The 21st Century Cures Act — Will It Take Us Back in Time?

From the New England Journal of Medicine

By Jerry Avorn, MD; Aaron S. Kesselheim, MD, JD, MPH

SUMMARY: This NEJM Perspective by Avorn and Kesselheim offers a critical analysis of the 21st Century Cures Act, then under active congressional debate. The authors argue that the legislation, despite its forward-looking name, would effectively lower FDA approval standards by authorizing the agency to rely on observational data and other forms of real-world evidence in place of rigorous randomized controlled trial data for drug approval. Avorn and Kesselheim contend that relaxing these evidentiary standards would create pathways for approval of drugs with insufficient evidence of safety and efficacy, ultimately harming patients who would receive treatments not genuinely proven to work.

BACKGROUND: The 21st Century Cures Act was proposed in Congress to modernize drug and device approval processes, framed as enabling faster access to medical innovations.

KEY FINDINGS: The legislation would give the FDA greater discretion to approve drugs based on observational analyses and other forms of real-world evidence rather than the randomized controlled trials that have historically been required for drug approval decisions.

IMPLICATIONS: Relaxing evidence standards for drug approval could allow drugs without genuine proof of safety and efficacy to reach patients, potentially causing harm and eroding the reliability of FDA approval as a signal of clinical value.