Spread the word » Facebook Twitter


Capitol Hill Updates

With the election heating up, Members of Congress are anxious to complete their legislative business in Washington, D.C. before heading back to their home states to campaign. At the time of this writing, Congress has yet to take any new legislative action on 21st Century Cures, despite reports that a revamped package is in the works. Some have predicted that the issue will be taken-up again after the November elections, and with time running out this appears to be the most likely scenario.

In related news, the Priority Review Voucher program, championed by Kids V. Cancer, was reauthorized by the Senate through the end of this year. The legislation now moves to the House for consideration.  In addition, Congress has yet to reach an agreement on how to fund the government in the next fiscal year. Insiders are prediciting that an agreement will be reached in the coming days, which will likely provide stop-gap funding until after the elections. Public health advocates are also hopeful that new funding for Zika control measures will also be included in the final package. 

Meanwhile in California, Governor Brown signed into law newborn screening legislation championed by the EveryLife Foundation for Rare Diseases and supported by over 120 patient organizations! The legislation will greatly accelerate the rate at which new diseases are screened for in California and requires screening of Pompe disease and MPS I. For more information about the legislation, click here.

Community Action Alerts & Policy Resources

The EveryLife Foundation for Rare Diseases is calling on advocates to help move 21st Century Cures with an advocacy photo-blast. Advocates can send in photos with their name, state, and #CuresNOW hashtag, which will be shared with Congress. To see examples and to submit your own, click here. 

Research!America is asking advocates to step-up the fight for 21st Century Cures with several ways to take action. To get engaged and learn more, click here.

With debate around the costs of prescription drugs swirling, a new report was issued from IMS Health detailing future spending on orphan drugs. The authors found that despite growth in the number of available treatments, overall oprhan drug spending will continue to be sustainable in the future. Click here to read the full report published in Health Affairs.

Community Events

On Wednesday, October 5th, Amgen is holding its Annual Health Equity Summit at The George Washington University in Washington, D.C. The meeting will highlight ways to incorporate health equity into various advocacy platforms. Email Meg Hanhan to learn more and to register.

On Monday, October 17th, NORD will kick-off its annual Rare Diseases and Orphan Products Breakthrough Summit in Arlington, Virginia. The event will include representatives from over 80 patient organizations, industry leaders, and FDA staff. Click here for more information and to register

On Thursday, October 20th, the Food and Drug Administration (FDA) will host a public meeting to discuss the reauthorization of the Biosimilar User Fee Act (BsUFA II) from 9:00 AM to 2:00 PM EST at the FDA’s White Oak Campus in Silver Spring, MD.  If you would like to attend this meeting, please register here by Wednesday, October 19.  The FDA will accept electronic and written comments from the stakeholder community until Wednesday, October 19.  Instructions on how to submit comments are available on the Federal Register Notice here.  

On Wednesday, October 26th, RDLA will host its next webinar and conference call at 1pm Eastern time. The agenda is open, suggestions may be emailed to vganapathy@everylifefoundation.org. To register, click here.

On October 27-28th, the National Health Council (NHC) will host the Inaugural Conference for Chief Medical/Scientific Officers and Research Directors, Measuring Our Impact on Science, Delivery of Care, and Patient Outcomes. The Conference will be held in Baltimore, MD, and will bring together research leaders from the patient community, nonprofit health organizations, and the biotechnology industry.  For more information and to register, please visit the NHC’s website.

On Thursday and Friday, November 3-4th, the Biotechnology Innovation Organization (BIO) will host its annual Patient & Health Advocacy Summit at the Mayflower Hotel in Washington, D.C.  Topics will include patient-focused drug development, and value assessment frameworks. For more information and to register, click here.

On Tuesday, November 15th, the Rare Disease Congressional Caucus will convene a lunch briefing on Capitol Hill at noon. The event will highlight the upcoming legislative activies surrounding the next Prescription Drug User Fee Act (PDUFA), the agreement that funds drug review at the FDA. To register, click here.

On Wednesday, November 16th, RDLA will host the annual RareVoice Awards in Washington, DC. Help us celebrate outstanding rare disease advocates! Senator Orrin Hatch (R-UT) and Representative Joseph Crowley (D-NY) will be receiving awards along with other champions from the rare disease community. For more information and to register, click here.

On Wednesday, November 16th, the EveryLife Foundation for Rare Diseases will host its annual Community Congress meeting in Washington, DC. The event will feature keynote speakers (TBA), a networking lunch, and afternoon working group meetings. To register, click here.

Join Our Mailing List and On Social Media

Stay up to date on breaking rare disease legislative news by following @RareAdvocates on Twitter and Facebook.

Receive this from a friend? Sign-up for our email list to make sure you don’t miss monthly newsletters and action alerts!

***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.

Click here to unsubscribe