Tag: risk-benefit assessment]

Navigating Compassionate Use Programs from a Regulatory Perspective

August 20, 2025

Navigating Compassionate Use Programs from a Regulatory Perspective How to Navigate Compassionate Use Programs in Rare Disease Regulation Introduction: The Role of Compassionate Use in Rare Disease Access For patients with rare and life-threatening diseases, access to investigational therapies before formal marketing authorization can be life-saving. Compassionate Use—also known as Expanded Access—provides a regulated pathway…

Rare and Orphan Disease Trials, Regulatory Frameworks

Building Ethical Frameworks for Emergency Access to Rare Therapies

August 18, 2025

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Building Ethical Frameworks for Emergency Access to Rare Therapies Ethical Frameworks for Emergency Access to Rare Disease Therapies The Ethical Imperative of Emergency Access For patients with rare and life-threatening conditions, time is often the most precious resource. When no approved therapies exist, compassionate use or expanded access to investigational products becomes the only hope….

Ethics & Patient Engagement, Rare and Orphan Disease Trials

How Patient Registries Support Regulatory Decision-Making

July 8, 2025

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How Patient Registries Support Regulatory Decision-Making Leveraging Patient Registries for Regulatory Decision-Making: A Practical Guide Patient registries have emerged as critical tools in the regulatory landscape, providing real-world data (RWD) to support evidence-based decisions on drug approvals, safety monitoring, and post-marketing commitments. As pharmaceutical professionals and clinical trial experts navigate evolving regulatory expectations, understanding how…

Real-World Evidence (RWE) and Observational Studies, Registry Studies