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.

Research!America Asks Advocates to Contact Congress to Complete FY19 Appropriations

Research!America asks advocates to contact Congress to complete the FY19 Appropriations bills. Critical science agencies like FDA and NSF are currently caught up in a partial government shutdown. Encourage your elected officials to end the shutdown and pass all remaining FY19 funding bills, ending the harmful impact on crucial scientific progress.

Click here to take action.

Sick Cells and the Sickle Cell Disease Association of America Asks Advocates to Write Their Representatives to Pass S. 2465 in the House

In February 2018, the sickle cell community celebrated with the announcement of two legislative victories in Congress. First, the U.S. House of Representatives passed H.R. 2410 on February 26, 2018, the Sickle Cell Disease Research, Surveillance, Prevention, and Treatment Act, which was introduced on May 11, 2017, by Rep. Danny Davis (D-IL). On February 28, 2018, Senator Cory Booker (D-NJ) and Senator Tim Scott (R-SC) introduced S.2465, the Senate companion bill to H.R. 2410 in the Senate. The Senate companion bill incorporated changes to include “other heritable blood disorders” in addition to sickle cell disease. The bill passed the Senate on October 11, 2018.

The bill moved back to the House for a final vote before it can become law. It’s vital that the sickle cell disease community contact their House members and ask them to “consider S.2465 for a vote this year.” The new changes to the bill requires the House to vote on it again.

About the bill

S. 2465 would authorize the Secretary of Health and Human Services to conduct surveillance and collect data on the prevalence of sickle cell disease (SCD). In addition, the bill would authorize the Secretary to develop public health initiatives that support community-based organizations in education activities and to support regional and state health departments in testing to identify SCD.

Key Talking Points about S.2465

● S. 2465 – Sickle Cell Disease and Other Heritable Blood Disorders Research, Surveillance, Prevention, and Treatment Act of 2018 was introduced on February 28, 2018 by Sen Tim Scott [R-SC] and cosponsored by Sen. Booker, Cory A. [D-NJ]; Sen. Jones, Doug [D-AL]; Sen. Cassidy, Bill [R-LA]; Sen. Stabenow, Debbie [D-MI]; and Sen. Warren, Elizabeth [D-MA]
● S. 2465 represents a commitment by the government to continue much-needed research geared towards increasing understanding of prevalence, distribution, outcomes, and therapies associated with SCD
○ The legislation reauthorizes SCD prevention and treatment grants awarded by the Health Resources and Service Administration (HRSA)
○ Authorizes the Centers for Disease Control and Prevention (CDC) to award SCD surveillance grants to states, academic institutions, and non-profit organizations
● It is important that the House vote on the bill this year. The ultimate goal is the passage by both Congressional houses and signed into law by the President.


The National Foundation for Ectodermal Dysplasias Asks Advocates to Contact Congress to Support the Ensuring Lasting Smiles Act

The National Foundation for Ectodermal Dysplasias (NFED) asks you to write your member of Congress and ask them to co-sponsor the Ensuring Lasting Smiles Act (S.3369/H.R.6689). The Ensuring Lasting Smiles Act (also known as ELSA) will assure that individuals born with congenital anomalies receive health benefits for the medically necessary treatments they need. ELSA would require all groups and individual health care plans to covers procedures to restore or repair normal body function for any missing or abnormal body part, including teeth. It closes a loophole that allows health care plans to deny such procedures as “cosmetic”. The NFED, American Association of Oral and Maxillofacial Surgeons, American Society of Plastic Surgeons and seven organizations are supporting this bi-partisan legislation.

Click here to take action.

The National Leiomyosarcoma Foundation is Asking Patient Advocates to Call Their Members of Congress in Support of Resolution Establishing National Sarcoma Awareness Month

The National Leiomyosarcoma Foundation is asking patient advocates to call their Members of Congress in support of a resolution to create a National Sarcoma Awareness Month and a National Leiomyosarcoma Awareness Day in 2018.  A National Sarcoma Awareness Month for JULY would allow awareness and support on a national scale.

