Travel Stipends for Rare Disease Week on Capitol Hill Increased!

The EveryLife Foundation for Rare Diseases will provide travel stipends to enable advocates from across the country to participate in Rare Disease Week on Capitol Hill, to be held February 27th through March 2nd, 2017.

Thanks to a generous donation to our Rare Giving program by Horizon Pharma, we are able to increase the amount offered to recipients! Advocates in Maryland and Virginia are eligible to receive $400 stipends, while advocates in other states in the continental U.S. are eligible to receive $800. Advocates in Alaska, Hawaii and Puerto Rico are eligible to receive $1,000 stipends.

In order to receive a stipend, advocates must attend the Legislative Conference and Lobby Day. Applications are available online through December 18th. All applicants will be notified whether they will receive a stipend or are on the waitlist on January 3rd.

We hosted a webinar earlier this month to provide an overview of the events during Rare Disease Week on Capitol Hill, highlight travel options to Washington, DC and answer questions.  You can find the webinar recording and schedule of events online.

We hope you will plan to join us to educate the new Congress and help shape healthcare policy to better meet the needs of the rare disease community. You can sign up to receive news from Rare Disease Legislative Advocates to ensure that you don’t miss any updates on Rare Disease Week on Capitol Hill. You can also follow us on Twitter and Facebook.

Thank you to

President Obama Signs Landmark 21st Century Cures Bill into Law

NEVER doubt that your voice as a rare disease advocate matters! President Obama just signed the 21st Century Cures Act into law, after it passed both the House and Senate with broad bipartisan support. This would not have been possible without advocates from EveryLife Foundation for Rare Diseases, Global Genes, National Organization for Rare Disorders, Inc. (NORD) and many disease-specific organizations who called, emailed and met with Members of Congress in the past year and a half.

The 21st Century Cures Act includes:

  • $4.8 billion in new funding for the National Institutes of Health (NIH)
  • $500 million in new funding for the Food and Drug Administration (FDA)
  • Formally establishes the Precision Medicine and Cancer Moonshot initiatives
  • Reauthorization of the Rare Pediatric Disease Priority Review Voucher program through 2020
  • Funding for the establishment of a national neurological disease surveillance system coordinated by the Centers for Disease Control and Prevention (CDC)
  • Improved biomarker qualification
  • Allowances for the FDA to recruit and retain additional specialized employees
  • Strengthened patient engagement at the FDA through the Patient Focused Impact Assessment Act
  • A regenerative medicine designation to allow such products to qualify for priority review and accelerated approval
  • Provisions to foster programs to improve mental health and deter substance abuse

These provisions and additional funding would boost our nation’s research capacity and help modernize the drug review and approval process at the FDA.

How should Congress use its lame-duck session? First, it can save lives.

Originally published in the Washington Post:

REPUBLICANS ARE signaling that they will pursue an ambitious conservative agenda when they take the reins of government next year. But before that happens, the current Congress will convene in its lame-duck session, valuable legislative time that Senate Majority Leader Mitch McConnell (R-Ky.) said this week he hopes to put to good use.

The session may be consumed by arguments over federal budgeting. But if there is time for anything else, Mr. McConnell may push the 21st Century Cures Act, a bipartisan effort that has taken years to get close to passing. Congress should nudge it across the finish line — taking care to repair a few problems along the way.

The act, a version of which passed in the House last year, proposes a one-time, multibillion-dollar increase in funding for the National Institutes of Health. The money could provide a sharp boost to the Obama administration’s cancer initiative, or to research into precision medicine, which tailors treatments to people’s genomes. Rapid progress in both is possible and could save many lives; new cancer drugs have emerged targeting specific mutations in tumor cells, and they have shown encouraging initial results in treating even some of the most complex cancers. The new funding could also go into competitive grants for scientists with particularly innovative projects that are nevertheless underfunded. Given that so many lifesaving pharmaceuticals have their origins in government-sponsored scientific research, the funding boost would be a good investment.

The new money alone, however, would struggle to attract strong bipartisan backing. So lawmakers linked it to various reforms of the Food and Drug Administration’s approval process, arguing that the agency has been hamstrung in getting new drugs to market. One reform on the table would adjust hiring standards at the FDA, which is perpetually short-staffed. The agency has improved on the time it takes to approve new medications, but a more flexible hiring policy could help further. There are also worthwhile provisions that would give gravely sick patients with few options easier access to experimental medication.

Critics have raised some valid concerns. For example, lawmakers should ensure that a proposed adjustment to rules on the approval and use of new antibiotics does not have the unintended side effect of encouraging antibiotic overuse and resistance — the very problem the provision is supposed to combat.

The bill’s backers insist that the FDA’s bottom-line legal mandate would continue to ensure that drugs were safe and effective, even as the agency was granted more flexibility in meeting that standard. This means, then, that it would be up to the FDA to use its new powers wisely.

Despite the caveats, though, the act is worth supporting. If the lame-duck Senate can pass its version and merge it with the House’s, addressing some of the concerns in the process, it would be a valuable use of Congress’s time.

