ULTRA Act UPDATE and REQUEST from Julia Jenkins, Everylife Foundation

Congress is planning to introduce the Ultra-orphan Life-saving Treatments Act of 2012 or ULTRA Act to promote the discovery and development of safe and effective drugs and biologics to treat ultra-rare diseases.  Your help is needed to ensure this legislation is successful!

The ULTRA Act will improve access to the existing Accelerated Approval pathway for patients with life threatening ultra-rare diseases. The small number of patients with a particular ultra-rare disease makes it virtually impossible under the current FDA requirements to use the Accelerated Approval pathway. This legislation will empower the FDA to consider the full scope of existing scientific data when reviewing surrogate endpoints for use under the Accelerated Approval pathway, instead of requiring prior clinical data that is nearly impossible to collect for ultra-rare diseases.

It is important to show our House and Senate champions as well as the members of the House Energy and Commerce and Senate Heath, Education, Labor and Pension Committees that this legislation is supported by the rare disease community. If you would like to have your organization listed as a supporter, simply reply to this email stating that your organization would like to be added to the letter. Please share this action alert with your partner organizations. If you have any questions, contact Julia Jenkins, EveryLife Foundation at 415-884-0223.

The goal is to have 100 organizations signed on before Thanksgiving and 150 organizations signed on by Dec. 2nd. Thank you to the 60 organizations that have already signed on to support the ULTRA Act:

Abigail Alliance for Better Access to Developmental Drugs
Addi & Cassi Fund
American Behcet’s Disease Association
Amschwand Sarcoma Cancer Foundation
BDSRA (Batten Disease Support and Research Association)
Beyond Batten Disease Foundation
Blake’s Purpose Foundation
Breakthrough Cancer Coalition
Canadian PKU & Allied Disorders
Center for Orphan Disease Research and Therapy, University of Pennsylvania
Children’s Cardiomyopathy Foundation
Cooley’s Anemia Foundation
Dani’s Foundation
Drew’s Hope Research Foundation
EveryLife Foundation for Rare Diseases
GIST Cancer Awareness Foundation
Hannah’s Hope Fund
Hope4Bridget Foundation
Hypertrophic Cardiomyopathy Association – HCMA
I Have IIH
ISRMD (International Society for Mannosidosis and Related Diseases)
Jacob’s Cure
Jain Foundation
Jonah’s Just Begun-Foundation to Cure Sanfilippo Inc.
Kids V Cancer
Kurt+Peter Foundation
LGMD2I Research Fund
Lymphangiomatosis & Gorham’s Disease Alliance
MAGIC Foundation
Manton Center for Orphan Disease Research
Mary Payton’s Miracle Foundation
Midwest Asian Health Association (MAHA)
MPD Support
National Gaucher Foundation
National MPS Society
National Organization Against Rare Cancers
National PKU Alliance
National Tay-Sachs & Allied Diseases Association
New Hope Research Foundation
NextGEN Policy
Noah’s Hope – Batten disease research fund
Our Promise to Nicholas Foundation
Oxalosis and Hyperoxaluria Foundation
Partnership for Cures
Periodic Paralysis Association
RARE Project
Ryan Foundation for MPS Children
Sanfilippo Foundation for Children
Sarcoma Foundation of America
Solving Kids’ Cancer
Taylor’s Tale: Fighting Batten Disease
Team Sanfilippo Foundation
The Alliance Against Alveolar Soft Part Sarcoma
The Life Raft Group
The NOMID Alliance
The Transverse Myelitis Association
The XLH Network, Inc.
United Pompe Foundation

Please contact Julia Jenkins with questions or to sign on – patientadvocate@kakkis.org

2 thoughts on “ULTRA Orphan Act – Fast Track Treatments for Ultra Rare Diseases”

  1. Anonymous says:

    Please post my organization and keep me posted for further updates.
    Cryoglobulinemia Vasculitis Education & Awareness Organization

    Marianne Vennitti
    PR Manager
    Cryoglobulinemia Vasculitis Organization

  2. Anonymous says:

    We understand there is some confusion about this bill. This bill officially empowers the FDA to use existing biology and preclinical data instead of requiring clinical qualifying data. Typically this clinical data does not exists in the 83% of RARE Diseases referred to as “Ultra-Orphan” resulting in significant delays in approving clinical trials for new therapies. Ultra-Orphan diseases have trouble getting this data because of their very small patient populations. The larger population Orphan diseases, those with over 6,000 US patients, generally have adequate populations to get this data. All the rest of the FDA review rules, including the Orphan Drug Act, remain intact and unchanged.

    The draft bill has been available for review for a number of weeks.

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