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.

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

On September 13th, the Rare Disease Congressional Caucus will host a briefing on “Curing Rare Disease: Policy and Regulation Needed for Emerging Technology.” This event will brief Members of Congress and staff on the need for policy and regulation that does not only keep pace in this new era of innovation, but also ensures that patients benefit the application of new technologies to safe and effective treatments.

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

SYNGAP Asks Patient Advocates to Sign Letter on Quality-Based Physician Reimbursement

When Congress replaced the Sustainable Growth Rate (SGR) formula for how clinicians got their annual raises and bonuses (and penalties), they replaced it with a system where clinicians had to meet certain quality measures, make improvements to their practices, use technology to aid the patient experience, etc. Now kicking off it’s second year of this very different approach to physician payments, CMS is asking for suggestions. In this letter, SYNGAP asks CMS to recognize that clinicians will not always have quality measures for rare and ultra rare diseases, their costs may be greater to treat our patients, and practice improvements or technology adoption for just a few patients may be more difficult to implement. The system should not fail those clinicians who are treating ultra rare patients.  SYNGAP recommends some common sense changes for CMS to adopt related to how clinicians can still get their raises and bonuses when treating rare and ultra rare patients.

Click here to see sign-on letter.

Please email Monica Weldon at if you have questions and/or would like to sign this letter by August 20.

The Alliance for a Stronger FDA Encourages Advocates to Ask Their Legislators for Robust FDA Appropriations

The Alliance for a Stronger FDA is encouraging patient advocates to ask their legislators to support robust funding for the Food and Drug Administration (FDA). The FDA plays a critical role for rare disease patients because:

  1. The Agency ensures access to safe and effective medical products and is key to preserving public health.
  2. FDA-regulated industries are world leaders in discovery and innovation in large part because of the stability and oversight provided by FDA.
  3. FDA’s well-trained staff is indispensable. The products overseen by FDA cannot be evaluated without specialized scientific and technical knowledge.

In order for patients to have access to innovative treatments, robust appropriations funding for the FDA is critical. To read the Alliance for a Stronger FDA update on challenges and threats to the FDA, click here.

To send a a note to your legislators, click “take action.”

Rare New England Asks Massachusetts Residents to Contact Their State Legislators in Support of MA Rare Disease Advisory Council

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, click here.

SYNGAP Asks Patient Advocates to Sign Letter to Change Hospital Reimbursement System

Hospitals are paid today according to an antiquated system set up in 1983, the same year the Orphan Drug Act was passed to incentivize the development of orphan disease treatments. We’ve come a long way in getting those treatments, but nothing about hospital reimbursement has changed in all that time. The more extremely rare a condition and patient, the more a hospital may want to turn them away with minimal care.

Please sign this letter to kick off an effort to change this almost 35 year old inpatient hospital payment system so that hospitals are able to care for rare patients like the ones we serve.

Click here to see sign-on letter.

Please email Monica Weldon at if you have questions and/or would like to sign this letter.

Click here for more information about Bridge the Gap- SYNGAP Education and Research Foundation.

NORD Asks Advocates to Contact Their Senators to Urge Them to Vote NO on the Better Care Reconciliation Act (BCRA).

NORD asks advocates to call, email or message their Senators to urge them to vote no on the Better Care Reconciliation Act (BCRA). The Senate released the Better Care Reconciliation Act (BCRA), an amended version of the American Health Care Act (AHCA) that passed the House, earlier in June.

NORD believes that, if enacted, the bill will harm rare disease patients by cutting hundreds of billions of dollars from Medicaid, and removing key pre-existing condition protections. You can read NORD’s statement here.

Click here for more information on ways you can take action.

CAL Rare Asks California Residents to Have Their State Representatives Join the Rare Disease California Caucus

The EveryLife Foundation and CAL Rare are urging California advocates to have their state representatives join the Rare Disease California Caucus.  The bipartisan Rare Disease California Caucus is led by Assemblymember and Health Committee Member Rob Bonta (D-Oakland) and Assemblymember and Health Committee Vice Chair Brian Maienschein (R-San Diego) to promote awareness of rare disease issues.

The Rare Disease California Caucus will help to bring public and legislative awareness to the unique needs of the rare disease community – patients, physicians, scientists, and industry. The Caucus will give a permanent voice to the rare disease community in California. Working together, we can find solutions that turn hope into treatments.

Please take just 3 minutes to email your legislators today to ask them to join the Caucus!



EveryLife Foundation Asks Advocates to Have Their Members of Congress Co-Sponsor the OPEN ACT

From the EveryLife Foundation for Rare Diseases:

Ask Your Representative to Co-Sponsor the OPEN ACT to Repurpose Drugs for Rare Disease Patients

Take action to support the Orphan Product Extensions Now, Accelerating Cures and Treatments (OPEN ACT; HR 1223).  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 and pediatric cancers. The OPEN ACT could allow for insurance reimbursement of otherwise off-label treatments or procedures, and reduce the cost of orphan therapies. EveryLife Foundation, the National Organization for Rare Disorders (NORD), Global Genes and Genetic Alliance are among the more than 220 patient organizations that support this bipartisan legislation.

To contact your Representative in support of the OPEN ACT, click here.

For further information on the OPEN ACT, click here.


March of Dimes Asks Organizations to Sign-on to Letter Rejecting Proposed Cuts to Newborn Screening Programs

March of Dimes calls on organizations to sign-on to a letter to Congress to oppose the proposed budget cuts included in President Trump’s fiscal year (FY) 2018 budget that would eliminate newborn screening programs. Specifically, they ask that you reject the elimination of the Health Resources and Services Administration’s (HRSA) Heritable Disorders program.

If enacted, the cuts would result in adverse health outcomes for many of our nation’s infants. Elimination of the Heritable Disorders program would roll back state newborn screening progress and limit states’ ability to quickly add new conditions to their newborn screening panels.

To join the organizational sign-on letter, please email Rebecca Abbott, ( by COB Wednesday, June 28th, and include your organization’s name as you would like it to appear on the letter. Please feel free to share with your networks!

Letter available here.




NORD Asks You to Call Your Senator to Protect Medicaid

The National Organization for Rare Disorders (NORD), is seeking individuals to call their Senator to protect Medicaid from harmful cuts.

Last month, the House passed the American Health Care Act (AHCA) which included billions of dollars of funding cuts to your state’s Medicaid program. The Senate is currently considering passing the same or similar legislation, which could delay or deny access to vital care for some of our most vulnerable citizens, individuals with rare diseases.

To identify your legislators and obtain their contact information, click here.