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

Rare Disease Congressional Caucus Briefing May 7th

Rare Disease Legislative Advocates & National Organization for Rare Disorders in coordination with
Rare Disease Congressional Caucus Co-Chairs Representatives Leonard Lance (R-NJ) and Joe Crowley (D-NY), held a briefing

“Access to Care & Therapies in the New Healthcare System: A Rare Disease Perspective”

Wednesday May 7, 2014

Read more

New Op-Ed With Details on 21st Century Cures Initiative

by Rep. Fred Upton,U.S. Congressman representing Michigan’s 6th District &  Rep. Diana DeGette, Chief Deputy Whip

Originally published by The Huffington Post on May 6th:  Talk to any American adolescent and it quickly becomes clear that keeping pace with 21st century technology is a constant challenge. But staying on top of today’s technology goes far beyond trending hashtags and Instagram likes; it presents a unique opportunity to bring researchers, innovators, caregivers, and patients together in a new way that advances our collective understanding of disease and how we combat it.

Read more

Rare Disease Advocate featured in CNN article about Compassionate Use

The program that allows patients to use experimental drugs not yet approved by the FDA is called “compassionate use”. Sandy Barker wonders, as do many others, if there might be a way to make it a little more compassionate.    

The CNN article gives a detailed overview of the compassionate use approval use process, including how it can be improved. It also illustrates how drug companies are increasingly concerned about how patients and parents of patients are using social media as a tactic in gaining approval for compassionate use.