As an advocate for patients with rare diseases you are a very important part of the legislative process. You can make the difference as you are the voices your legislators and congressmen want, or in some cases do not want to hear. Please complete the form below to take action and contact your Member of Congress

After you take action your job is not done! The final step is to share the action alert with your family, friends, co-workers and any other people that might be interested in taking action on behalf of the rare disease community.

ACTION ALERT: The Advancing Research for Neurological Diseases Act (S. 849)

Invite your U.S. Senators to attend a briefing on the Advancing Research for Neurological Diseases Act (S. 849) on September 16 from 11:30 to 12:30 in the Senate Russell Building, room 485. The briefing is sponsored by the American Academy of Neurology, American Brain Coalition, Brain Injury Association of America, Epilepsy Foundation, International Essential Tremor Foundation, National Multiple Sclerosis Society, Parkinson’s Action Network, Rare Disease Legislative Advocates, Research!America, Tourette Association of America, and United Spinal Association.

For more information and to contact your Representative, click here.

ACTION ALERT: Thank the House for 21st Century Cures

The House passed historic bipartisan legislation called the 21st Century Cures Act HR 6, and they deserve our thanks for ensuring that the voice of the rare disease community was heard.  Let’s show every member who supported 21st Century Cures how much their hard work means to us, and how eager we are to see this meaningful legislation make its way through the Senate.  Please take a moment to thank your representative for supporting the 21st Century Cures Act and the rare disease community.

NDD United Sign On Letter to End Sequestration

Sequestration poses significant risk to the funding NIH and FDA receives  – agencies which are critical to advancing our understanding of and treatments for rare diseases. Help end sequestration and the threat of cutting billions of dollars from our most crucial facilities by getting your organization to sign-on to the letter linked below: Read more

Sign-on to Protect FDA Funding

From The California Healthcare Institute (CHI): We are working to protect critical funding for the drug review divisions at FDA, which are under threat of budget cuts that could greatly hamper the drug approval process. Organizations are being asked to endorse legislation, FDA SOS Act HR 1078, to protect these vital funds.

Read the full letter here – organizations that wish to sign-on may email Jenny Carey,

Rare Disease Patients are a Priority in 21st Century Cures; RDLA’s Lobby Days & Action Alerts are Having an IMPACT

After hundreds of Hill meetings, emails and calls to Congress, we are seeing the true impact of Rare Disease Patient Advocacy on the 21st Century Cures Legislation.  The latest draft released this week includes many priorities for the rare disease community – including 3 out of the 4 pieces of legislation advocates lobbied for during Rare Disease Week 2015:

  • HR 971, Orphan Product Extensions Now Accelerating Cures and Treatments Act(OPEN ACT)
  • HR 292, Advancing Research for Neurological Diseases Act
  • HR 909, Compassionate use Reform & Enhancement (CURE) Act

Other important provisions benefiting the rare disease community include, making the Pediatric Priority Review voucher program permanent, the NIH Innovation Fund, language on Precision Medicine for Orphan diseases, and patient focused drug development.

Read more

Sign-on to Thank Reps. Upton & DeGette for their Leadership

Rare disease groups are circulating an organizational sign-on letter to thank Chairman Upton (R-MI) and Representative DeGette (D-CO) for leading the 21st Century Cures Initiative, and for incorporating the patient voice. To date, over 40 organizations have signed, including: the EveryLife Foundation, Global Genes, Parent Project Muscular Dystrophy, and the Sarcoma Foundation of America. View the sign-on letter here and email to get signed-on. Please share with your networks so we can send a strong message to Congress!

Sign-on to Preserve GINA & ADA Protections

Genetic Alliance is circulating a sign-on opportunity for groups looking to protect the Genetic Nondiscrimination Act and the Americans with Disabilities Act. Recently introduced legislation seeks to exempt employer-based wellness programs from GINA and the ADA. This could allow employers to inquire about employees’ private genetic information or medical information unrelated to their ability to do their jobs, and penalize employees who choose to keep that information private. Click here to view the full letter and to sign-on.

Stand Up for Health Research Funding

Research!America has issued an action alert on behalf of health research funding. Annually, members of Congress have the opportunity to influence the appropriations process by submitting their funding priorities to appropriators. Now is the time to ask your representatives in the House and Senate to champion increased funding for medical and health research in the fiscal year 2016.

Patient Focused Drug Development

Parent Project Muscular Dystrophy (PPMD) is taking the lead on strengthening the voice of the patient throughout the drug and larger medical product development process. They are doing this by urging congress to add  transparency requirements in upcoming 21st Century Cures Legislation.

PPMD has written a sign-on letter to urge that a provision known as the Patient-Focused Impact Assessment Act (PFIA) be included in 21st Century Cures draft legislation when released in early 2015.

Read the letter here: PFIA Sign On Letter

If you have any questions, or to sign-on please feel free to contact PPMD or Annie Kennedy with Parent Project Muscular Dystrophy