EveryLife Foundation for Rare Diseases Announces Formation of Bicameral Rare Disease Congressional Caucus

12-16-15 – Washington, D.C. – In a show of bipartisan support for the rare disease community, members of the House and Senate have come together to form the first bicameral Rare Disease Congressional Caucus.

The Caucus will be critical in raising awareness of the challenges faced by rare disease patients and serve as a platform for giving those individuals a voice in both the House and Senate. In addition, the Caucus will convene briefings throughout the year to educate Congressional staff on topics of importance to the rare disease community.

The Caucus will be co-chaired by Representatives Lance (R-NJ) and Crowley (D-NY)  and Senators Hatch (R-UT) and Klobuchar (D-MN).

“Throughout my time in the Senate, I have worked hard to help the 30 million Americans with rare diseases,” Sen. Hatch said. “While we have previously taken important steps, like passing the Orphan Drug Act, the fact that 95 percent of rare diseases currently have no treatment shows that much more needs to be done. Senator Klobuchar has been a terrific partner on legislation to get vital treatments to patients in need, and I am honored to join her as Senate Co-Chairs of the Rare Disease Congressional Caucus.”

“While there are about 7,000 known rare diseases, there are fewer than 500 drugs approved to treat any of those conditions,” Sen. Klobuchar said. “It is critical that we work together to increase the number of safe, effective, and affordable treatments that are available for people with rare diseases. I have long worked with Senator Hatch on this issue and I look forward to continuing this work as a Senate Co-Chair of the Rare Disease Congressional Caucus.”

The Rare Disease Congressional Caucus, originally formed in 2009 by Rep. Upton (R-MI), is currently comprised of 88 Members of the House and will be expanded to include the Senate.

“We are grateful to our House champions and to Senators Hatch and Klobuchar for stepping-up to lead this Caucus on behalf of the rare disease community. We are looking forward to raising awareness about the importance of public policy to spur the development of new therapies for rare disease patients,” said Julia Jenkins, Executive Director of the EveryLife Foundation for Rare Diseases.

Rare Disease Legislative Advocates (RDLA), a program of the EveryLife Foundation for Rare Diseases, will continue to coordinate regular Hill briefings and act as a legislative clearinghouse for the patient community, with the goal of mobilizing patients to be effective legislative advocates (www.rareadvocates.org).

RareVoice Awards Gala Honors Rare Disease Advocates and Congressional Leaders

More than 300 patient advocates and industry leaders as well as staff from Capitol Hill and federal agencies joined us last week for the fourth annual RareVoice Awards Gala at Arena Stage in Washington, DC.

Dr. Steve Groft received a Lifetime Achievement award for his dedication to stimulating research and advancing development of therapies during his tenure at the Food and Drug Administration (FDA) and National Institutes of Health (NIH), where he served as Director of the Office of Rare Disease Research from 1993 until his retirement in 2014.

The judges had difficulty selecting the winners among the passionate patient advocates making a difference in states across the country and on Capitol Hill.  Patricia Weltin, President and Founder of the Rare Disease United Foundation, received an Abbey for patient advocacy at the state level. Lisa and Max Schill with RASopathies Network received an Abbey for patient advocacy at the federal level for their efforts in support of the 21st Century Cures Act.  Ronald Bartek, President and Founder of the Friedreich’s Ataxia Research Alliance, received a Lifetime Achievement Award for his tireless advocacy before Congress as well as the FDA and Social Security Administration.

The Chairman and members of the House Energy and Commerce Committee were recognized for their leadership on the 21st Century Cures Act which has several provisions critical to the rare disease community, including incentives to spur development of new therapies. Representative Fred Upton (R-MI) received a Lifetime Achievement Award. Representative Gus Bilirakis (R-FL), G.K. Butterfield (D-NC) and Diana DeGette (D-CO) were honored with Congressional Leadership Awards. Saul Hernandez, Deputy Chief of Staff and Legislative Director for Representative G.K. Butterfield, and Clay Alspach, Chief Health Counsel of the Energy and Commerce Committee, received Abbeys for their work on behalf of the rare disease community.

