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Capitol Hill Updates

After a contentious debate about the future of healthcare, the Trump Administartion and Republican leadership have retreated from the goal of repealing and replacing the Affordable Care Act. During the debate, rare disease advocates weighed-in with concerns about how repeal would impact critical provisions for patients like out-of-pocket maximums, coverage for patients with pre-existing conditions, and support for the Medicaid program. At the time of this writing, the Administration and Congress are shifting their focus to begin work on tax reform. 

Meanwhile, the House Energy and Commerce Committee and Senate HELP Committees have held their first hearings on reauthorizing the Prescription Drug User Fee Act (PDUFA) legislation, a vital agreement between industry and the Food and Drug Administration (FDA) to help ensure funding of drug review. The hearings highlighted that the new PDUFA agreement must be reauthorized no later than July 31st to avoid any disruptions to drug review at the FDA. Democrats and Republicans have successfully worked together to ensure that this deadline was met in prior reauthorizations. 

The President's budget was also released, a blueprint indicating how the Administration would prioritize federal spending over the next fiscal year. Broadly speaking, the budget proposes to substantially boost military spending and support for veterans by deeply cutting various domestic agencies. Among the agencies targeted for the deepest cuts was the National Institutes of Health (NIH), which would lose $5.8 billion in funding or nearly 20% of its budget authority. Further, the FDA would lose hundreds of millions in funding, and the Adminstration has proposed dramatically increasing industry fees to make up for the loss. It is important to note that the President's budget is entirely non-binding and serves as a recommendation to Congress. Members from both sides of the aisle have expressed concern about the President's budget and it is highly unlikely that these deep cuts would be enacted by Congress. 

Community Action Alerts and Policy Resources

The EveryLife Foundation for Rare Diseases is proud to announce that the OPEN ACT (HR 1223), legislation to dramatically increase number of rare disease treatments, has been reintroduced. Take a moment to contact your Representative to ask for co-sponsorship of this legislation. EveryLife is also seeking support from patient organizations to endorse the legislation, and you can click here to see the list of the nearly 200 supporting organizations

Kids V Cancer is seeking support for the Race for Children Act (S. 456, HR 1231), which would update existing law to require companies to gather data about drug safety and efficacy in pediatric populations. To learn more about the legislation and to take action, click here

The National Organization for Rare Disorders (NORD) is asking patients to take action against a new House bill, HR 1313, that would allow employers to obtain information on their employee's genetic data and medical history. Click here to learn more about the bill and to take action. 

Research!America is calling on advocates to reach out to Congress and request that they pass a budget for the current fiscal year that prioritizes medical research by increasing funding for critical agencies like the NIH and FDA. Click here to take action and contact Congress

Videos from the Rare Disease Congressional Caucus briefing on March 2nd on Advancing Rare Disease Treatments in the Era of Cures and Health Care Reform are now available online. Videos from previous briefings and a list of Caucus members in the House and Senate are available on the Caucus webpage

Community Events

On Wednesday, March 29th (TODAY), at 1pm the EveryLife Foundation's Community Congress program will convene a free webinar on Clinical Development for Rare Diseases: A Primer for Rare Disease Patients and Advocates. For the agenda and to register, click here. 

On Tuesday, April 4th, the U.S. Senate Committee on Health, Education, Labor and Pensions (HELP) will hold its next FDA user fee hearing at 10am in 430 Dirksen Senate Office Building. The hearing will focus on innovation for patients and improving medical regulation. The event is open to the public and will be livestreamed here

On Tuesday, April 11th, the American Cancer Society Cancer Action Network (ACS CAN) will convene its sixth public policy conference at the National Press Club from 8:30am-2:00pm. The conference will gather stakeholders from across the cancer spectrum to discuss changes in health insurance. Click here for more information and to register. 

Rare Disease Legislative Advocates (RDLA) will hold its next meeting on Wednesday, April 19th, at 2:30pm. This meeting will be online and in-person for those in the DC-area who wish to join. Click here to register or contact vganapathy@everylifefoundation.org if you would like to speak about a state or federal policy issue. 

On Saturday, April 22nd, the March for Science will be held in Washington, DC. To learn more and the event, along with satellite marches occuring around the country, click here

The FDA and Center for Drug Review (CDER) is convening a workshop for advocacy groups on Friday, May 12th, from 9am to 3pm. The goal of the workshop is to help patient organizations learn how to successfully engage with CDER. Click here for more information and to register. The EveryLife Foundation is providing a limited number of $500 travel stipends for this event. Click here for more information and to apply by the deadline of April 2nd

The EveryLife Foundation is partnering with Global Genes to convene RARE on the Road - Leadership Tour this summer. It will conist of three events designed to educate rare disease patients on how how to successfully advocate and build their networks. Click on the links below for additional information:

On Thursday, May 18th, RDLA will convene the next Rare Disease Congressional Caucus Briefing on Capitol Hill. Save the Date! Details to follow. 

On Thursday, May 18th, the National Organization for Rare Disorders (NORD) will hold the 2017 Rare Impact Awards at the Ronald Reagan Building in Washington, DC. NORD will honor advocates and policymakers for their advocacy at the state and federal levels. For more information and to register, click here

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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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