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Capitol Hill Updates

Dr. Califf Confirmed as FDA Commissioner
Dr. Robert Califf has been confirmed by the U.S. Senate for the role of FDA Commissioner by a vote of 89-4. Dr. Califf has stated that his priorities will relate to enhanced patient engagement and looking at new models to improve the clinical development process. 

The next
Prescription Drug User Fee Agreement, or PDUFA VI, has been completed and is awaiting sign-off from the Department of Health & Human Services (HHS). Key priorities are likely to include provisions to increase the staffing level at FDA, reduce turnover, and improve hiring practices. Patient-focused drug development is another area of emphasis, with commitments to enhance the tools used to capture patient preference. Assuming the deal is approved by HHS, it will then go to Congress for consideration in January 2017.

Senate Biomedical Innovations Initiative
The Senate HELP Committee has completed the first of three scheduled hearings on its biomedical innovation initiative and companion to the House 21st Century Cures legislation. In the first hearing, the Advancing Targeted Therapies for Rare Diseases Act (S 2030) was passed, along with the Advancing Neurological Diseases Act (S 849). However, the Advancing Neurological Diseases Act was substantively altered so that the established disease registry would only apply to five of the most prevalent diseases. Advocates are hopeful that the bill can be altered during the conference process so that rare diseases may also be included. 

Community Action Alerts 

The EveryLife Foundation for Rare Diseases is asking advocates to contact Congress in support of the OPEN ACT, legislation that could help double the number of treatments for rare diseases. Ask your representatives in Congress to stand-up for patients by co-sponsoring the OPEN ACT

Parent Project Muscular Dystrophy (PPMD) is circulating an action alert asking Congress to support the continued development of treatments for Duchenne by calling for increased funding for the Centers for Disease Control & Prevention (CDC), expanding research at NIH, incorporating new endpoints in the drug approval process at FDA, and ensuring continued commitment to the MD-CARE Act. Click here for more information and to sign-on

The Food and Drug Administration (FDA) has been holding periodic Patient-Focused Drug Development meetings in order to increase patient engagement. The FDA is seeking input from patient organizations looking to organize external meetings. If your rare disease organization is interested in participating, you may send a letter of intent to patientfocused@fda.hhs.gov. To learn more, visit the FDA website

Community Events

On Monday, February 29th, Rare Disease Week on Capitol Hill kicks off! Hundreds of advocates from around the country will be in Washington, D.C. for a full week of events. Space remains for the Caucus Briefing on Thursday March 3rd and the Rare Artist Reception. Click here for more information and to register. Can't make it to Washington D.C.? NORD is helping coordinate State House Events across the U.S. Click here for more information and a list of participating states

On Wednesday, March 2nd, the EveryLife Foundation for Rare Diseases is holding a Virtual Lobby Day for advocates who cannot attend the events in D.C. The event will ask advocates to contact Congress and ask that they co-sponsor the OPEN ACT, legislation to double the number of rare disease treatments. Please share widely on social media. 

On Thursday, March 3rd, the first bicameral Congressional Rare Disease Caucus briefing will be held in the Auditorium of the U.S. Capitol Visitor Center at noon. Attendees will hear from the co-chairs of the Caucus and a panel discussion featuring key thought-leaders from the patient, regulatory, and industry communities who will discuss the Rare Disease Ecosystem. Click here for full agenda and to register. 

On Wednesday, March 9th, the Senate Health, Education, Labor, and Pensions Committee (HELP) will hold its second hearing as part of its biomedical innovation initiative. To date, the Advancing Hope Act (Priority Review Voucher program at FDA) is scheduled for consideration. The final hearing is slated for April 6th, although no bills have been announced for consideration.  

On Wednesday, March 16th, the EveryLife Foundation for Rare Diseases will hold a public webinar on newborn screening. The Foundation has just launched newborn screening legislation in California to expand and streamline screening for rare diseases. Click here to register for the event

On Wednesday, March 23rd, RDLA will hold its next monthly webinar. The agenda is OPEN! Please send suggestions for action items or policy issues to Vignesh Ganapathy at vganapathy@everylifefoundation.org. You may click here to get registered

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***This E-Blast shares action alerts, legislative, and policy news and events from the patient advocacy community. RDLA does not take positions on the issues herein, but serves as a supportive clearinghouse for the rare disease community. Send us an email if you'd like your alerts and/or events included! Email mbronstein@everylifefoundation.org.

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