FDA Webinar on Accelerated Approval Summary



CureDuchenne was honored to host a webinar today, in collaboration with PPMD and MDA to discuss accelerated approval for drugs for rare diseases.   Dr. Robert Temple, director of the Office of Medical Policy of FDA’s Center for Drug Evaluation and Research presented detailed information on the different options available to speed the approval of drugs.  Dr. Temple also answered questions posed by the 277 participants who logged on to today’s webinar.

To logon to the recorded webinar please click here:


A quick summary of today’s discussion:

  •  The FDA Safety and Innovation Act (aka FDASIA) amends the expedited drug development and review process by providing a fast track designation for drugs with “serious or life-threatening disease” indications and those defined as “breakthrough therapies.”
  • Breakthrough designation, fast-track designation, and the accelerated approval process are expedited review programs that primarily differ in the effectiveness      that the product must demonstrate against a serious or life-threatening disease or condition.
  • For designation as a breakthrough therapy, preliminary clinical evidence must indicate that that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. Drugs that are given breakthrough therapy designation will benefit from (among other things) an organizational commitment from the FDA involving senior FDA managers and experienced review staff.
  • A fast-track product must demonstrate the potential to address unmet medical needs.
  • For accelerated approval, there must be evidence that the product has an effect on an endpoint that is reasonably likely to predict clinical benefit (i.e. an effect on a clinical endpoint other than survival or irreversible morbidity).
  • Accelerated approval includes fast track products indicated for a serious or life-threatening  disease or condition, and approval under this mechanism comes with a requirement that the drug be studied post-approval to confirm its clinical benefit.
  • Priority review means review and action on the application no later than six months after FDA’s receipt of the application.
  • The FDA does not require a phase lll trial, their focus is on having enough data available to make the best decisions.
  • Patients/Parents can contact the FDA through the  Office of Special Health Issues:  oshi@oc.fda.hhs.gov, 301-796-8460

I’d like to thank Dr. Temple, Dr. Pariser, Mr. Bauer and Ms. Furia-Helms from the FDA and also Dr. Michael Kelly and Karen Harley at CureDuchenne for facilitating this webinar.  We are hopeful that we will have drugs for our kids very soon, and it will take a team to make it happen.

Thank you so much!

Debra Miller


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