Friday, April 6, 2012

URGENT - Please take action TODAY - Tell the Senate to include the FAST Rare Disease Language in PDUFA

The Senate’s discussion draft for the Prescription Drug User Fee Act (PDUFA) was released late 
Wednesday.  Unfortunately, the rare disease language from the ULTRA/FAST Acts that allows improved
 access to the Accelerated Approval process for rare diseases was not included.

We need your help to ensure the language will be included in the second draft of PDUFA.  
Senators Harkin (D-IA) and Enzi (R-WY), as the Chairman and Ranking Member of the 
Senate HELP Committee, have the power to include this important language in PDUFA.  
Please take 5 minutes TODAY to call Chairman Harkin’s and Ranking Member Enzi’s offices and 
tell them why this is important to you.

We will only be successful if YOU take ACTION immediately!  Senators Harkin & Enzi need to 
receive hundreds of calls before 5pm EST today from advocates to understand that it is important for 
rare disease patients.  YOU can make the difference!  Make your calls and then please share this action
 alert via email, Twitter and Facebook.

Thank you for you for taking action and for your continued support.
Below are the numbers for Harkin & Enzi’s offices.  Please speak with the staff member who answers the
 phone, using the script below.  If no one answers, leave a voicemail.  If the voicemail box is full, call one
 of the district offices.  Keep trying until you are sure they received your message.  If you want a direct 
response, you should leave your contact information and specifically request a written response.  
Be respectful to the staff, but express your urgent concern.  
Chairman Harkin (D-IA) 
DC Office:  202-224-3254
District Offices: 319-365-4504, 563-322-1338, 515-284-4574, 563-582-2130, 712-252-1550      
(if you live in Iowa, please say so on the call)

Ranking Member Enzi’s (R-WY)
DC Office:   (202) 224-3424      Toll-Free:  (888) 250-1879     
District Offices: (307)261-6572, (307)772-2477, (307)527-9444, (307)682-6268, (307)739-9507      
(if you live in Wyoming, please say so on the call)

Sample Scripts:
Parent/Family/Caregiver/Friend:  I am a parent of a child with a rare disease that has no treatment.  
I am calling you because I’m concerned that the Senate’s version of PDUFA does not include language
 to improve access the Accelerated Approval Pathway for rare diseases.  This language, found in HR 4132
 the FAST Act is essential to spur the development of lifesaving treatments for my child.  Please make sure
 the rare disease language is included in the final version of PDUFA.  My child will die without treatment.
Patient:  I’m calling today because I have a rare disease and I deserve a treatment.  The Senate has failed
 to give rare diseases access to the Accelerated Approval pathway in the draft PDUFA language.  
The language being circulated is missing essential language from HR 4132 the Faster Access to 
Specialized Treatments Act that fixes a catch-22 for rare diseases.  I demand that the Senate listen to the 
voices of the patients!
Physician/researcher/industry:  I am a physician working to treat rare diseases calling because I’m 
concerned that the Senate failed to include language in the PDUFA draft that allows rare diseases to 
have access to the Accelerated Approval pathway.  These diseases are so rare that it will be extremely 
difficult to ever get this treatment to patients without access to the Accelerated Approval Pathway. 
 Please include the language from HR 4132 that was included in the House version of PDUFA.
Please share how your calls went on our Facebook event page: or 
respond to this email letting us know that you took action. 
More information on PDFUA:
The Breakthrough Therapies & EXPERRT Acts were included in the Senate’s draft along with the 
Accelerated Approval language in TREAT (S.2113).  While this is very important for rare disease patients,
 it is not enough.  The language, as it is written in the Senate version does not encourage the FDA to allow
 achievable criteria to qualify surrogate endpoints for rare diseases, leaving us with the same limited access
 to Accelerated Approval pathway that we have now and does not place any pressure on the FDA to 
change the status quo.

Currently, the House draft version of PDUFA does include the ULTRA/FAST language that is needed to
 spur the development of treatment for rare diseases.  While the Senate does not include our language, 
the discussion draft is not final; we still have time to influence the Senate’s language.  We must have 
our language in both the House & Senate versions of PDUFA or we risk having our language debated 
in Conference Committee where we have little influence and anything could happen.  In order to be
 includes the ULTRA language in both the House & the Senate.


Matthew Alsante
Executive Director

Address postal inquiries to:
Sarcoma Foundation of America
9899 Main Street, Suite 204
Damascus, MD 20872
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