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.

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.

Orphan Drug Expenditures In The United States: A Historical And Prospective Analysis, 2007–18

With debate around the costs of prescription drugs swirling, a new report was issued from IMS Health detailing future spending on orphan drugs. The authors found that despite growth in the number of available treatments, overall oprhan drug spending will continue to be sustainable in the future.  Below is the article abstract, click here to read the full article.

The Orphan Drug Act of 1983 established incentives for the development of drugs that treat rare, or orphan, diseases. We used the IMS Health MIDAS database of audited biopharmaceutical sales to measure US annual spending on orphan drugs in the period 2007–13, and we estimated spending on the drugs for the period 2014–18. We identified 356 brand-name orphan drugs that were approved by the Food and Drug Administration in the period 1983–2013. While we included orphan drugs with both orphan and other indications, we adjusted spending to include only spending for orphan indications. In 2014 dollars, expenditures on orphan drugs totaled $15 billion in 2007 and $30 billion in 2013—representing 4.8 percent and 8.9 percent of total pharmaceutical expenditures, respectively. Our future trend analysis for the period 2014–18 suggests a slowing in the growth of orphan drug expenditures. The overall impact of orphan drugs on payers’ drug budgets is relatively small, and spending on orphan drugs as a percentage of total pharmaceutical expenditures has remained fairly stable. Concerns that growth in orphan drug expenditures may lead to unsustainable drug expenditures do not appear to be justified.

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.

Rare Disease Congressional Caucus Adds Nine Members

Thanks to their diligent work educating Members of Congress, rare disease advocates were successful in expanding the ranks of the Rare Disease Congressional Caucus. In March, nine legislators agreed to join the Rare Disease Congressional Caucus:

Senator James Risch (Idaho)
Senator David Vitter (Louisiana)RDCC-Horizontal-CMYK
Representative Brad Ashford (NE-2)
Representative David Cicilline (RI-1)
Representative John Garamendi (CA-3)
Representative Joe Kennedy, III (MA-4)
Representative Donald Norcross (NJ-1)
Representative David Rouzer (NC-7)
Representative Juan Vargas (CA-51)

We applaud these legislators for taking leadership on the issues that are important to the rare disease community. You can find the complete Rare Disease Congressional Caucus membership at

Action Alert from the EveryLife Foundation: Ask Your Senators to Co-Sponsor the OPEN ACT to Repurpose Drugs for Rare Disease Patients

Take action to support the OPEN ACT – Orphan Product Extensions Now, Accelerating Cures & Treatments (HR 971/S 1421).  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. EveryLife Foundation, NORD, Global Genes, Genetic Alliance and an additional 155 patient organizations support this bipartisan legislation.


Click here to take action!

Save the Date for Rare Disease Week on Capitol Hill: February 29 – March 3rd

Please pull out your calendars and save the date for RDLA’s Rare Disease Week on Capitol Hill 2016! Read more

2014 RDLA’s Legislative Scorecard

Rare Disease Legislative Advocates compiled a congressional scorecard to educate advocates on how their Representative has voted on issues affecting the rare disease community.  Each Representative’s score is based on membership in the Rare Disease Congressional Caucus as well as action taken on legislation important to the rare disease community.

The most powerful tool a legislator has is their vote, but a sampling of votes and actions on bills does not always provide the full story.  The rare disease community followed many bills that are not on this list and legislators may have taken action in other ways not captured by this scorecard. Some bills progressed to laws, while others may have been halted during the legislative process or had provisions incorporated into other bills.  In addition, legislators may have worked in committee or in other ways to secure an amendment or enable a compromise.  Thus, a voting record provides crucial, fact-based insights into legislative activities, but is not intended to be a comprehensive indicator of rare disease impact.

How to Use the Scorecard:

  • Have a conversation about rare disease issues with your legislator and include the Scorecard.
  • If your legislator has a strong record on rare diseases, be sure to thank them publicly. They deserve the recognition.
  • If your legislator’s record is weak, ask them why. Members will often try to find ways to help a constituent.
  • Evaluate the conversation and follow-up. Is there a commitment for an improved level of support for rare disease issues? If so, keep in touch with their office and nurture the relationship.


Scorecard Key:

Members we given a √ for being an author, original co-sponsor, co-sponsor, co-chair, signer, and a yes vote.

8 √ = A+           7 √ = A            6 √ = A-            5 √ = B+             4 √ = B              3 √ = C+             2 √ = C             1 √ = D            0 √ = F

Rare Disease Congressional Caucus Membership: The bipartisan House caucus is made up of 79 representatives. Points were also added if the representative was a Co-Chair of another rare disease caucus, such as:

Mitochondrial Disease Caucus

Cystic Fibrosis Caucus

Hydrocephalus Caucus

Childhood Cancer Caucus

– Crohn’s Colitis Caucus

Accelerated Approval Sign-on Letter: Representatives received a point for signing-on to the bipartisan letter calling on FDA to help ensure that recently passed legislation (FDASIA) provides rare diseases access to accelerated approval.

