or unusual sores that look like pimples or pus-filled blisters on the face, body and genitals, fever, chills, headache, muscle aches or swelling of lymph nodes,” the L.A. County Department of Public Health said. Other symptoms can include a sore throat.

“Anyone who develops an unexplained rash or lesions should avoid sex and intimate contact and seek medical evaluation as soon as possible,” the Long Beach Department of Health and Human Services said.

People should get tested if they have symptoms, officials said. Those who have symptoms should also avoid sex or close contact.

Clade II mpox generally causes mild-to-moderate illness and has been circulating at low levels throughout the United States since 2022, according to the L.A. County Department of Public Health.

There have been 118 cases of Clade II mpox reported to the L.A. County Department of Public Health so far this year.

Before this week, there had been a total of six cases of Clade I mpox in the U.S. — all among people who had recently traveled to areas where this type of mpox is circulating, namely central and eastern Africa. None of those cases was linked to each other, according to the Centers for Disease Control and Prevention.

The CDC says there have been more than 40,000 cases of Clade I mpox in central and eastern Africa.

In the Democratic Republic of the Congo, one of the countries with confirmed cases, multiple modes of Clade I mpox transmission have been documented, including “contact with infected dead or live wild animals” and “household contact often involving crowded households,” in addition to sexual contact, according to the CDC.

The risk to the general U.S. population in the U.S. from Clade I mpox is considered “low,” the CDC says. The agency classifies the risk to gay and bisexual men who have sex with more than one partner as “low to moderate.”

Travel-associated cases of Clade I mpox have also been found in a number of other regions globally, including Asia, Australia, Europe and South America.

The first Clade I mpox case in the U.S. was reported 11 months ago — in someone in California who had traveled to Africa and received care in San Mateo County, according to the CDC and California Department of Public Health. That person had mild illness, the San Mateo County health department said at the time.

Most people who are infected get better within two to four weeks, the Long Beach Department of Health and Human Services said, “but antiviral treatments may be considered for individuals with or at risk of developing severe illness.”

The two-dose Jynneos vaccine is also available to help prevent the spread of mpox.

Those who only got one dose can get their second doses “no matter how long it’s been since the first dose,” the L.A. County Department of Public Health said.

Vaccines are widely available, and can be found at pharmacies such as Walgreens and CVS. People can look up locations to get vaccinated through the vaccine’s manufacturer, Bavarian Nordic. The L.A. County Department of Public Health also maintains a list of vaccination sites.

The vaccine is available to people at higher risk for the illness, including those who were exposed to an infected individual over the last two weeks.

Also eligible for vaccination are gay and bisexual people and other men who have sex with men; transgender, nonbinary or gender-diverse people; people with HIV; people who are eligible or are taking medicine to prevent getting HIV from sex or injection drug use; people traveling to sub-Saharan Africa or areas with Clade I mpox outbreaks; people who plan to attend a commercial sex event or venue, such as a sex club or bathhouse; people who have a sex partner at higher risk for getting infected; and anyone else who requests mpox vaccination.

Officials recommend people with occupational risks for infection, such as certain lab workers, also get vaccinated.