September 20: Monthly Legislative Meeting

RDLA’s September Legislative Webinar and In-Person Meeting
Wednesday, September 20th, 2017
12:00 – 1:00 pm ET

1. The Haystack Project: Bridging Innovation and Patient Access, Saira Sultan, President and CEO, Connect 4 Strategies

2. The Orphan Drug Tax Credit, Paul Melmeyer, Director of Federal Policy, National Organization for Rare Disorders (NORD)

3. The California EXPERRT Act, Siri Vaeth, Programs and Outreach Manager at Cystic Fibrosis Research, Inc.

4. The Children’s Health Insurance Program (CHIP), Marielle Kress, Assistant Director of Federal Affairs, American Academy of Pediatrics

5. Rare Disease Week on Capitol Hill, Stephanie Fischer, Senior Director of Patient Engagement and Communications, EveryLife Foundation for Rare Diseases

6. The 6th Annual RareVoice Awards, Sabah Bhatnagar, Program Director, Rare Disease Legislative Advocates, EveryLife Foundation for Rare Diseases

9.20.17 Slide Deck

Once a month, RDLA convenes in person and/or over the phone to discuss legislation and developments that affect the rare disease community. The meeting/conference calls are essentially a clearing house for legislation and participation does not imply support for any of the policy proposals or legislation that are discussed or promoted.

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 monicaw@bridgesyngap.org if you have questions and/or would like to sign this letter by August 20.

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.

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, (rabbott@marchofdimes.org) 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.

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 (jlagoy@researchamerica.org) by COB Friday, June 16, and include your organization’s name as you would like it to appear on the letter.

 

Debra of America Shares Sign-On Letter for H.R. 1703, the Medical Product Communications Act of 2017

The Dystrophic Epidermolysis Bullosa Research Association of America (debra of America) is seeking other patient advocacy organizations to sign-on in support of H.R. 1703, the Medical Product Communications Act of 2017. The letter of support is available here and any organization interested in signing can contact Joe Murray at joe@debra.org. The deadline to sign is close of business on May 10th.

Rare Disease Week on Capitol Hill Engaged 600+ Advocates

Thank you to the 600+ rare disease patients, caregivers, researchers and other advocates who joined us for at least one event during Rare Disease Week on Capitol Hill in Washington, DC from Monday, February 27th, through Thursday, March 2nd.

The week began at the National Institutes of Health (NIH), where the EveryLife Foundation’s Chief Advocacy and Science Policy Officer presented an update on our work to improve the state newborn screening system and create incentives to encourage biopharmaceutical companies to repurpose approved medicines for rare diseases.

That evening, we hosted a cocktail reception, screening of the documentary Up for Air, and panel discussion. Senator Ed Markey (D-MA) and Representative Jim McGovern (D-MA) made brief remarks.

Aproximately 350 advocates joined us for the Legislative Conference on Tuesday, which was livestreamed for the first time. Experts from Capitol Hill and patient advocacy organizations discussed what to expect from the new Congress and Trump Administration, how to build effective relationships with Members of Congress and staff, and key legislation. Video and presentations will be available on the Legislative Conference resource page.

Advocates began Lobby Day at breakfast with remarks by Dr. Janet Woodcock, Director of the Food and Drug Administration’s Center for Drug Evaluation and Research, as well as Representative Gus Bilirakis (R-FL) and former Representative Brian Baird (D-WA), who urged advocates to be brief, polite and persistent in their meetings with Members of Congress and staff.

328 rare disease advocates participated in 270 Lobby Day meetings, discussing the Rare Disease Congressional Caucus, healthcare reform, incentives for rare disease drug development and other key legislative topics.

On Wednesday evening, we hosted the annual Rare Artist Reception which featured winning entries from the 2016 contest and remarks from several of the artists.

The final event of the week was a Rare Disease Congressional Caucus briefing entitled, ” Advancing Rare Disease Treatments in the Era of Cures and Health Care Reform.” Caucus Co-Chair Senator Amy Klobuchar (D-MN) welcomed advocates and Congressional staff. Video will be posted on the Caucus webpage within a few weeks.

Follow Rare Disease Legislative Advocates on Twitter and Facebook for news on upcoming events.

Top Dem: Cures bill funding cut to $4B

Originally published in The Hill:

A top Democratic negotiator said on September 28th that new funding in a major medical cures bill has been cut significantly as lawmakers look for a path for passage.

Rep. Gene Green, the top Democrat on the House Energy and Commerce health subcommittee, told The Hill that a new version of the 21st Century Cures bill will allocate about $4 billion over five years for research at the National Institutes of Health (NIH), down from the original $8.75 billion.

He also said funding for the Food and Drug Administration (FDA) is down to $300 million, from about $500 million in the original bill.

However, as negotiations have continued, the final number could end up higher.

“We’re working to finalize the Cures package, so any numbers would be preliminary to share,” a committee spokesperson said.

Lawmakers are looking for a bipartisan deal to move this slimmed down version of the bill when Congress returns for a lame-duck session after the elections. The measure could be a way to fund medical research priorities such as Vice President Joe Biden’s cancer “moonshot.”

The original version of the bill, which seeks to accelerate the FDA’s approval process for new drugs and invest in medical research, passed the House on a bipartisan vote last year.

But it has been mired in the Senate amid months of negotiations over a bipartisan way to pay for the new spending.

Sensing that the clock is ticking, House Energy and Commerce Chairman Fred Upton (R-Mich.), who has made the bill his signature issue, is looking to jumpstart the process by passing a new, slimmed down bill through the House in consultation with the Senate. The upper chamber could then take up that new measure.

“We’re only here a week in November, so that will be the week we need to deal with it, so that’s what Chairman Upton said,” Green said.

He said the House would pass the new bill first, after consultations with the Senate to make sure it can pass in that chamber as well.

“Basically we’re going to try to make sure that we do what the Senate said they can do,” Green said.

Top negotiators in both parties in the House and Senate on Wednesday released statements pledging to work to pass the bill after the election.

Green noted that while the new research funding is less than he hoped, he views it as a starting point, and noted that the regular appropriations process could also increase some funding for the NIH.

“To me, it’s like a down payment,” he said. “We’re not going to get everything we started with.”