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Rare Diseases And Orphan Drugs: FDA’s FDASIA Calendar

November 25, 2013

The FDA Safety & Innovation Act (S. 3187), FDASIA, is signed by President Obama in July 2012. FDASIA provides for the development of effective and safe treatments for rare diseases and orphan drug development in the United States. FDASIA provides for the following :

•   Acceleration of new medical treatments for patient access

•   Development of Humanitarian Use Devices (medical devices) for small patient populations

•   “Breakthrough Therapy” designation for drugs that show early promise in the development process

•   Consultation with rare disease medical experts

•   Creation of a rare pediatric disease priority review voucher incentive program.

The task of implementing FDASIA is complex and a large undertaking. FDASIA is a 140-page law that is divided into 11 separate sections, with each section addressing different aspects of the new drug and device law. To ensure that FDASIA is implemented successfully, the FDA set up a steering committee. One of the committee’s projects is to create a table that tracks what the FDA has to do to comply with FDASIA – a type of project plan. The FDA establishes in November 2012, a website that tracks the FDA’s progress for the implementation of FDASIA. The website (FDASIA-TRACK) includes a table that contains information for each task :

•   The title and section of FDASIA applicable to the task
•   Deliverable type
•   Description of the deliverable
•   Target Date for completion of the task
•   FDA Lead organization for the task.

Below is a chart of 9 FDASIA tasks (as of 11/24/13) that affects rare diseases and orphan drug development from FDASIA-TRACK (in chronological target date order) :

Title

Section

Deliverable    Type

Deliverable    Description

Target Date

FDA Lead    Organization

IX 901 Draft Guidance Clarified standard for accelerated approval & fast   track processes, including issues for drugs for rare diseases. 7/9/2013** CDER
VI 617 Draft Guidance Replication of custom devices used to treat rare conditions. 8/30/2013 CDRH
I 100 Public Meeting Discuss issues in clinical trials for studying drugs for rare diseases. 1/6/2014 CDER
V 510 Public Meeting Discuss ways to encourage/accelerate development of new therapies for pediatric rare diseases. 1/8/2014 OOPD
IX 902 Draft Guidance Implementation  requirements for Breakthrough Therapies. 1/9/2014 CDER
V 510 Public Report Strategic plan for  encouraging & accelerating development of therapies for pediatric rare diseases. 7/7/2014 OOPD
VI 617 Final Guidance Replication of custom devices used to treat rare conditions. 7/9/2014 CDRH
IX 901 Final Guidance Clarified standard for accelerated approval and fast track processes, including issues for rare diseases. 7/9/2014 CDER
IX 902 Final Guidance Implementation requirements for Breakthrough Therapies. 7/9/2014 CDER

**  Completed 6/25/2013 & Draft Guidance

Please Note : “Schedule” by Gentaur (Gentaur) [Public domain] | Wikimedia Commons.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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