The new FDA guidelines allow lots of smart things to happen...if one gets creative...IMO, this will require a detailed understanding of new trial results. On the other hand, it looks like things can happen quickly when trial evidence presents the appropriate case. In fact, the new rules seem to say ...FDA expects and will support such action.
If these new rules are now effective or close then (IMO) it is no wonder that BP trials which were going no where have been dropped. My guess is that since Dr.M has been a principal sponsor for many of these initiatives that AVXL trials will be accordingly structured. Lots of moving parts here but the days of massive trail and error are gone...finally. And, the BP R&D structures will be(and are) being reconfigured to support more AI/tools and fewer petri dishes.
[quote Implement new streamlined orphan designation review template and expand orphan opportunities to new areas
• Modernize the Office of New Drugs to ensure policies are rooted in the best science and management principles, and staff have the support and tools to achieve the public health mission
• Develop a series of new, disease-focused medical product guidance documents to update and modernize approaches to clinical trial design and other aspects of the development of drugs targeting unmet medical needs. This will include a new suite of guidances and policies focused on the development of drugs targeting a group of serious neurodegenerative disorders
• Update and modernize FDA’s approach to applying evidentiary standards for establishing safety and effectiveness for new drugs to more clearly define the role of real-world data and evidence, especially when it comes to evaluating post-market safety
• Develop patient-focused drug development guidance to facilitate a more systematic approach to gathering and using patient perspectives to inform regulatory decision-making
• Advance the use of in silico techniques to develop novel methods for creating models of virtual patient outcomes and modernizing FDA’s evaluation of patient benefit and risk
• Develop and advance recommendations for an OTC monograph user fee program, to implement structural reforms to streamline and improve the timeliness of review activities, and foster innovation of OTC products
• Advance a new regulatory framework (NSURE) to enable more drugs to be made available in OTC forms through the use of technology (such as medical apps) that can help patients better selfselect when a product is appropriate
• Explore the development of a policy framework under which an accelerated approval approach could be used to support marketing of a drug that demonstrates a survival benefit early in clinical development. The goal is to expedite availability of a therapy while the magnitude of the benefit it provides is being confirmed
• Advance the use of new drug development tools and mobile technology for better capturing clinical trial data and the measurement of safety and benefit in pre- and post-market settings
][/quote]
News 
Market Data 
Discover 
AI
