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Rare Disease Legislative Advocates (RDLA) – Action Alerts, Updates, & Events

Capitol Hill Updates

The Children’s Health Insurance Program (CHIP) will expire on September 30 and must be reauthorized before that time. CHIP is essential for families who do not qualify for Medicaid, but struggle to afford private insurance. Some stakeholders support using the Creating and Restoring Equal Access to Equivalent Samples (CREATES) Act as an offset to renew CHIP funding. This bill would allow generics manufacturers to bring civil action against companies, alleging that they used Risk Evaluation and Mitigation Strategies (REMS) to limit access to samples needed to create generic products. Click here to read more about the CREATES Act. Click here to read about CHIP.

The Centers for Disease Control and Prevention (CDC) National Center for Health Statistics has released the National Health Interview Survey of health insurance coverage between January and March 2017. For the first three months of this year, 28 million individuals were uninsured, a 20.5 million decrease from 2010. You can read the rest of the report here.

The Senate Health, Education, Labor and Pensions (HELP) Committee will consider health insurance market stabilization proposals after Affordable Care Act (ACA) repeal and replace efforts failed to pass before August recess. Hearings will take place on September 6th and 7th. Click here to read more.

The President has signed the Food and Drug Reauthorization Act (FDARA). 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. The RACE for Children Act was included and will require drug manufacturers to enroll children into trials for treatments targeting pediatric cancers. For more information on the RACE for Children Act from Kids v Cancer, click here.

To read more about FDARA click here.

Community Action Alerts & Policy Resources

The National Organization for Rare Disorders (NORD) is encouraging patient organizations to sign-on to a letter on the Orphan Drug Tax Credit by September 7. The letter urges Chairmen Hatch and Brady and Ranking Members Wyden and Neal to keep the tax credit in place when considering proposals for tax reform. Click here to read letter and sign-on.

SYNGAP asks patient advocates to sign-on to a letter in order to change the hospital reimbursement system by September 8. The hospital reimbursement system has not evolved much since 1983, when the Orphan Drug Act was passed. As a result, hospitals may turn away patients with ultra-rare conditions. Please email Monica Weldon at if you have questions and/or would like to sign this letter. Click here to read letter.

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.

RDLA is asking patients to participate in remote In-District Lobby Days. Join the more than 230 advocates who are meeting with their federal legislators during August recess by contacting your Members of Congress. Click here to find out how to participate.

Community Events

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 National Institutes for Health (NIH); Gary Reedy, Chief Executive Officer of the American Cancer Society; and Joe Selby, Director of the Patient Centered Outcomes Research Institute (PCORI). The program will be livestreamed on WebMD, details will be provided closer to the date of the event.

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 the toolkit and on-line resource portal. Click here to register.

On September 12, the EveryLife Foundation for Rare Diseases will host the 9th Annual Rare Disease Scientific Workshop. Titled “Emerging Technologies for Rare Diseases: Clinical and Regulatory Case Studies and Approval Pathways”, the workshop will feature speakers from the Food and Drug Administration (FDA), National Institutes of Health (NIH), biopharmaceutical companies and advocacy organizations. Free registration for the livestream is available here.

On September 13, RDLA will hold a Rare Disease Congressional Caucus briefing entitled, “Curing Rare Disease: Policy and Regulation Needed for Emerging Technology.” 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 NIH and raise awareness about the importance of medical research. Click here to register for the event by September 4th.

On September 14, the Childhood Cancer Caucus will host a summit. The Caucus of over 100 Congressional members strives to raise awareness about pediatric cancer. More information available here.

On September 14 to 15, Global Genes will host the RARE Patient Advocacy Summit. 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. 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 September 15, Kids V Cancer will host a youth lobby day. Youth Advocates will meet for a lobby training session, then spend the morning visiting Congressional offices on behalf of kids with cancer. Click here to register.

On September 16 and 17, CureFest for Childhood Cancer will host a series of events, including a rally, a candlelight vigil, a walk, games, activities and live entertainment. Click here for more information.

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.

On October 11 to 12, the FDA will hold the first meeting of the new Patient Engagement Advisory Committee (PEAC). The Committee will focus on incorporating patient perspectives into medical device clinical trials. Click here to learn more.

On October 30, FDA’s Center for Drug Evaluation and Research (CDER) will host a free public workshop entitled, “Strategies, Tools and Best Practices for Effective Advocacy in Rare Diseases Drug Development.” The EveryLife Foundation will offer a limited number of $500 travel stipends through its Rare Giving program to enable rare disease patients and caregivers the opportunity to participate. You can apply online through September 17th. Click here to register.

On November 15, RDLA will host the sixth annual RareVoice Awards to recognize and celebrate advocates who have made an impact at the state or federal level. Registration opens on September 1. This event will take place at Arena Stage and is free for patient advocates. Click here for more information.

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


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