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FDA Publishes Case Studies and User Guide for Rare Disease Drug Developers

Drug development programs face unique challenges in demonstrating the safety and effectiveness of drugs for treating rare diseases. The FDA’s Accelerating Rare disease Cures (ARC) Program started the Learning and Education to...more

FDA Provides Perspective on Goals and Challenges for Regulation of Artificial Intelligence in Medical Devices, Drug Design, and...

A recent article authored by Haider J. Warraich, MD; Troy Tazbaz; and Robert M. Califf, MD in the Journal of the American Medical Association, reviews the history of artificial intelligence (AI) regulation by the U.S. Food...more

Draft Framework for Exercise of March-In Rights under Bayh-Dole Act Targets High Drug Pricing

The National Institute of Standards and Technology (NIST) released for public comment a “Draft Interagency Guidance Framework for Considering the Exercise of March In-Rights” (“March-In Framework”) on December 8, 2023...more

FDA Approves Qalsody™ for Treatment of ALS

On April 25th, 2023, the U.S. Food and Drug Administration (FDA) approved QalsodyTM (tofersen) for the treatment of amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene...more

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