Regulatory changes at the U.S. Food and Drug Administration are drawing industry scrutiny. The agency’s move toward a one‑adequate‑trial default for marketing authorization has raised questions about global acceptance and payer expectations. In parallel, the FDA proposed a 'plausible mechanism' framework intended to speed personalized genomic drug approvals for ultrarare conditions. The policy proposals prompted calls for clearer guidance from sponsors and highlighted tensions between accelerating access and ensuring robust evidence.