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Orphan Drugs, Rare Diseases, And FDASIA Dates

March 8, 2013

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

– 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 putting FDASIA into effect and enforcing it is complex and quite an undertaking. FDASIA is a 140-page law that is divided into 11 separate sections. Each section addresses different aspects of the new drug and device law. To ensure that FDASIA is implemented successfully, the FDA set up a steering committee with the task of “integrating the requirements of FDASIA into the agency’s ongoing workload. 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 lets one follow the FDA’s progress. The website (FDASIA-TRACK) includes a table that contains information for each task :

  1. The title and section of FDASIA applicable to the task
  2. Deliverable type
  3. Description of the deliverable
  4. Task deliverable date
  5. FDA lead organization for the task.

Below is a chart of 6 FDASIA tasks (as of 03/07/13) that affects rare diseases and orphan drug development from FDASIA-TRACK :

Title

Section

Deliverable Type

Deliverable    Description

Statutory Date

FDA Lead    Organization

IX

901

Draft guidance

Not later than 1 year after the date of   enactment, FDA shall issue draft guidance on the clarified standard for   accelerated approval and fast track processes, including issues for drugs for   a rare disease or condition.

7/9/2013

CDER

IX

902

Draft guidance

No later than 18 months after date of   enactment, FDA shall issue guidance implementing requirements on breakthrough   therapies to treat a serious or life-threatening condition where early evidence   indicates drug may offer substantial improvement over existing treatments

1/9/2014

CDER

V

510

Public Meeting

FDA shall hold at least one public meeting   to discuss ways to encourage/accelerate development of new therapies for   pediatric rare diseases.

1/9/2014

OOPD

I

100

Public Meeting

“By mid-FY 2014, FDA, through the Rare   Disease Program, will conduct a public meeting to discuss complex issues in clinical   trials for studying drugs for rare diseases, including such questions as   endpoint selection, use of surrogate endpoints/Accelerated Approval, and   clinical significance of primary endpoints; reasonable safety exposures;   assessment of dose selection; and development of patient-reported outcome   instruments.”

3/31/2014

CDER

IX

901

Final Guidance

Not later than 1 year after the issuance of   draft guidance (above), FDA shall issue final guidance on the clarified   standard for accelerated approval and fast track processes, including issues   for drugs for a rare disease or condition.

7/9/2014

CDER

VI

617

Final guidance

Not later than 2 years after the date of   enactment, FDA shall issue final guidance on replication of custom devices   used to treat rare conditions where production of such devices is limited to   no more than 5 units per year of the device type.

7/9/2014

CDRH

Please Note: Additional deliverables will be added to the chart over time as the FDA continues to advance its implementation planning efforts.

References

Arnold & Porter LLP, titled,“ A Reference Guide to the Food & Drug Administration Safety and Innovation Act”.

FDASIA website

Pharmaceutical Law & Industry Report (Vol. 10, NO. 29) titled, “A Guide to the Food and Drug Administration Safety and Innovation Act”.

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

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

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