FDA’s PFDD Guidance: Turning Patient Voices into Regulatory Evidence 

For the last decade, the FDA emphasized that what matters most in modern drug development is the patient’s voice. The latest guidance on Patient-Focused Drug Development (PFDD) charts a detailed roadmap for integrating patient experience data into the heart of clinical trials. For drug and biologic sponsors, this isn’t just a regulatory update—it’s a strategic … Read more

Compassionate Use vs. Right to Try: Which Path Saves More Lives? 

For patients facing terminal or debilitating illnesses, time is not on their side. The FDA’s Expanded Access (Compassionate Use) program and the more recent Right to Try (RTT) pathway both offer hope when no approved therapies remain. As the FDA’s 2025 guidance reaffirms, decades of experience with compassionate use and the emergence of RTT have … Read more

Pivoting Through Politics: A Strategic Guide for Vaccine Developers

The landscape for vaccines in the United States has undergone a fundamental change. Federal signals once aligned around promoting innovation and rapid deployment; now, they increasingly emphasize restraint, cost containment, and skepticism toward newer technologies. Vaccine developers who view this shift not as an existential threat but as a call to adapt will be best … Read more

Data Integrity in Drug Trials: What the FDA’s GCP Inspection Data Reveals

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

FDA’s Domestic Push: PreCheck and the Global Reality of Generic Drugs

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

Cosmeceuticals and Supplements: The Pseudo-Science Behind the Labels

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

ICH E20 vs. FDA 2019: Global Adaptive Trial Standards Redefined

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

Expedited FDA Approval of Stem Cell Therapies

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

FDA Two Guidances Try to Bridge Clinical Trials for Cell Therapy Products 

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