Leiomyosarcoma Awareness Day-July 15 was granted by Congress in 2016. This year, the focus and hope is for a National Sarcoma Awareness Month of July and a National and Leiomyosarcoma Awareness Day to follow for July 15, 2018. Click here to read a letter from the National Leiomyosarcoma Foundation along with the resolution. 

1. Sarcoma is a designated rare cancer because it represents 1% of all cancers.
2. There are between 50 – 70 sarcoma subtypes. Some researchers have even indicated possibly
Up to 100 lately.
3. Sarcomas know no age boundaries – children, young adults, older adults are diagnosed
4. It is expected that the increase in sarcoma diagnoses will continue to rise annually.
5. Sarcoma is difficult to treat and the life expectancy after diagnosis is short.
6. For example, for Leiomyosarcoma, there is a 64% survival rate of up to 5 years.
For Leiomyosarcoma, there is no immunotherapy that is working yet.
7. Since sarcoma is a rare cancer, it is important for the patient population to be
heard, and the disease better understood throughout communities.
8. No longer having a ” rare voice” in the cancer landscape can help spotlight sarcoma and its
Many subtypes – for national awareness, support, and elevated funding to accelerate treatment
options that can advance survival rates.
9. The results of this are evident in the common cancers – breast cancer especially.
10. Rare Cancer would benefit from the same level of involvement in awareness, support, funding.


Children’s Cause Cancer Advocacy is Encouraging Patients to Ask Their Members of Congress to Support the STAR Act

The Childhood Cancer STAR Act is designed to advance pediatric cancer research and child-focused cancer treatments, while also improving childhood cancer surveillance and providing enhanced resources for survivors. Children’s Cause Cancer Advocacy is asking advocates to write to their Members of Congress to voice their support.

Click here to take action.

EveryLife Foundation Urges Advocates to Ask Their Members of Congress to Support the OPEN ACT

The EveryLife Foundation asks you to take action to support the Orphan Product Extensions Now, Accelerating Cures and Treatments (OPEN ACT, H.R. 1223 / S. 1509). The OPEN ACT could bring hundreds of safe, effective, and affordable medicines to rare disease patients within the next several years by incentivizing drug makers to repurpose therapies for the treatment of life-threatening rare diseases. EveryLife Foundation, the National Organization for Rare Disorders (NORD), Global Genes, and Genetic Alliance are among the more than 220 patient organizations support this bipartisan legislation.

For further information on the OPEN ACT, click here.


The EveryLife Foundation, NORD and Global Genes are Asking Patient Advocates to Tell Congress to Save Orphan Drugs by GIVING THANKS for Rare Disease Treatments

The Orphan Drug Tax Credit (ODTC) is a key provision of the Orphan Drug Act, and promotes research spending on rare diseases by lowering development costs for manufacturers – but this is in jeopardy: the tax reform bill passed in November by the House would eliminate the credit altogether, and the Senate bill, which passed earlier this month, cut it nearly in half.

The EveryLife Foundation for Rare Diseases, NORD and Global Genes feel that without the Orphan Drug Tax Credit, investment in therapies for rare disease patients is at risk!  Before the Orphan Drug Act, manufacturers were often hesitant to invest in developing new treatments for rare diseases because the small patient population made it difficult to recover development costs.

TAKE ACTION NOW to preserve the ODTC by telling Congress why YOU are grateful for it!  Use #RareGratitude and put it to good use!

Here are 4 things you can do to show your #RareGratitude.  It only takes a few minutes to help 30 million Americans waiting for treatments to be developed.

