A pillar of drug safety crumbles: FDA drops two-study rule

• The FDA will now require only one clinical trial for most new drug approvals, a major shift from its decades-old two-study standard.
• Agency leaders argue modern science makes a second confirmatory trial unnecessary, promising faster access to new medicines.
• Critics warn this reduces evidence quality and increases uncertainty about drug effectiveness and safety for patients.
• The policy contrasts with the FDA’s recent stricter demands for vaccines and gene therapies, highlighting inconsistent standards.
• The change is the latest in a series of deregulatory moves by the current FDA leadership aimed at speeding drug development.

In a move that fundamentally reshapes the landscape of pharmaceutical regulation, the U.S. Food and Drug Administration announced it will abandon its long-standing requirement for two rigorous clinical trials to approve new drugs. On February 18, FDA Commissioner Dr. Marty Makary and a top deputy declared that a single trial will now be the agency’s “new default standard.” This seismic shift, aimed at accelerating drug availability, has ignited a fierce debate about scientific integrity, patient safety and the very role of a regulator historically tasked with ensuring that medicines are both effective and safe before they reach the public.

The end of a decades-old safeguard

For over half a century, the FDA’s two-study standard served as a critical scientific checkpoint. Established in the early 1960s, the requirement was rooted in a simple, prudent principle: replication. A second, confirmatory trial was essential to verify that the promising results of a first study were not a statistical fluke or a product of trial-specific conditions. This redundancy was a cornerstone of evidence-based medicine, designed to protect patients from false hopes and potential harm. While the FDA had increasingly made exceptions for rare or fatal diseases since the 1990s, the dual-study mandate remained the bedrock for most new drug evaluations—until now.

A new era of “precision” or premature approval?

Commissioner Makary and his colleagues frame the change as a necessary evolution. They argue that modern biological science and “precise” drug discovery provide such clear mechanistic understanding that a single, well-designed trial, coupled with post-market surveillance, is sufficient. They contend this will slash development costs and time, spurring a “surge in drug development.” Former FDA drug director Dr. Janet Woodcock endorsed the logic, stating that with greater biological understanding, “we don’t need to do two trials all the time.”

However, this optimistic view is met with profound skepticism from many quarters of the medical community. Critics see it not as an evolution, but a dangerous dilution of evidence standards. The core purpose of a second trial is to ensure reliability; biological plausibility is not a substitute for reproducible clinical outcomes. This policy, they warn, will increase the risk that drugs with marginal benefits, exaggerated results, or unforeseen safety issues will enter the market, leaving patients and doctors with greater uncertainty.

A glaring double standard in regulation

The timing and context of this deregulation raise additional red flags. It comes amidst a series of controversial actions by the current FDA leadership, including mandating the use of artificial intelligence in reviews and offering expedited one-month assessments for drugs deemed in the “national interest.” Furthermore, the permissive new standard for drugs starkly contrasts with the agency’s recent approach to other products. Just last week, the same officials initially rejected Moderna’s mRNA flu vaccine for insufficient data, demanding an additional study—highlighting an inconsistent and seemingly arbitrary application of scientific rigor. This duality fuels accusations that the agency is prioritizing pharmaceutical industry speed over methodical, uniform safety standards.

Historical echoes and eroding trust

This policy shift cannot be viewed in a vacuum. It echoes a long-standing pattern of regulatory capture and industry influence. Since the 1992 Prescription Drug User Fee Act, the FDA has derived a significant portion of its budget from fees paid by the very companies it regulates, creating a problematic financial dependency. Historical precedent, from the revolving door of officials between the agency and industry to the suppression of unfavorable trial data, provides a sobering backdrop. As noted by former FDA Commissioner Dr. Herbert Ley decades ago, “The thing that bugs me is that people think the FDA is protecting them. It isn’t.” The move to weaken a foundational evidence requirement appears to validate such enduring concerns, suggesting that commercial and political pressures are overriding cautious, patient-centric science.

A gamble with public health

The FDA’s decision to dismantle a key protective barrier represents a monumental gamble with public health. While the promise of faster cures is politically potent and commercially attractive, it risks inundating the market with poorly vetted treatments, undermining the practice of evidence-based medicine, and further eroding public trust. In an era already rife with medical misinformation, the agency’s role as a rigorous, impartial arbiter of science is more vital than ever. By lowering the bar for approval, the FDA is not merely cutting red tape; it is shifting the burden of proof from drugmakers to patients, who will now bear the ultimate risk of therapies approved on thinner, less reliable evidence. The real-world trial of these drugs will increasingly begin not in controlled research settings, but in the medicine cabinets and bloodstreams of the American public.

Sources for this article include:

TheEpochTimes.com

MedPageToday.com

SBSun.com