FDA's Single-Study Approval Policy: A Prescription for Legal Challenges and Industry Disruption

8 predicted events · 6 source articles analyzed · Model: claude-sonnet-4-5-20250929

The Regulatory Earthquake at FDA

The Food and Drug Administration has announced a fundamental shift in how new drugs will be approved in the United States. In a New England Journal of Medicine article published February 19, 2026, FDA Commissioner Dr. Marty Makary and deputy Dr. Vinay Prasad declared that the agency will abandon its longstanding requirement for two rigorous clinical trials, making a single study the new "default position" for drug approvals. This policy change represents one of the most significant alterations to pharmaceutical regulation in decades and sets the stage for a contentious battle over drug safety standards.

The Makary Revolution

Since arriving at the FDA in April 2025, Commissioner Makary has pursued an aggressive deregulatory agenda under the Trump administration's mandate to "slash bureaucracy" and accelerate access to medicines. As reported across all six articles, Makary's initiatives include mandating AI use by FDA staffers, implementing one-month drug assessments for medications serving "national interests," and now reducing the clinical trial requirements that have been the gold standard of drug approval since the modern FDA regulatory framework was established. The justification offered by Makary and Prasad centers on the claim that modern drug research has become "increasingly precise and scientific," making the traditional two-trial requirement obsolete. They argue there are now "powerful alternative ways to feel assured that our products help people live longer or better" without requiring duplicate confirmatory studies.

Predicted Fallout: Legal Battles and Congressional Scrutiny

**Immediate Legal Challenges** Within the next 30-60 days, we can expect patient advocacy groups and medical safety organizations to file federal lawsuits challenging the FDA's authority to unilaterally abandon the two-study standard. The legal theory will likely center on whether such a fundamental shift requires formal rulemaking procedures under the Administrative Procedure Act, rather than being implemented through policy statements and journal articles. Organizations like Public Citizen and the Center for Science in the Public Interest have historically challenged FDA deregulatory moves, and this policy provides them with a clear target. **Congressional Investigation and Hearings** Within 1-3 months, expect Congressional Democrats and potentially some Republicans to call for oversight hearings examining the safety implications of single-study approvals. The contrast noted in Articles 1, 3, and 4 between the FDA's accelerated approach to drugs while maintaining "more restrictive" policies on vaccines creates a political vulnerability. Democrats will likely frame this as a reckless experiment with public safety, while Republicans may split between supporting deregulation and concerns about pharmaceutical industry oversight. **First Controversial Approval** The most critical development will occur within 3-6 months: the first drug approval based solely on a single clinical trial that would not have met the previous two-study standard. This will serve as the flashpoint for broader public debate. If that drug shows unexpected side effects or lacks efficacy in real-world use, the political pressure to reverse the policy will become overwhelming. Conversely, if early single-study approvals prove safe and effective, Makary's position will be strengthened.

Industry Realignment and Market Dynamics

Pharmaceutical and biotechnology companies will rapidly adjust their development strategies. Within 3-6 months, we'll see: - **Smaller trials**: Companies will design studies with minimum viable sample sizes, reducing R&D costs but potentially compromising statistical power - **Expedited timelines**: Drug development cycles could shorten by 18-36 months, accelerating time-to-market - **Increased market volatility**: Biotech stocks will experience greater swings as single-study results carry more weight - **Post-market surveillance burden**: Insurance companies and healthcare systems will demand more robust real-world evidence before coverage decisions

The International Regulatory Divide

Within 6-12 months, expect a significant divergence between U.S. and international drug approval standards. The European Medicines Agency (EMA) and other major regulators are unlikely to follow the FDA's lead without substantial evidence supporting the change. This will create a two-tiered system where drugs approved in the U.S. face additional hurdles abroad, potentially undermining America's traditional leadership in pharmaceutical regulation. Foreign governments may also begin questioning the validity of U.S. clinical trial data for their own approval processes, requiring supplementary studies and effectively negating the time savings Makary promises.

The Safety Reckoning

The ultimate test of this policy will emerge within 1-2 years when post-market surveillance data accumulates. The two-trial requirement existed precisely because initial positive results sometimes fail to replicate. History provides cautionary examples: drugs like Vioxx passed initial studies but showed serious risks only after widespread use. If single-study approvals lead to increased drug withdrawals or safety warnings, the policy will be reversed amid significant political and legal consequences.

Conclusion: A High-Stakes Experiment

The FDA's single-study policy represents either a bold modernization of outdated regulations or a dangerous experiment with public health, depending on one's perspective. What's certain is that this change will not proceed smoothly or without challenge. Legal battles, Congressional oversight, and the real-world performance of drugs approved under the new standard will determine whether Makary's reforms survive beyond the current administration. The pharmaceutical industry, healthcare providers, patients, and regulators worldwide are watching closely as the United States conducts what amounts to a live experiment with its drug approval system.