Anavex Life Sciences Corp (AVXL)

[polarbear77]

Xena/power, likely the FDA’s new 2018 Strategic Policy Roadmap will be useful for our Rett Syndrome trial, as well as, of course, our upcoming AD trial.

As Nidan pointed out in post 137220, quote: “Check out pg 13. The new rules are written to allow discretionary authority to FDA when interim trial results warrant such. Very cool. IMO.”

In addition: new streamlined orphan designation review, accelerated approval potential and use of RWE and patient perspectives with this new 2018 framework.

From the January 2018 FDA release titled: HEALTHY INNOVATION, SAFER FAMILIES: FDA’S 2018 STRATEGIC POLICY ROADMAP
https://www.fda.gov/downloads/AboutFDA/ReportsManualsForms/Reports/UCM592001.pdf

Excerpted page 13:
“FDA will cultivate new policies and guidance for product regulation in key areas of novel medical science, with the goal of creating pathways that allow beneficial new technologies to efficiently reach patients while strengthening FDA’s gold standard for product safety and effectiveness”

• “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 modernizeapproachestoclinicaltrialdesignand 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 OTCproducts
• 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 self- select 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”