Dr. Kakkis, President and Founder of the EveryLife Foundation for Rare Diseases, ended the awards ceremony with a call to action, urging everyone in attendance to contact their Senators to ask that the many provisions of the 21st Century Cures Act supported by the rare disease community be included in Innovation for Healthier Americans, the Senate companion bill.

Photos of the awards ceremony and reception will be posted on the Rare Disease Legislative Advocates Facebook page and the RareVoice Awards website, so be sure to visit both.

Thank you to Shire, the Presidential Sponsor, and other sponsors including AbbVie, Alexion, Amgen, Amicus, Genzyme, Novartis, PhRMA, Raptor, and Vertex. Proceeds from the Gala fund Rare Disease Week on Capitol Hill, to be held February 29 through March 3 next year.

Press Release: RareVoice Awards Gala Honors Rare Disease Advocates and Leaders on Capitol Hill

Contact: Stephanie Fischer


RareVoice Awards Gala to Honor Rare Disease Advocates and Leaders on Capitol Hill

Washington, DC (November 4, 2015) – Rare Disease Legislative Advocates will honor rare disease advocates and leaders on Capitol Hill at the RareVoice Awards Gala tonight.

Members of Congress will be recognized for their leadership on the 21st Century Cures Act which has several provisions critical to the rare disease community, including incentives to spur development of new therapies. Representative Fred Upton (R-MI) will receive a Lifetime Achievement Award. Representative Gus Bilirakis (R-FL), G.K. Butterfield (D-NC) and Diana DeGette (D-CO) will be honored with Congressional Leadership Awards.

Advocates are hopeful that key provisions of the 21st Century Cures Act will be included in the Senate companion legislation, Innovation for Healthier Americans, and that the Senate will act quickly to advance it. A delay in legislation is a delay in new treatments.

Congressional staff to be honored for efforts on behalf of the rare disease community include Clay Alspach (House Energy and Commerce Committee), Saul Hernandez (Rep. G.K. Butterfield), Sara Mabry (Sen. Bob Casey), Harrison Matheny (Rep. Ryan Zinke), Molly McDonnell (Rep. Leonard Lance), and Carly McWilliams (House Energy and Commerce Committee).

Rare disease advocates to be recognized for federal advocacy include Annette Bakker (Children’s Tumor Foundation), Ronald Bartek (Friedreich’s Ataxia Research Alliance), Noah Coughlan (Run for Rare), Woody Crouch (Sturge-Weber Foundation), Lisa and Max Schill (RASopathies Network), Steve Smith (National MPS Foundation), Steve Walker (Abigail Alliance), and Rob Whan (Caleb’s Crusade).

Rare disease advocates recognized for state advocacy include Sharon King (Taylor’s Tale), Kirsten Norgaard (Adrenal Insufficiency United), Elisa Seeger (Aidan Jack Seeger Foundation), Bruce Thompson (ALS parent advocate), and Patricia Weltin (Rare Disease United Foundation).

A Lifetime Achievement Award will be presented to Dr. Stephen Groft for his dedication to stimulating research and advancing development of therapies during his tenure at the Food and Drug Administration (FDA) and National Institutes of Health (NIH).

Thank you to Shire, the Presidential Sponsor, and other sponsors including AbbVie, Alexion, Amgen, Amicus, Genzyme, Novartis, PhRMA, Raptor, and Vertex.

Additional information is available at http://rarevoiceawards.org/.

 Rare Disease Legislative Advocates is a collaborative organization designed to support the advocacy of all rare disease groups. By growing the patient advocacy community and working collectively, we can amplify our many voices to ensure rare disease patients are heard in state and federal government. For more information, visit http://rareadvocates.org/.