  • Tweet your #RareGratitude!
    • Get inspired with the sample posts below. Tag your Senator and Representative by checking their username here.  Ask your followers to RT!
  • Facebook
    • Post a picture of yourself or your family holding this sign to show your #RareGratitude! Tag your Senator and Representative by checking their username here.
    • Visit your Senator’s and Rep’s pages by checking their username here, and post in the Comments section using a sample post below.
    • Ask your friends and family to do the same, or to share your post.
  • Email your Senator and Representative
  • Call your Senator and Representative


  • Orphan Drugs saved my life. Now we have to save the Orphan Drug Tax Credit for the 95% who don’t have treatments yet. #RareGratitude #SaveOrphanDrugs
  • #SaveOrphanDrugs for 30M Americans w/ #rarediseases who rely on future development of #OrphanDrugs: org/saveorphandrugs #RareGratitude
  • 30M Americans are counting on the Orphan Drug Tax Credit to save their lives. #RareGratitude #SaveOrphanDrugs
  • I’m giving thanks for the 600 new treatments developed because of the Orphan Drug Tax Credit. #RareGratitude. Preserve the ODTC for the 6,400 still needed! #SaveOrphanDrugs
  • The #OrphanDrug Tax Credit gives me hope for a future treatment for my #raredisease. #RareGratitude. Please #SaveOrphanDrugs {insert picture of yourself}
  • I am a #raredisease patient who does not have a treatment. I’m giving thanks for the #OrphanDrug Tax Credit, because it gives me hope for the future. #RareGratitude. Please #SaveOrphanDrugs!
  • #RareGratitude means giving thanks this holiday for #OrphanDrugs. Please RT & take action to #SaveOrphanDrugs. Millions of #raredisease patients rely on it for the hope of a future treatment! Call Congress using org/saveorphandrugs


Every action showing #RareGratitude helps #SaveOrphanDrugs and millions of lives!  THANK YOU!

Danny’s Dose is Asking MN Patient Advocates to Call Their State Legislators in Support of EMS Protocol Bills

HB972 and SF1023 are EMS Protocol Bills that support individuals with “special medical needs.” In order to preserve these bills in their current state, Danny’s Dose is encouraging advocates to call or email their legislators to voice their support. If authors change legislative language to only cover Adrenal Insufficiency, thousands of patients could be left unprotected.


(area code is 651)

House Bill – HB972    

Author: Rep. Clark Johnson   –  296-8634


Senate Bill – SF1023 

Author: Sen. Nick Frentz   –  296-6153


Co-Authors: Sen. Julie Rosen   –  296-5713

Sen. Tony Lourey  –  296-0293

Sen. Scott Jensen  –  296-4837**

Sen. Michelle Benson  –  296-3219**


Click here to look up your Minnesota legislators. For questions please contact Darlene Shelton at

Call on Your Members of Congress to Attend the November Rare Disease Congressional Caucus Briefing!

On November 15th, the Rare Disease Congressional Caucus will host a briefing on “Diagnostic Challenges for Rare Disease Patients.” This event will brief Members of Congress and staff on challenges to diagnosing rare disease, emerging technologies and possible policy solutions. Rare disease patients often have to see multiple doctors over many years before they receive a diagnosis.

You can find out more about the event by CLICKING HERE or contact Sabah at the EveryLife Foundation at SBhatnagar@everylifefoundation.

Please take a minute to phone your legislators and urge them to send staff to the event!

NORD is Asking Patient Advocates to Encourage Their Senators to Save the Orphan Drug Tax Credit

NORD is encouraging patient advocates to call, email and tweet their Senators in support of the Orphan Drug Tax Credit (ODTC). Yesterday, the House passed a tax reform bill that would get rid of the ODTC, a critical incentive for orphan drug creation. These provisions are now being considered by the Senate. The ODTC allows drug manufacturers to claim a tax credit of 50 percent of the qualified costs of clinical research and drug testing of orphan drugs (drugs for diseases affecting 200,000 Americans or fewer). The ODTC is part of a package of provisions enacted in 1983 in the Orphan Drug Act (ODA) that provides incentives for drug companies to develop products for rare diseases. This legislation has been extremely successful.