We (PPMD and Cure Duchenne) are working with FDA to organize a Webinar. The purpose of the webinar is education - about FDA, about policy, about process and about approvals. You are already aware FDA has created a new position specifically for rare disease. Dr. Anne Pariser has taken this position and is working to streamline efforts within FDA. There are other initiatives taking place to overcome regulatory hurdles to include providing additional expertise for review and additional funding.
I would like to know from you what you are interested in learning about FDA. Keep in mind, this webinar will not cover questions such as expedited approval for exon skipping, rather will be a primer about FDA.
It is important that this webinar (though may be covered in a 2-part series) serve the needs of the Duchenne and Becker community. We expect the seminar will be available on podcast, either on our respective sites or on the FDA site with links.
Please email me directly (email@example.com) with thoughts, questions and concerns. I will organize, circulate and send them to FDA as we begin to plan the webinars. Sincerely, Pat