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.

Please ask your California State Legislators to Join the Bipartisan State Rare Disease Caucus

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. Help us strengthen the rare disease community’s voice by inviting your legislators to join the bipartisan Caucus.

Click here to invite YOUR legislators to join the bipartisan Caucus!


Research!America Shares Sign-On Letter Urging Congressional Leaders to Boost Funding for Health Research Agencies

Research!America, is seeking organizations to sign-on and share this letter that makes the case for a bipartisan FY18 budget deal that increases the spending caps and overrides sequestration. To join the organizational sign-on letter, please email Jacqueline Lagoy ( by COB Friday, June 16, and include your organization’s name as you would like it to appear on the letter.


NORD Urges Advocates to Ask Members of Congress to Vote NO on Revised AHCA

From the National Organization for Rare Disorders:

Tell Your Congressman to Please Vote NO on Revised AHCA

Congress is currently reviewing the newest version of the American Health Care Act for a vote later this week, and we need your help.

The new proposal (known as the MacArthur Amendment) could substantially roll back protections for individuals with pre-existing conditions. The bill would allow states to set their own Essential Health Benefit (EHB) categories. The Affordable Care Act eliminated annual and lifetime caps, but only for services that fell in the ten EHB categories. If the EHBs are turned over to states, states could choose to allow insurers to once again set annual and lifetime caps on medically necessary services for rare disease patients by only mandating coverage of certain EHBs.

The bill would also allow states to once again permit insurers to charge higher premiums to sicker individuals. Patients may again face entirely unaffordable premiums if this proposal passes. 

This would be devastating for individuals with rare diseases. Medically necessary prescription drugs and medical care could go uncovered and insurers could once again charge thousands of dollars per month.

Lawmakers could vote THIS WEEK. Please take a minute to phone your legislators and ask them to vote no!

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

Coalition for Health Funding Asks Orgs to Sign-On for Additional HHS Funding

From the Coalition for Health Funding:

Sign Letter in Support of Increased Funding for Labor, HHS, Education and Related Agencies Appropriations

Attached for your consideration is a letter—being circulated throughout the health, education, child development, social services, and workforce communities—urging appropriators increase the allocation for the Labor, HHS, Education and Related Agencies Appropriations Subcommittee for fiscal year (FY) 2018.


Please note that in the interest of expediency, and as a courtesy to the hundreds or organizations that will sign, we will not accept editorial changes to the letter. Signers must agree to sign the letter “as is.”

Below are some FAQs to help you understand this advocacy effort, and build support for the letter within your organization and throughout the community.

What is the 302(b) letter?

The 302(b) allocation is the amount of funding the House and Senate Appropriations Committees provide to the subcommittees. The subcommittees then divvy up the funding between the agencies and programs within their respective jurisdictions. Each year, the Committee on Education Funding, the Coalition for Health Funding, and the Campaign to Invest in America’s Workforce partner to circulate a letter throughout our respective communities in support of strong 302(b) allocations for the House and Senate Labor-HHS-Education Appropriations Subcommittees. The bigger the allocations, the better our chances of securing funding for our agencies and programs; or in the current environment, preventing further cuts.

Why should my organization sign the letter?

At the allocation stage in the appropriations process, discretionary health programs are competing for funding with all of the other nondefense discretionary programs within the austere spending caps. If you care about federal funding for any/all public health, health research, or social safety net programs you should sign this letter. Without a strong 302(b) allocation for Labor-HHS-Education, there is very little chance of increasing funding for your priorities. And there is an even greater likelihood that your priorities will be cut.

What happens once my organization signs?

All signatures will be added to the letter and sent to the House and Senate Appropriations Committees in early May. We will forward you a copy of the letter for your records. A copy will also be posted on the Coalition for Health Funding’s website:

What can I do to help with this effort?

Sign on of course, and/or forward this message and letter far and wide. We are accepting signatures from national, state, and local organizations, institutions, businesses, companies, etc. Essentially, any entity that cares about funding for programs in the Labor-HHS-Education appropriations bill. But no individuals, please!

What is the Coalition for Health Funding?

