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Capitol Hill Updates

Senate HELP Continues Biomedical Innovations Initiative
The Senate Health, Education, Labor, and Pensions (HELP) Committee is continuing its work to produce companion legislation to the 21st Century Cures Act, originally passed by the House in July 2015. The HELP Committee has held two markups to date, and a third and final markup is scheduled for April 6th. At the time of this writing, two bills have been scheduled to be heard: the FDA & NIH Workforce Modernization Act and language relating to the White House's Precision Medicine Initiative. There remains controversy over how to fund proposed budget increases for the NIH and FDA. In addition, the Committee has not indicated whether they will markup the OPEN ACT (S. 1421), legislation to encourage companies to repurpose therapies for rare diseases.

Report Released: Pediatric Priority Review Voucher Program
The Government Accountability Office (GAO) has completed its report on the Pediatric Priority Review Voucher program, as mandated by the legislation. The GAO interviewed dozens of stakeholders for the report and has found that it is "Too Early to Gauge Effectiveness of the FDA's Pediatric Voucher Program." The authorizing legislation for the program was included in the Senate HELP Committees markup, held in March. 
You may read the full GAO report here


Community Action Alerts & Policy Resources

As the election season heats up, patient organizations are joining with Research!America to ask candidates for their views on medical research through the Campaign for Cures. You can download their toolkit for resources to help make medical research a national campaign issue. 

The EveryLife Foundation for Rare Diseases will launch an action alert on Tuesday, April 5th, asking advocates across the country to call the Senate to urge them to support the OPEN ACT, legislation that could double the number of rare disease treatments. At the time of this writing, the bill has still not been scheduled for consideration by the Senate and the clock is ticking. Click here to join rare disease advocates in the call-in

To expand the number of disease areas addressed in the Patient-Focused Drug Development initiative (PFDD), the U.S. Food and Drug Administration (FDA) welcomes patient advocacy organizations to organize externally-led PFDD meetings. The FDA recommends submitting a Letter of Intent (LOI), guidelines for which are available here. If your organization is interested, please submit your LOI to patientfocused@fda.hhs.gov. To read more about the externally-led PFDD meetings, please visit the FDA’s website.

The Coalition of State Rheumatology Organizations has launched a petition in protest of proposed changes to Medicare Part B, which could impact patient access to medications. Advocates are concerned about how changes to reimbursement rates could affect the ability of treatment centers to operate. Click here to learn more and to take action. 

The EveryLife Foundation for Rare Diseases has a launched newborn screening legislation in California to expedite the process of screening for diseases, and with the broader goal of ensuring rare disease patients get access to treatments as early as possible. To date, over 45 patient organizations have endorsed the legislation and EveryLife is seeking additional patient orgs to sign on. Click here to learn more about the initiative. 


Community Events

On Thursday, March 31st, The Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER), is sponsoring a public workshop entitled Navigating CDER: What You Should Know for Effective Engagement.  The purpose of this public workshop is to help the public and patient advocacy groups gain a better understanding of how to effectively engage CDER. There will be an opportunity for questions and answers following each presentation. Click here for more information and to register.

On Tuesday, April 19th, the American Cancer Society Cancer Action Network (ACS CAN) will host the National Forum on the Future of Health Care: The Role of Technology in America’s Shifting Health Care Landscape. The event will be held from 8:30 AM – 3:00 PM, EST at The National Press Club in Washington, DC. The conference will provide a forum to examine how technology is improving diagnosis and treatment options for cancer patients and others with chronic illness, and will advance the discussion about the role of technology in America’s shifting health care landscape. For more information or to register, please visit ACS CAN’s website or email Anna Pugh at anna.pugh@cancer.org

On Friday, April 22nd, the U.S. Food and Drug Administration’s (FDA) Offices of Hematology and Oncology Products, and Health and Constituent Affairs, will host a public meeting entitled Childhood Cancer Advocacy Forum 2016. The Forum will be held from 9:00 AM – 12:00 PM, EST, at the FDA’s White Oak Campus in Silver Spring, MD. Additional information about this event is available here. Please note, you must register by Wednesday, April 20, in order to participate in this meeting.

On Wednesday, April 20, RDLA will hold its next webinar. Those in Washington D.C. may join in-person and the regular webinar option will be available as well. If you have suggested agenda topics, please email vganapathy@everylifefoundation.org with suggestions. 

On Wednesday, May 18th, the Rare Disease Congressional Caucus will convene on Capitol Hill for a Senate briefing. Stay tuned for additional details. 


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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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