H.R. 2019 – Gabriella Miller’s Kids 1st Research Act: This bill redirected funding from political conventions to the National Institutes of Health (NIH) Common Fund to support transformative research for childhood cancer, autism, fragile X syndrome and other rare pediatric diseases. This bill was voted on (12/11/13), became law, and was appropriated on 12/16/14. Members received four points for introducing the bill, three points for being an original co-sponsor and voting ‘yes’, two points for being a co-sponsor and voting ‘yes’, and one point for voting ‘yes’.

H.R. 225 – National Pediatric Research Act:  Enables the establishment of a National Pediatric Research Network managed by the NIH. This bill was voted on and members received four points for introducing a bill that passed, three points for being an original co-sponsor and voting ‘yes’, two points for being a co-sponsor and voting ‘yes’, and one point for voting ‘yes’.

H.R. 1281 – Newborn Screening Saves Lives Re-authorization Act: Promotes expanded screening of newborns and children for heritable disorders. This bill passed the house by voice vote, so no record of individual votes was made.  Members received two points for being one of the original co-sponsors on the bill and one point for co-sponsoring the bill at a later point in time. This Bill is currently awaiting presidential signature.

H.R. 3116 – Modernizing Our Drug & Diagnostics Evaluation and Regulatory Network (MODDERN) Cures Act of 2013: The language to improve access to diagnostics and spur diagnostic innovation was included in Medicare Sustainable Growth (SGR) reform, passed by Congress earlier this year.  The dormant patent provision is designed to encourage the development of drugs to treat conditions with few or no treatment options and has gained significant support, leading to a companion bill in the Senate and potential for the provision ti be included in the broader 21st Century Cures legislation.

H.R. 460 – Patients’ Access to Treatments Act of 2013: Prohibits health insurance plans from imposing cost-sharing requirements, including co-payment and co-insurance, applicable to prescription drugs in a specialty drug tier. This bill did not pass this Congress but gained significant support from patient groups, industry and legislators.

H.R.2725 – FDASOS:  Amends the Balanced Budget and Emergency Deficit Control Act to exempt the user fees paid by industry from sequestration (mandatory across-the-board spending cuts).  Support of this legislation enable Congress to ensure the FDA received the full revenues from User Fee in the current budget.  The legislation needs to pass to ensure the FDA received the full revenues from User Fee in the current budget. The legislation needs to pass to ensure the exemption becomes permanent.

Rare Disease Week 2015 On Capitol Hill

RareDiseaseWeek-Logo-cmykThanks for playing your part in such a monumental week! Over the next few months we will start to see the positive outcomes of all our hard work, as rare disease legislation makes its way through The Congress.

View Photos and Presentations from Rare Disease Week 2015.

View presentations and photos from this year’s Rare Disease Legislative Conference and all the other events below!:

All the photos taken at the Legislative Conference can be viewed here: at our Facebook page!

View videos and photos from the event below:

Monday, February 23rd

Rare Disease Documentary Screening and Cocktail Reception
@ Carnegie Institution for Science

Doc Screening 2015

View photos from the Documentary Screening to kick off Rare Disease Week!

Tuesday, February 24th


Legislative Conference
@ FHI 360

Click here to see the 2015 Legislative Conference Agenda


Wednesday, February 25th

Lobby Day Breakfast
@ Women’s Democratic Club

Rare Disease Lobby Day
@ U.S. Senate and House Office Buildings
9:00 am – 5:00 pm Meetings with your elected officials

Thursday, February 26th

Rare Disease Congressional Caucus Briefing
@ U.S. Capitol Visitor Center
12:00 pm – 1:00 pm


Rare Artist Reception
Rayburn Foyer
5:00 pm – 7:00 pm

Lance Rare Artist

Click here to see more photos of the RareArtist event

Friday, February 27th

Rare Disease Day NIH logo

Rare Disease Day at the NIH
@ NIH Campus

NIH event

Advocates: Please take a moment to fill out our quick survey regarding your conference experience. Your feedback is critical in our ongoing effort to improve this annual event. Thank you again for being part of such a wonderful conference experience!

We are proud to bring together members of the rare disease community from across the country to share their unique stories, ideas, and make our collective voice heard on Capitol Hill.

The large presence of dedicated advocates helped to make this event a great success. Their enthusiasm and positive spirit helped make Rare Disease Week 2015 both productive and fun.

Help spread the word with advocates that weren’t able to make our conference by writing blog posts and testimonials, and sharing photos on social media. Also, we are pleased to have presentations and photos from this year’s conference available on our website soon, so stay tuned and stay engaged with RDLA.

Throughout the year we have opportunities for advocates to learn about the most recent Rare Disease Legislation (monthly webinars), meet with their representatives (In-District Lobby Days), and celebrate those who have raised the profile and voice of the rare disease community (RareVoice Awards).

Thank you to our sponsors:

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