Established in 1970, the Coalition for Health Funding is the oldest and largest nonprofit alliance working to preserve and strengthen public health investments in the best interest of all Americans. Our dues-paying member organizations together represent more than 100 million patients and consumers, health providers, professionals, and researchers. Please consider joining us as we fight to protect federal investments in health. Learn more about membership at

Questions, please contact Emily Holubowich, Executive Director, Coalition for Health Funding at

NHC and NORD Share PDUFA Sign-On Letter

From the National Health Council (NHC) and National Organization for Rare Disorders (NORD):

People with chronic and rare diseases and disabilities rely on FDA to access innovative, safe, and effective treatments. Not only do user fees provide the funding for FDA to quickly review products, but the current user fee agreements also provide significant improvements and advancements to the health care community that simply cannot wait.

For more information about the letter and to sign using our online form, please click here. We ask that all signatures be submitted by April 20.

Learn more at:

Research!America Urges Congress to Pass FY2017 Budget

From Research!America:

Instead of completing work on an updated budget for Fiscal Year 2017 (FY17) before 2016 ended, Congress passed a temporary stop-gap or continuing resolution (CR) that flat-funds government until April 28, 2017. A draft FY17 “omnibus” spending bill that would direct more funding to National Institutes of Health (NIH) research and other key research and public health priorities sits waiting for further Congressional action. Congress has indicated they are nearing the finish line on this legislation, which needs to be signed into law by midnight on April 28th or the government will shut down. Let them know you support their efforts to get the job done!

Click here to take action!

NORD Asks Congress to Keep Medical and Genetic Privacy Protections Intact

From the National Organization for Rare Disorders (NORD) action alert:

Federal lawmakers are currently considering a bill that intends to clarify regulations of employee wellness programs, but instead threatens our medical and genetic privacy.

The Preserving Employee Wellness Programs Act (H.R. 1313) would exempt employee wellness programs from critical patient protections included in the Americans with Disabilities Act (ADA) and Genetic Information Nondiscrimination Act (GINA). This bill would roll back decades of critical patient privacy protections and allow employers to collect information on their employee’s genetic makeup and medical history.

The privacy of millions of individuals with rare diseases could be violated by making incredibly personal and private genetic information legally accessible to employers under the threat of increased premiums. For patients with genetically detectable rare diseases or predispositions to such diseases, privacy is crucially important for individual independence, dignity, and protecting against workplace discrimination.

This bill intends to clarify regulation of wellness programs, but instead could significantly undercut patient privacy protection for employees at high risk for workplace and societal discrimination given the nature and severity of their illnesses or risk of illness.

Please join us in telling Congress that this is unacceptable. Tell your U.S. Senators and Representative to oppose H.R.1313, and stand up for the privacy of patients.

Click here to take action!

Kids v Cancer is asking YOU to support the RACE for Children Act

The RACE for Children Act (Research to Accelerate Cures and Equity Act) would provide that companies developing a cancer drugs would undertake Pediatric Research Equity Act studies of their drug in children when the molecular target of their drug is relevant to a children’s cancer. To learn more and sign on in support, click here.

The EveryLife Foundation seeks your support to end the NIH/FDA hiring freeze

On January 23rd, President Trump instituted an immediate hiring freeze that would affect many federal agencies including the National Institutes of Health (NIH) and the Food and Drug Administration (FDA). Unfortunately, this freeze could significantly hamper research into rare disease treatments as well as the review and approval of new medicines. The EveryLife Foundation for Rare Diseases is asking patient organizations to sign-on to a letter describing the critical importance of the NIH and FDA along with their ability to hire new staff.

CLICK HERE to sign your organization on to the Foundation’s letter!

The Foundation is accepting signatures until Friday, February 24th, so sign on today! Your voice provides needed perspective on how the hiring freeze could harm rare disease patients. Share this action alert with your networks and on social media.

Join Research!America in calling for a standing fund to enable CDC/NIH response in public health emergencies

Earlier this week, National Institute of Allergy and Infectious Diseases (NIAID) Director Anthony Fauci, M.D. made the case for a standing fund to enable rapid Centers for Disease Control and Prevention (CDC) and National Institutes of Health (NIH) response in the event of public health emergencies. Research!America has issued an action alert to echo his call to action.

Click here to support the standing CDC and NIH fund to respond to public health emergencies!