National Health Council Urges Patient Organizations to Show Support for 21st Century Cures

The National Health Council is circulating a sign-on letter to Congressional leadership to urge passage of the 21st Century Cures Act by the end of 2016. It is imperative that the tireless work by Congress and the patient community does not go to waste by delaying action until the next Congress in 2017.

For to view the letter and sign, please click here. The goal is to present the letter to House and Senate leaders before Congress comes back on Monday, November 14th, so signatures are requested no later than Thursday, November 10th.

Only the name of organizations will be listed on the letter, not the individual signers.  Please note that you must be authorized on behalf of your organization to commit to signing publicly.

If you have any questions about the letter, please contact Eric Gascho, Vice President of Government Affairs at the National Health Council, at egascho@nhcouncil.org or 202-973-0545.

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.”

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.

Pediatric Priority Review Vouchers Saved in the Eleventh Hour

From FDA Law Blog:

One day before the program’s sunset, President Obama signed a bill to temporarily reauthorize the rare pediatric disease priority review voucher program for 3 months.  The program has had significant interest from industry, and the program’s extension on September 30 was the product of substantial efforts on the part of stakeholders, including rare disease advocates. 

This is the second short-term reauthorization of the program, which was originally slated to end in March 2016. Unlike the first reauthorization (which we discussed here), the Advancing Hope Act (S. 1878) was a more ambitious undertaking.  As such, it amended the definition of “pediatric rare disease,” likely expanding eligibility for the program.  We discussed the law’s impact on the program’s scope in a post last week.

The law makes several other key changes. First, the Advancing Hope Act now requires Sponsors to notify FDA of the Sponsor’s intent to request a pediatric voucher upon the submission of a rare pediatric disease product application.  This requirement comes into effect 90 days after the enactment of the Advancing Hope Act, which incidentally is just a few days prior to the program’s December 31, 2016 sunset.  Second, Sponsors are now expressly prohibited from receiving more than one priority review voucher per drug.

Reading the law makes it abundantly clear that the September 30, 2016 reauthorization is a stop-gap measure. Congress’s effort to quickly pass the bill and avoid creating a gap in the program, meant making a last-minute amendment to the program’s end date of at least September 2022 to December 31, 2016.  As a result of the last-minute amendment, some parts of the bill are fairly nonsensical.  For example, by 2022, GAO will be required to issue another report on the program, similar to the one it issued in March 2016.  It would be odd if the pediatric voucher program was not reauthorized past 2016 and yet the GAO was required to report on the program in 2022 (more than 5 years after the end of the program).

Members of Congress have expressed a strong desire to address FDA-related legislation (including the 21st Century Cures Act, which we discussed here) during the lame duck session of Congress after the November elections.  Part of this attention will go towards discussing the pediatric voucher program and its long term or permanent reauthorization.

Editorial: “Why I’m Stepping Out of My Comfort Zone and Into My Congressman’s Office for Rare Disease

TheMighty published an editorial by Sharon Rose on the need for the rare disease community to advocate in support of the 21st Century Cures Act:

Rare disease has changed my life in many ways, but the most unexpected recent change has been my interest in laws and political issues regarding health care and treatment options. You see, I am a right brainer. I enjoy and flourish in the arts. I’ve never jumped for joy while taking a math or science class. I can recall looking up medical terminology after diagnoses and subsequent MRI or test reports years ago, and how strange it was for me to learn about medicine. I was in a new arena and out of my comfort zone.

However, I realized that in order to advocate for my care, it was a necessary step, and I was finding it interesting! I was being forced to expand in ways I had never imagined. Being creative had always been my focus whether in study, or for fun. But more recently, as a patient and advocate, I’ve experienced another surprising shift in interests. Why, you ask?  Because for the past 6+ years, I’ve been relentlessly and urgently seeking treatments as a patient for Klippel-Feil syndrome, cervical dystonia, Ehlers-Danlos syndrome, and vasculitis – all rare diseases. Time and time again, I have found little understanding or relief. I’ve traveled to five states seeking care. Yes, there are a few great doctors out there. Great doctors who have limited time, limited accurate information, and limited treatment and medication options to offer me as a patient dealing with such an odd laundry list of rare conditions.

To read more from this article, click here.

Little News is Good News

An editorial published by the Alliance for a Stronger FDA staff:

What do the opioid legislation, the Zika legislation and Cures/Innovations bills have in common, other than that they are “hot button” issues? In each case, Congress has done its best to seriously consider healthcare problems facing our nation and managed to find compromises in order to reach consensus. Also — significantly — the three pieces of legislation are stalled by funding issues.

Some Members of Congress (mostly Democrats) think that action on these three topics is so pressing that the legislation should contain enough mandatory funding so that they can be implemented as soon as possible. Other Members of Congress (mostly Republicans) agree on the importance of the three issues, but feel strongly that funding should be left to the appropriations committees. For the moment, the three bills are deadlocked, but it is money, not substance, that is holding them back.

One or more of the three bills may move next week or in September or (possibly) during a lame duck session. But then again, there is no guarantee that they aren’t deadlocked for the year because of the funding issues.

Meantime, the appropriations process seems to be facing a similar fate. There is some compromise and consensus baked into the current set of bills, but also lots of “poison pill” amendments and games-playing. Analysts are still predicting either a continuing resolution or an omnibus bill.

Heading into a presidential election and a difficult transition (regardless of who wins), it is already clear that the most important issue next year, as this year, will be money to fund programs. Under the existing budget agreements, there is not enough domestic discretionary spending to meet high priority needs, no less to handle emergency situations (like Zika and opioids). Under the circumstances, it is hard to say where the money will come from to pay for the new administration’s initiatives. The primacy of funding concerns will occur regardless of which party controls the House and the Senate.

