FDA Imitates Backwards Europe on Drug Regulations

 Europe shows what can happen when countries let bureaucratic procedures take precedence over the well-being of people suffering from rare diseases. 

But it's not just Europe. For more than a year, families have staged protests in Georgia, which sits at the crossroads of Asia and Europe, where one in three children with Duchenne muscular dystrophy (DMD) dies annually due to a lack of access to widely available medical treatments. Duchenne is a progressive, debilitating disease that results in mobility loss, progressive heart failure, and respiratory collapse. Most patients are wheelchair-bound by the time they’re teenagers. Few live beyond their early 20s. 

Poland is witnessing a similar debate surrounding Duchenne, centering on the Polish government’s refusal to fund Duchenne therapies, including a steroid treatment whose cost, according to one estimate, amounts to roughly 0.06% of that country’s healthcare budget.

We like to think we’re immune to such abject policy failures, but they’re a warning of what could go wrong in the United States when public health agencies like the Food and Drug Administration (FDA) act less like a patient ally and more like a barrier to rare disease treatment. 

FDA procedures have created a nightmarishly complex regulatory environment, characterized by touch-and-go delays and painfully slow approval timelines. Recent rejections of promising new rare disease treatments have also angered and worried patient advocacy groups, who understand that people with terminal conditions cannot wait for bureaucrats to make their minds up about what drugs they can take. Similarly, major overhauls affecting surrogate endpoints and stricter criteria for accelerated approval suggest regulation could tighten even further, causing more delays in access to life-saving therapies for patients who have no time to wait.

Last year, the FDA restricted the use of Elevidys, an emerging gene therapy that could slow the progression of Duchenne. Trial data reveal promising results, including improved muscle function and improved quality of life in most patients who received this therapy. Citing safety concerns, the FDA placed clinical trials on hold and revoked its use for non-ambulatory patients while arbitrarily and confusingly approving it for ambulatory patients aged four and older.

It’s not the only time recently that the FDA has stopped the release of a treatment that could help people suffering from severe, debilitating diseases. It made headlines earlier this year when it halted the approval of a promising gene therapy for Huntington’s disease, even though data from the treatment’s manufacturers showed that it slowed progression of the disease by 75 percent. The FDA later determined that the comparison to an external control group wasn’t good enough, so it demanded a full Phase 3 trial, which would require control-group participants to be anesthetized and undergo a fake brain-injection procedure -- a concept that Senator Ron Johnson (R-WI) called “bureaucratic idiocy.” 

Stories like these highlight the Kafkaesque absurdity undergirding our regulatory agencies, often framed as a debate between speed and safety, recklessness and prudence -- a false dichotomy. No one is arguing for eliminating patient safety standards. America’s historically strict drug approval process -- where the average time to go from development to FDA approval is between 10 and 15 years -- reflects the fact that the entire world benefits from our well-studied medical products. 

But there’s a lot of daylight between safety and dysfunctional bureaucratic machinery that stifles progress and innovation -- and worse, degrades health outcomes for terminal patients with few options. 

The goal is smarter regulation, not deregulation.

This is why the Right to Try Act, signed into law by President Donald Trump, is a step in the right direction. It helps ensure that drug approval timelines align with the urgency of the diseases they treat by allowing terminally ill patients to use experimental treatments that haven’t received full FDA approval. It balances theoretical risks with the very real dangers posed by letting a disease go untreated. The most successful healthcare outcomes are borne on the shoulders of a free market that embraces competition, provides clear and consistent regulatory standards, rewards innovation, and gives patients a voice.

The balancing act between bureaucratic procedure and patient outcome isn’t merely a story about medicine. It’s fundamentally a story about institutional trust and how that trust is intrinsically bound to citizens’ experiences.

The United States remains a world medical leader not only because our country embraces innovation and entrepreneurship, but because we put people first. We lose that standing whenever capricious bureaucratic processes and suppressive government decisions take precedence over human lives and American voices. But we stand as a global leader anytime we empower patients to have a say in their own futures.

Mike Feuz is an economist and holds a Master’s Degree in Economics from George Mason University. He writes frequently on healthcare policy and regulatory reform.

https://www.americanthinker.com/articles/2026/06/the_fda_imitates_backwards_europe_on_drug_regulations.html