Click here for a PDF of this press release

Precision Medicine: New Frontiers for Rare Diseases


Rare Disease Legislative Advocates in coordination with Rare Disease Congressional Caucus Co-Chairs:

Representatives Leonard Lance (R-NJ) and Joe Crowley (D-NY), will host a briefing on:
Precision Medicine: New Frontiers for Rare Diseases

Thursday, November 5th, 2015
12:00 pm – 1:00 pm (lunch provided)
Rayburn House Office Building
Room: B318 

speakers for precision Medicine briefing

Moderator: John Crowley, Chairman & CEO, Amicus Therapeutics

  • Matt Might, President, NGLY1.org & Associate Professor, University of Utah
  • Erynn Gordon, Director of Clinical Development, 23andMe
  • John Stone, Counsel, Committee on Energy and Commerce – U.S. House of Representatives
  • Dr. Christopher Austin, Director, National Center for Advancing Translational Sciences, NIH
  • Sean Sigmon, Business Development Director, Oracle Health Sciences

Limited Seating, RSVP to attend

Can’t attend? Click here to contact your congressional office to ask them to send a representative!

Hosted by:

Precision Medicine Logo Mashup

2015 Rare Disease Legislative Conference: Videos


2015 Legislative Conference & Lobby Day

Presented by   

Abbive Logo

FHI 360, 1825 Connecticut Avenue Northwest, Washington DC

Tuesday, February 24, 2015


(Click on the linked titles to view the presentations)

9:15 – 9:30 am           Welcome – Overview of the Week 

Conference Moderator:  Dean Suhr, Genetic Alliance


9:30 – 10:10 am          Overview of the 114th Congress

Congress After the Election and a Health Policy Forecast, Joanne Kenen, Health Care Editor, Politico

Congressional Committees That Shape the Future of Health, Nick Mannetto, Principal, FaegreBD Consulting


10:10 – 10:40 am        Know Your Lobbying RightsSiobhan Rausch, Partner, Hogan Lovells


10:40 – 11:40 pm         Avenues for Impact

Appropriations – Katie Schubert, Vice President, Coalition for Health Funding & CRD Associates

Legislation – Cheryl Jaeger, Principal, Williams & Jensen

Regulatory Joel White, Founder & President, Horizon Government Affairs


12:10 – 1:30 pm         21st Century Cures Initiative

The Path to 21 Century Cures – Carly McWilliams, Professional Staff – Subcommittee on Health, House Energy & Commerce Committee

OPEN ACT HR 971 (Orphan Product Extensions Now Accelerating Cures & Treatments) – Saul Hernandez, Legislative Director, Office of Rep. Butterfield (D-NC)

CURE (Compassionate Use & Reform & Enhancement) Act HR 909 (Expanded Access) –Andy Taylor, Legislative Director, Office of  Rep. McCaul (R-TX)

Dormant Therapies/MODDERN – Eric Gascho, Assistant Vice President for Government Affairs, National Health Council

Advancing Research for Neurological Diseases Act HR 292 – Jennifer Sheridan Palute, Policy Director, Parkinson’s Action Network


1:30 – 2:10 pm           Prepping for your Hill Meetings  

 Lobbying Tips & Overview of Schedules Stephanie Vance, Owner & Founder, Advocacy Associates

Giving your two minute personal story – Kristine PJ Caalim, Founder, Zequing For a Cure

A Symbol for the Rare Disease Community: Gene Ribbons – Carrie Ostrea, Director of Advocacy, Global Genes

Leave behinds & the Caucus Ask – Andy Russell, Associate Director, EveryLife Foundation for Rare Diseases

2:10 – 2:40 pm           Break & Distribution of Hill Day Schedules

2:40 – 3:30 pm           Break-Out Skill Training Workshops   

Starting your own Non-profit to Grow your Advocacy Impact –Barbara Lavery, Vice President of Strategic Alliances, Global Genes Project & Mike Illions, Vice President, Pediatric Hydrocephalus Organization

  • Tips and tools for getting your rare disease organization to the next level. Learn about obtaining 501c3 status, creating a mission, vision, and a board of directors. Essential skills for any aspiring organization.

Polish & Practice your Presentation Pitch – Joe LaMontain, Vice President, Reingold Inc.

  • Learn about persuasive and effective communication for maximum advocacy impact. Get tips for making a lasting impression and laying the groundwork to build a first-time meeting into a lasting relationship.

Introducing Legislation – Julia Jenkins, Executive Director, EveryLife Foundation

  • Hear about the do’s and don’ts for introducing legislation, coalition building, and how to move a bill through the legislative process.