None of this is good news for FDA or any of the public health service agencies. As the foremost advocate for FDA funding, the Alliance will continue to tell Congress about the broad mission and ever-increasing responsibilities on FDA. It will take all of us to make a dent against so much downward funding pressure.

Note: This week’s Analysis and Commentary was written by Steven Grossman, the deputy executive director of the Alliance for a Stronger FDA.

Join Us to Ensure the Rare Disease Community is Heard This Summer

During the summer recess, Members of Congress return to their home states to hear from constituents and attend community events.  Meeting with them during this time is a critical opportunity for rare disease advocates to build relationships, educate them on key legislative needs and follow-up on requests made during Rare Disease Week on Capitol Hill or phone calls or emails about legislative issues since then.

As a FREE service to the rare disease community, Rare Disease Legislative Advocates (RDLA) will set-up a meeting for you and fellow advocates at your convenience. Any rare disease advocate is encouraged to join us for In-District Lobby Days, held from July 18th to September 5th.  Registration is free and available online through Friday, July 15thYou can specify when you are available during the summer recess and how far you are willing to travel for a meeting.

This is a critical juncture for our community.  Although the 21st Century Cures Act passed the House of Representatives with broad bipartisan support last July, the Senate has yet to pass the companion legislation.  In the current partisan environment in Washington, it is important for Members of Congress to hear first-hand how united the American public is on the subject of medical innovation.  A recent poll found that 78% of Americans think Congress should prioritize fostering medical innovations, with an emphasis on finding cures for diseases that still lack treatments.  By joining us for In-District Lobby Days, you can urge your Senators and Representatives to support 21st Century Cures and advocate for other legislation benefiting our community.

To register for In-District Lobby Days, please click HERE.  RDLA will use the information you provided to schedule meetings for you, and help you prepare by providing key background materials and hosting a preparatory webinar to be held on July 13th.

It is up to each of us to ensure the voices of rare disease patients and caregivers are heard in Washington.  Please help us tell Congress loud and clear:  WE NEED CURES NOW!

Nominations for Fifth Annual RareVoice Awards Due by July 31st

Do you know anyone who has gone above and beyond to become a rare disease policy leader or advocate in a state or in our nation? Rare Disease Legislative Advocates (RDLA) is seeking nominations for the RareVoice Awards to be given on November 16th in Washington, DC.

Nomination categories include:

Congressional Staff

Has a staff member of a member of the U.S. House, Senate or Congressional committee been instrumental in helping to create and/or advance legislation that benefits the rare disease community?

Government Agency Staff

Do you know a government agency staff member who has gone out of his or her to listen to rare disease patients and then taken action to benefit the community?

Patient Advocate – Federal Level Advocacy

Do you know an advocate or organization that has worked with Congress or federal agencies to spearhead change on behalf of the rare disease community?

Patient Advocate – State Level Advocacy

Do you know an advocate or organization that has crusaded on the state level to support and advance rare disease legislation or regulatory reform?

RareVoice Award nominations are open to the public. Winners will be selected by an independent committee composed of representatives from the rare disease community including previous RareVoice winners.

To nominate a rare disease champion, please click HERE. Nominations close on July 31st, 2016.

The winners will be announced LIVE at the fifth annual RareVoice Awards ceremony on November 16that the Arena Stage in Washington, DC and will receive the coveted Abbey award.

The award is named after Abbey Meyers, the founder of the National Organization for Rare Disorders (NORD), and represents the “rare voice” speaking on behalf of patients, especially children, who might not otherwise be heard. The Abbey statue was commissioned for the RareVoice Awards from the renowned sculptor Nobe, who crafts each statue by hand in bronze.

Senate Holds Third and Final Markup of Biomedical Innovation Legislation

On April 6th, the Senate Health, Education, Labor and Pensions (HELP) Committee held the last of three markups to discuss biomedical innovation legislation.  The legislation considered and approved in those mark-ups will be conferenced with the 21st Century Cures Act which passed the House of Representatives with broad bipartisan support last summer. In addition to approving the five bills under consideration, members of the Committee offered several amendments of particular interest to the rare disease community. Read more

RECAP: White House Meeting On 21st Century Cures

From the House Energy and Commerce Committee:

WASHINGTON, DC – House Energy and Commerce Committee Chairman Fred Upton (R-MI), Ranking Member Frank Pallone, Jr. (D-NJ), and Rep. Diana DeGette (D-CO) issued the following statement following a meeting at the White House with Vice President Joe Biden on H.R. 6, the 21st Century Cures Act. Senate HELP Committee Chairman Lamar Alexander (R-TN) and Ranking Member Patty Murray (D-WA) also attended this afternoon’s meeting.

Upton, Pallone, and DeGette commented:

“Today’s meeting with Vice President Biden and Senate leaders was positive and productive. The #Path2Cures connects both the White House and U.S. Capitol, and we welcome the vice president’s enthusiastic participation in this bipartisan effort. As many patients and their families are too painfully aware, there’s not much time. But the good news is, we are more than half way there as we’ve already done the legwork in the House and the Senate is making headway on its innovation package.

“We are seeing a tremendous opportunity for Cures, with our legislative efforts coupled with the ‘moon shot’ and the administration’s Precision Medicine Initiative, there is no question that 21st Century Cures is the right vehicle to get this done. We will all continue working together – House, Senate, and White House – until we are successful in delivering #CuresNow for patients across America.”