Sign your Organization to Support the Rare Pediatric Disease Priority Review Voucher Program in 21st Century Cures!

The National Organization for Rare Diseases (NORD) has drafted a sign-on letter for organizations to show their support in reauthorizing the Rare Pediatric Disease Priority Review Voucher program as part of 21st Century Cures. NORD will distribute this letter throughout Congress in mid-November, so they urge your support in the next week.

CLICK HERE to sign your organization onto this letter today!

Apply Today for a Travel Stipend for Rare Disease Week on Capitol Hill 2017

Please mark your calendar and plan to join us for Rare Disease Week on Capitol Hill, to be held February 27 through March 2, 2017 in Washington, DC.  The week of events brings together rare disease community members from across the country to be educated on federal legislative issues, meet other advocates, and share their unique stories with legislators. There will be new Members of the House and Senate next year, and it is critical for them to meet members of their communities affected by rare disease.

All events are free for patient advocates, and registration will open in early January.

Applications for Travel Stipends Now Open

The EveryLife Foundation is now accepting applications for travel stipends!  We awarded more than $55k in stipends this year, and hope to enable even more advocates to join us in Washington, DC next year. Patients, caregivers, and others in the rare disease community can apply online. The deadline to apply is December 18th, and all applicants will be notified in early January. Please note that stipends are limited to one per family, and attendance at the Legislative Conference and Lobby Day is required.

Not Able to Attend?

We want every Member of Congress to hear from constituents affected by rare disease, and you can help even if you can’t join us in person. Please share your unique perspective and let your legislators know what issues matter most to you by filling out our online form by February 12th so that we can hand-deliver it on the Lobby Day.  And please share this opportunity with your network to make sure the voice of your rare disease community is heard!

Please sign up for our email list or check the Rare Disease Week on Capitol Hill webpage for more information and updates.

Video from Rare Disease Congressional Caucus Briefing on Strengthening Medical Innovation Now Available

On September 14, 2016, the Rare Disease Congressional Caucus gathered a contingent of thought-leaders in the rare disease space to discuss Strengthening Medical Innovation in America for Rare Disease Patients.

Energy and Commerce Committee Chairman’s Open Letter to Advocates on #CuresNow

Rare disease advocates across the country, including Emily Muller and Sharon Rose Nissley of Illinois as well as Shira Strongin of California, are helping to generate support for 21st Century Cures. And their efforts, as well as those of advocates meeting with Members of Congress and their staff in In-District Lobby Days meetings, are making a difference!

As U.S. House of Representatives Energy and Commerce Committee Chairman Fred Upton wrote in a recent editorial:

“Thanks to a remarkable outpouring and mobilization of rare disease advocates, August 2016 will go down in the books as the “Summer of Cures”… Together, we’ve made incredible strides in our effort to deliver #CuresNow. Every story, every single voice matters, and we are grateful for your support and willingness to share your personal experiences with disease.”

But time is running out for the Senate to act. As Chairman Upton noted, “It’s been a tremendous effort by the rare disease community and I am proud to report that we are closing in on the finish line. But we can’t get there without everyone’s continued efforts — now is the time to double down on all our efforts.

This month, the EveryLife Foundation for Rare Diseases is asking patients and advocates to submit photos and hashtag them with #CuresNOW. Lives are depending on it.

Take Part in the Rally for Medical Research!

Held every September, this Capitol Hill Day event continues the momentum established in 2013, and includes nearly 300 national organizations coming together in support of the Rally for Medical Research. Led by the American Association for Cancer Research, the purpose of the Rally is to call on our nation’s policymakers to make funding for National Institutes of Health (NIH) a national priority and raise awareness about the importance of continued investment in medical research that leads to MORE PROGRESS, MORE HOPE and MORE LIVES SAVED.

Click here to get learn more and get registered today!

Ask Congress to Fund More Childhood Cancer Research

When Congress returns from their August recess, they will be considering how much funding to provide the National Institutes of Health — and the National Cancer Institute for 2017. As reported on the Children’s Cause Cancer Advocacy blog, a House Appropriations committee recently approved a spending bill that would increase NIH funding by $1.25 billion, while Senate appropriators would increase the NIH budget by $2 billion.

Children’s Cause Cancer Advocacy is urging advocates to write to their Senators and Representatives and ask them to support the higher Senate number, so that the NIH and NCI continue to work for new treatments and explore the promise of immunotherapy.

Take action now and tell Congress why each additional research dollar matters for children with cancer.

Then, consider scheduling a district office meeting during this August recess. Face-to-face meetings with your elected officials and their staff in the district office are an extremely effective way to get to know them and express your views on key issues like this one. CCCA’s new step-by-step Guide to Congressional District Meetings can help you!

Join the coalition in support of a National Emergency Treatment Database

The Cambria Lord Foundation invites rare disease non-profits nationwide to join in their efforts to establish a National Emergency Treatment Database for rare disease patients. If your organization is interested in joining the coalition, visit their website here, or email wear4rare365@cambrialordfoundation.org!