Social Media Advocacy – Joel Cohen, Non-Profits, Politics & Advocacy, Facebook

  • Learn some insider tips for leveraging social media to raise awareness and build an action network for your cause.

  3:40 – 4:30 pm           State/Regional Break-Out Sessions Practice Meetings

      4:40 pm                      Closing Remarks

      5:00 pm                     End Conference

Thank You to ALL the advocates that came to DC to participate in Rare Disease Week on Capitol Hill!

RDWoch Logo for banner

Thank you for attending this year’s annual Rare Disease Legislative Conference in Washington D.C. We were proud to bring together over 300 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.

Your presence helped to make this event a great success and your enthusiasm and positive spirit helped make our time together both productive and fun.  We want you to continue to be engaged with RDLA and your legislative representatives, so please follow up with the offices you visited (if you haven’t already) and send us stories and pictures of your meetings either by email or use the AdvocacyApp (https://advocacyassociates.constituentvoice.net//).  Help spread the word with advocates that weren’t able to make our conference by writing blog posts and testimonials, and sharing your photos on social media. Also, we are pleased to let you know that presentations and photos from this year’s conference will be 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 voice of the rare disease community (RareVoice Awards).

If you attended the conference 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!



October RDLA Meeting

Please join us for an
Advocates Luncheon/Conference Call

Thursday, October 16th, 2014
12 noon to 1 p.m. EST

RDLA DC Office
1101 14th Street NW, Suite 700
Washington, DC,  20005

Lunch will be provided

Click the titles to view PowerPoint Sides:

Please click here to listen to the audio recording of the call

Mark your Calendars!

Nominate someone today for a RareVoice Award!


Rare Disease Legislative Advocates is pleased to open nominations for the 3rd Annual rarevoicelogoweb

A celebration to honor advocates who give rare disease patients a voice on Capitol Hill

Thursday, November 13, 2014 Arena Stage in Washington, DC

www.RareVoiceAwards.org RareVoice Award nominations are open to the public.  We encourage the community to nominate individuals and organizations who advocate for legislation and policy that benefits the rare disease community.  If you know of someone who has been a “Voice” for the rare disease community and should be honored for their work, please click here to submit your nomination.  We have nominations open for three award categories, Member of Congress or Congressional Staff, Patient Advocate or Patient Organization, and Government Agency Staff.  Deadline to submit nominations is  Friday August 29, 2014. Sponsorship Opportunities are available, contact Julia@RareAdvocates.org.   Thank you to our sponsors:

Gala Sponsor Mashup

Rare Disease Congressional Caucus Briefing


Rare Disease Legislative Advocates in coordination with Rare Disease Congressional Caucus Co-Chairs

Representatives Leonard Lance (R-NJ) and Joe Crowley (D-NY), will host a briefing on

Implementation of the Rare Disease Provisions in the
Food & Drug Administration Safety and Innovation Act of 2012 (FDASIA) PL 112-144

Wednesday, September 17, 2014

12:00 noon – 1:30 pm

Rayburn House Office Building, Room: B-318

Lunch Provided by event sponsors Amicus & Shire


  • Sec. 901. Enhancement of accelerated patient access to new medical treatments: Accelerated Approval for rare diseases, Emil Kakkis, MD PhD, EveryLife Foundation for Rare Diseases
  • Sec. 902. Breakthrough therapies, TBD, Novartis Therapeutics
  • Sec. 903. Consultation with external experts on rare diseases, Stephanie Krenrich, Cystic Fibrosis Foundation
  • Sec. 908. Rare pediatric disease priority review voucher incentive program, Nancy Goodman, Kids v Cancer
  • Sec. 1137. Patient Participation in Medical Product Discussions, Allison Greenstein, Narcolepsy Patient Advocate
  • FDA’s Strategic Plan for Accelerating the Development of Therapies for Pediatric Rare Diseases – TBD, FDA
  • Emerging Biotech Perspective of the combined impact of FDASIA, TBD, Amicus Therapeutics

Limited Seating, RSVP to attend: To register click here.

Briefing Hosted by:

Sept. Caucus briefing logo mashup

RDLA’s June Webinar/Conference Call


Tuesday, June 17th, 2014
11:30 a.m. to 12:30 p.m. EST