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Capitol Hill Updates

The Food and Drug Reauthorization Act (FDARA) passed the House on July 12 and is now awaiting action in the Senate. FDARA is made up of four user fee programs including the Prescription Drug User Fee Act (PDUFA). Notably, PDUFA VI strengthens efforts to incorporate patient experience into the drug development process. If user fees are not reauthorized by September 30, more than 5,000 Food and Drug Administration (FDA) employees could be laid-off. For more information on FDARA, click here.

The RACE for Children Act, which would require drug manufacturers to enroll children into trials for treatments targeting pediatric cancers, has passed the House as part of FDARA. For more information on the Act from Kids v Cancer, click here.  

Republican Senators have made amendments to their Affordable Care Act (ACA) replacement plan, the Better Care Reconciliation Act (BCRA). Some Senators have considered repealing the ACA without a replacement, which would result in coverage loss for 32 million individuals by 2026, per the Congressional Budget Office (CBO). The Senate vote is slated for today, July 25. To learn more about the BCRA, click here.

The House Appropriations Committee approved a Labor, Health and Human Services, and Education funding bill that contains increased funding for the National Institutes of Health (NIH). The bill also includes a $398 million cut to the Health Resources and Services Administration (HRSA), a $219 million cut to the Centers for Medicare and Medicaid Services (CMS), and a $1 million increase to FDA budget. The Heritable Disorders Program is housed under HRSA. You can read a summary of the bill here.

A Government Accountability Office (GAO) report released on July 11 assessed the FDA’s Expanded Access (EA) program. The EA program gives patients, especially ones with serious or life-threatening conditions, the ability to access an unapproved product outside of a clinical trial. The report noted that the FDA has taken steps to improve the EA process, but needed to provide clarity around how adverse event data will be used during the approval process. Click here to access the GAO report

Community Action Alerts & Policy Resources

The National Organization for Rare Disorders (NORD) is urging advocates to call their Senators to vote no on the BCRA. To identify your legislators and obtain their contact information, click here.
To read NORD’s statement, click here.

The EveryLife Foundation for Rare Diseases is asking individuals to contact their Members of Congress in support of the OPEN ACT (Orphan Product Extensions Now, Accelerating Cures and Treatments; HR 1223/ S1509) which has the potential to double the number of FDA-approved treatments for rare diseases. For more information and to take action, click here.

The California legislature has launched a Rare Disease Congressional Caucus that will help bring awareness to the needs to the rare disease community. The California Action Link for Rare Diseases (CAL RARE) is asking patients to encourage their state representatives to join the Caucus. Click here to take action.

Rare New England (RNE) asks Massachusetts residents to call or email state legislators to ask for their support for HB3714, "An Act to Create a Rare Disease Advisory Council.” RNE has been collaborating with MA State Representative Paul Heroux, who has championed HB3714, since 2015. To learn more about the bill and contact your representatives, click here.

RDLA is seeking nominations for the 2017 RareVoice Awards. Categories include federal agency staff, Congressional staff, patient organizations and patient advocates, state legislators and young adult advocates. We're looking to recognize and celebrate advocates who have made an impact at the state or federal levels. You may submit nominations here through July 31st. 

On July 19, RDLA convened a legislative webinar and meeting focusing on healthcare reform, the OPEN ACT, appropriations for the NIH and FDA, FDARA, and Reagan-Udall’s Expanded Access Navigator. Click here to check out a recording of the event.

Community Events

On July 26, The Hill is hosting an event on affordability and access to care at the Newseum in Washington D.C. The event will focus on addressing barriers to access. Click here to register.

On July 26, The House Energy and Commerce Committee will hold a hearing to discuss legislation to reauthorize special needs health plans. For more information about the event, click here.

On July 28, the Alliance for Health Reform will hold an event entitled, “Prescription Drug Affordability & Innovation: Policy Options & Stakeholder Views.” Click here to register.

From July 31 through September 1, RDLA will host In-District Lobby Days, which serves as an opportunity for rare disease advocates to meet with their Senators and Representatives in their local offices. RDLA will set meetings for you at your convenience. This is an opportunity to directly impact rare disease policy without the need to travel to D.C. Click here for more information

On September 3, Danny’s Dose is holding an event to educate Missourians with a rare disease or special medical needs. Click here for more information.

On September 7, Research!America will host the National Health Research Forum. The Forum will convene key stakeholders, including Francis Collins, Director of the NIH, Gary Reedy, Chief Executive Officer of the American Cancer Society, and Joe Selby, Director of the Patient Centered Outcomes Research Institute (PCORI). To register, click here.

On September 8, NIH’s National Center for Advancing Translation Sciences (NCATS), will host an event entitled, “Toolkit for Patient-Focused Therapy Development: Demonstration and Dissemination Meeting.” Patients and advocates are encouraged to attend and provide feedback on their toolkit and on-line resource portal. Click here to register.

On September 14, the Rally for Medical Research will host a Capitol Hill Day event. The rally will call on policymakers to maintain funding for the National Institutes of Health (NIH) and raise awareness about the importance of medical research. Click here to register for the event.

On September 14 to 15, Global Genes will host the RARE Patient Advocacy Summit in Irvine, CA. The conference brings together rare advocates to raise awareness and share best practices. Click here for more information.

On September 15, the 2017 Expanded Access Summit will be held by WideTrial at the Royal Sonesta Boston in Cambridge, MA. The event will feature high level decision-makers from patient advocacy organizations, industry, government, and academia to discuss pre-approval access. Click here for details and registration.  

On October 2 to 3, the 2017 Biotechnology Innovation Organization (BIO) Patient and Health Advocacy Summit will take place. The event will focus on policy issues and best practices for advocacy organizations, academia, regulators and the biotechnology industry. Click here for details and registration

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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 sbhatnagar@everylifefoundation.org.

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