Making U.S. biotech more competitive with China’s could help rare disease patients, experts say

The growth of China’s biotechnology sector has been staggering. Beijing is pumping money into the industry, backing research efforts and helping launch a new wave of labs and incubators in the country. That’s a problem for the U.S. biotech industry and also affects rare disease patients who are waiting for a cure.

Among the experts speaking out against China’s growing influence in the biotech sector is John Crowley, CEO of the lobbying group Biotechnology Innovation Organization, or BIO.

Crowley is something of a rock star in the rare disease community. His story is as incredible as it is inspiring.

When Crowley and his wife were told his two young children had Pompe disease, a fatal genetic disorder, Crowley left his job in marketing to try to find a cure. He partnered with a researcher who was working on Pompe, and started a company that eventually developed a treatment to save the lives of his children and thousands of others.

If it sounds like the plot of a movie, it is. Pulitzer Prize-winning journalist Geeta Anand wrote a book about Crowley’s story, which later became the Hollywood film “Extraordinary Measures,” starring Harrison Ford and Brendan Fraser. 

Crowley has certainly made his mark in the biotech space. He helped build two biotech companies focused on rare diseases that were later acquired by larger pharmaceuticals. Most recently, in December, BioMarin paid nearly $5 billion for Amicus Therapeutics, a company Crowley helped grow from a five-person startup in 2005 to a multibillion-dollar company when he left in 2024. Crowley left Amicus to become the CEO of BIO.

Since stepping into that post, he has become increasingly outspoken about China’s biotech sector, advocating for the U.S. to become more competitive.

“We need to reduce the reliance on Chinese biotechs,” Crowley said. “Once they are the dominant player, then they will decide who gets what medicines and technologies.”

Crowley has seen the growth of China’s biotech firsthand. “I go back to even just 10 years ago, maybe, working in China. There were maybe a couple hundred true R&D biotech companies in China. By our count, there’s over 4,000 today,” he said.

At the same time, Beijing is reducing the regulatory hurdles for drugmakers doing research in China, meaning the treatments they’re working on can get into clinical trials more quickly. That’s attractive to both major drug manufacturers and smaller researchers from around the world, who see developing drugs in China as a faster and cheaper option than in the United States. A recent article in STAT profiled the rise of one Chinese incubator, ATLATL, highlighting how it’s been able to develop relationships with clients spanning the entire drug development pipeline. 

To Crowley, who is a former naval intelligence officer, China’s rise in biotech is a threat not just to the industry he represents, but to the millions of patients who rely upon the rare disease research that comes out of American universities.

“Our research grounded in our great academic institutions [is] a remarkable strategic advantage for the United States,” Crowley said. “It’s threatened today.”

“The greatest threat comes from China and the rise of Chinese biotechnology,” Crowley said.

“We can’t let China win in biotech,” he said.

Crowley is not alone in his concerns. Former FDA Commissioner Scott Gottlieb, a member of the CNBC Cures Advisory Board, devotes a chapter in his forthcoming book, “The Miracle Century,” to China’s rise in biotech. In the book, Gottlieb lays out the case that as Beijing has simplified the regulatory approval process so breakthrough medical treatments get to market sooner, investment in those technologies has flowed from the U.S. to China. He wrote:

The shift in capital is not theoretical. It’s happening.

A September article published in Nature found that from 2020-2025, 11 of the largest pharma players committed more than $150 billion in deals for access to assets developed in Asia, primarily in China.

And data gathered for another forthcoming book, “Innovation is the Best Medicine,” by Dr. Roderick Wong, a physician and the founder and managing partner of life sciences investment firm RTW Investments, shows that from 2013-2025, China tripled its share of global clinical trial initiations. 

Political think tanks and lawmakers in the U.S. have taken notice.

In November, the nonpartisan Atlantic Council published an analysis that identified pharmaceuticals as China’s next trade weapon, likening the shift in biotech innovation to China to the offshoring of semiconductor chip manufacturing.

Spurred on by concerns about corporate espionage, access to sensitive genetic data, and memories of the supply chain bottlenecks the global medical supply industry faced after the Covid pandemic, Congress in late 2025 passed the Biosecure Act, which President Donald Trump later signed into law as part of the massive $901 billion defense spending bill.

The Biosecure Act prohibits biotech companies that receive federal funds from doing business with companies that the U.S. designates as “biotech companies of concern.” While it won’t prohibit all business U.S. biotech companies do with China, and language in the law was softened from an earlier version of the bill, the law is forcing some firms based in the U.S. to reexamine their ties to China.

But for people living with a rare disease, the issue isn’t so clear-cut. Rare diseases don’t respect borders. And parents looking for a lifesaving treatment for their child don’t care if it comes from the U.S. or China. Innovation in the rare disease space is a good thing. And in a field where there might be only two or three experts in the world on any given disease, that innovation is often the result of international collaboration. More and more frequently that innovation is coming from China.

It’s a conundrum that isn’t lost on Gottlieb, who acknowledged the innovation from Beijing is good for rare disease patients. At least in the short term. “If the end result, though, is that the fragile U.S. innovation sector gets hollowed out and we lose our own engine of innovation, that’s bad,” Gottlieb said in a text. “The priority targets of Chinese drug makers may not reflect our priority targets.”

“As China erodes other parts of our ecosystem, it could hollow out everything,” he added.

Both Gottlieb and Crowley said the real key to maintaining U.S. leadership in biotech is getting regulators to treat rare diseases, which might affect only a few hundred people, differently from those with larger patient populations. Rare disease researchers agree, arguing that a more streamlined approval process for rare disease treatments from the FDA would dramatically bring down the cost of bringing a new treatment to market in the U.S.

David Liu, a pioneer in gene editing whose lab at Harvard University and the Broad Institute is at the cutting edge of genetic research, said he’s asked the FDA to take a more lenient stance when evaluating new treatments for rare diseases. One example Liu pointed to: current guidelines for cell and gene therapies that require a company demonstrates three full-scale manufacturing runs before a treatment can get final approval.

“One full-scale manufacturing run typically costs $7 million for rare genetic disease gene editing treatments,” Liu said. “One production run can typically already treat more patients than exist in the whole world. So you’re just asking companies to throw away an extra $14 million.”

Critics of current FDA policies argue that using a different set of standards for rare disease treatments would bring down development costs and help the drugs get to the people who need them more quickly, and that it could spur a new wave of investment in the space. 

“Let’s think creatively,” Crowley said. “Don’t apply the same standards for a rare disease with 100 kids to a treatment designed for a disease with millions of people.”

“We need a system that works better,” he said.