For more than a century, drug development has relied on animal studies as a gatekeeper for safety and toxicity. But in 2025, does that scientific justification still hold water? The original rationale — that animals are a safe proxy for humans — is deeply flawed, and institutional inertia (especially regulatory mandates) is the main reason … Read more
The FDA relies on Good Clinical Practice (GCP) inspections to evaluate regulatory compliance and verify data integrity, but unlike the US-based clinical sites, foreign GCP inspections are rare. A recent retrospective analysis provides a comprehensive look at the FDA’s GCP inspections conducted between fiscal years (FY) 2016 and 2018, shedding light on the agency’s operational … Read more
The FDA has launched its PreCheck program and a new ANDA Prioritization pilot, ostensibly to address supply chain vulnerability by promoting domestic pharmaceutical manufacturing. Driven by the narrative that the globalization of drug production compromises U.S. safety and security, these programs offer expedited reviews for new U.S. sites and generic drugs made entirely with domestic … Read more
Supplement labels may look scientific, but many are nothing more than marketing spin cloaked in pseudo-medical jargon. Terms like “supports heart health” or “boosts brain function” trick consumers into believing supplements prevent diseases—without a shred of clinical proof. Add in industry-invented buzzwords like “cosmeceuticals,” and you have a billion-dollar deception thriving on FDA loopholes. Walk … Read more
Over the last two decades, adaptive trial designs have transitioned from being an avant-garde concept to a regulatory norm. The main challenge with adaptive trial design has been global acceptance of such designs in regions outside the US. The newly released ICH Guidance document harmonizes across regions the principles originally proposed in the FDA Guidance … Read more
Regenerative medicine therapies, such as stem cell therapies, have the potential to transform the future of healthcare, but navigating the FDA approval process can seem daunting. A new FDA Guidance Document repackages established expedited programs to describe a path to faster approval of regenerative therapies by the FDA. The guidance reaffirms that while stem cell … Read more
Cell and gene therapies (CGTs) are revolutionizing treatment for patients, particularly for those with rare and life-threatening diseases. Yet, the path from development to approval is challenging because rare disease clinical trials often involve very small patient populations and pose unique logistical challenges. This week, two FDA guidance documents outline how available innovative trial designs … Read more
Externally controlled trials (ECTs) are used when randomized controlled trials (RCTs) are impractical or unethical, particularly in rare diseases and oncology. But new analysis reveals that many ECTs fall short on methodological rigor, threatening the reliability of their findings. Unless the field embraces standardized practices, ECTs risk undermining the very evidence base they aim to … Read more
The FDA has long championed the irreplaceable value of on-site, in-person inspections for drug manufacturing oversight. Yet its latest guidance acknowledges that remote assessments can, in some contexts, be a more suitable and efficient regulatory tool. This balanced, risk-based approach reflects both the Agency’s pragmatic adaptation to modern realities. A final FDA guidance document released … Read more
The release of ICH E6(R3) marks a transformative shift in global clinical trial standards. By embracing risk-based quality management, modern technologies, and flexible trial designs, this guideline redefines how research should be conducted while safeguarding participants and ensuring reliable results. For sponsors, investigators, and regulators alike, E6(R3) represents a milestone in harmonizing innovation with patient … Read more
