By Patrizia Cavazzoni MD & Peter Marks MD PhD – Through the Hub, we plan to foster a community at the FDA for open dialogue and knowledge sharing to identify new approaches to drug and biologic development and overcome hurdles that have traditionally impeded progress for rare disease treatments.
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By Patrizia Cavazzoni MD & Peter Marks MD PhD – An estimated 10,000+ rare diseases affect more than 30 million people – approximately one out of every 10 people – in the U.S., and about half of these people are children. Many rare conditions are life threatening, and most do not have approved treatments.
By Hilary Marston MD MPH & Kevin Bugin PhD – Breakthroughs in science and technology are advancing innovation in more areas of medicine, providing novel treatment options for patients. The scientific community has a deepening understanding of disease and human biology, and of the enormous potential afforded through new technologies.
By Robert Califf – What we now call real-world data (RWD) have been used for years in medical product development to inform study design and endpoint selection, identify potential participants and trial sites, and assess safety in the post-marketing setting.
By Vid Desai & Jessica N. Berrellez – The U.S. Food and Drug Administration has released its comprehensive FDA Information Technology Strategy for Fiscal Years 2024 to 2027 (IT Strategy), marking a defining roadmap for the agency’s technological advancement and the alignment with its broader public health mission.
By Patrizia Cavazzoni MD – AI and ML are no longer futuristic concepts; they are now part of how we live and work. The FDA uses the term AI to describe a branch of computer science, statistics, and engineering that uses algorithms or models to perform tasks and exhibit behaviors.
The thought leaders in our community are good about sharing their thoughts on the issues of today. Here are the top read and shared guest posts of December that we think deserve sharing again.
By Vid Desai & Jessica N. Berrellez – Digital transformation is a continuous journey that touches every part of an organization. The FDA established the Office of Digital Transformation in September 2021.
By Vid Desai & Craig Taylor – The U.S. Food and Drug Administration is critical to protecting and promoting public health. The products the FDA regulates are in every supermarket, pharmacy, and home across the U.S. Cybersecurity touches every facet of the FDA’s broad, complex responsibility.