The Global Genes Project and R.A.R.E. team have been working with Rare Disease Legislative Advocates (RDLA) on legislative improvements that would help bring more drugs to rare disease patients more quickly.  This legislation promotes the use a variety of scientific data to qualify a surrogate endpoint for use in a clinical trial, when the required historical clinical information on the surrogate does not exist or could never practically be obtained.  We discussed the FAST and ULTRA legislation that started this proposal in a prior blog post. The language was incorporated into the House draft of PDUFA (Prescription Drug User Fee Act) but has been removed from the version that is about to be presented to the Senate.

Please respond today to the RDLA’s Call To Action by contacting Senators Harkin (D-IA) and Enzi (R-WY) asking them to add back in the language from HR 4132, the FAST Act.  We will re-post the RDLA’s Action Call if you wish to learn more about the details.

CALL TO ACTION – TODAY!

The community needs 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.
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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 voice-mail.  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)

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.

For more information please contact Julia Jenkins at patientadvocate@kakkis.org

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