2013 Rare Disease Legislative Conference Presentations


  1. Welcome – Overview of the Day

    Julia Jenkins, Rare Disease Legislative Advocates

  2. Message from Abbey Meyers on the Passage of the Orphan Drug Act
  3. The Power of One: Advocacy Matters

    Anthony J. Castaldo, US Hereditary Angioedema Association

  4. Back to Basics: HIV/AIDS Advocacy as a Model for Catalyzing Change

    Margaret Anderson, FasterCures

  5. The Creating Hope Act: An Advocacy Success Story

    Mobilizing advocates and working with Congressional Staff to introduce & pass legislation

  6. CureTheProcess: Improving the FDA Regulatory Process

    Building a grassroots coalition to move a policy discussion to successful legislation

    Emil Kakkis, MD, PhD, EveryLife Foundation for Rare Diseases

  7. Rare Disease Congressional Caucus

    • Helen Dwight, Office of Rep. Lance (R-NJ)
    • Nicole Cohen, Office of Rep. Crowley (D-NY)
  8. Overview of the Political & Legislative Process

    Make up of 113th Congress

    Jen Bernstein, JC White Consulting

  9. Health Funding: The Budget and Appropriations Process

    Updates on FDA, NIH funding and the threat of Sequestration

  10. Health Reform Overview

    State Based Exchanges and Essential Health Benefits

    Joel White, Council for Affordable Health Coverage

  11. Engaging Government Agencies

    Working to expand newborn screening

    Jim Bialick, Newborn Coalition

  12. Rare Disease Awareness: Genes Ribbons on the Hill

    Nicole Boice, Global Genes Project

Thank You to our Event Sponsors:

Conference Sponsors [/raw]

Webinar: The 113th Congress and Medical Research Funding: A Perfect Storm Approaching?

This is no garden-variety budget year in Washington – a tsunami of budget and fiscal issues threaten federal science programs. While Congress has pushed the date of the “sequester” off until the beginning of March, delaying the prospect of automatic 8.2 percent cuts in the budgets of NIH, FDA, and other agencies, it is not entirely off the table. Current funding for these programs expires in March, the fiscal year 2014 budgets are being drafted, and there will likely be a fight over the debt ceiling.  This Webinar will tell you what you need to know about what could happen on Capitol Hill, when, and who the important players are, including some new faces.

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RDLA’s 3rd Annual Rare Disease Cocktail Reception & Movie Screening

Join RDLA for our 3rd Annual Rare Disease Cocktail Reception & Movie Screening to kick-off Rare Disease Day (week) in Washington DC

Monday, February 25, 2013
5:00pm Cocktail Reception * 6:30 Movie Screening * 8:00 Panel Discussion
Carnegie Institution of Science, 1530 P Street Northwest, Washington DC

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Feb. 6 Briefing: The Blueprint of Medical Research: How New Medicines Get from the Lab to the Patient

Improving and accelerating the R&D path requires all stakeholders in the medical research ecosystem to work together. Co-hosted by FasterCures and Friends of Cancer Research, this briefing is designed to educate members of Congress, legislative staff and advocates about the vital role federal agencies and the medical research sector play in advancing medical progress, and to emphasize the importance of prioritizing this work.

Wednesday, February 6, 2013, 12:00pm – 1:30pm
Dirksen Senate Office Building, Room 106, Washington, DC

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FDA Public Hearing: Considering Alternative Approval Pathway Feb. 4th

From the FDA Law Blog Posted: 15 Jan 2013 07:07 PM PST  By Alexander J. Varond

FDA announced earlier this week that it will hold a public hearing to obtain input on a potential new pathway to expedite the development of drugs and biological products for serious or life-threatening conditions that would address an unmet medical need. The new pathway would be used to approve drugs studied in well-defined subpopulations of patients with more serious manifestations of a condition. By focusing on patients with more serious manifestations of a condition, the pathway opens the door to the use of smaller and faster clinical trials.

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Newborn Screening Webinar

Global Genes | RARE Project will be hosting a series of webinars in 2013 geared towards educating and empowering our RARE community.  Webinar #1:  Newborn Screening Register today!

Date:  January 24, 2013 Time:  11:00 am Pacific Time

Many more diseases can be detected at birth than are currently part of the newborn screening panels in each state. Join us for a vibrant discussion and to learn more about why the states have different screening panels, how a particular screen can become part of your state’s NBS panel, how the federal SACHDNC panel influences state screening, and the importance of renewing the Newborn Screening Saves Lives Act of 2007.

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Rare Disease Day at NIH (RDD@NIH)

On February 28, and March 1, 2013, the National Institutes of Health (NIH) will celebrate the sixth annual Rare Disease Day with a 2-day-long celebration and recognition of the various rare diseases research activities supported by the Office of Rare Diseases Research – National Center for Advancing Translational Sciences (NCATS), the NIH Clinical Center, other NIH Institutes and Centers; the Food and Drug Administration’s Office of Orphan Product Development; other Federal Government agencies; the National Organization for Rare Disorders; and the Genetic Alliance.

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December 6th Grifols Congressional Briefing: The Importance of Plasma Therapies In Treating Rare Diseases

Grofols cordially invites Members and Staff to attend a Congressional Luncheon Briefing on:  The Importance of Plasma Therapies In Treating Rare Diseases
Thursday, December 6th, 2012
2360 Rayburn House Office Building

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BIO Patient and Health Advocacy Summit & Reception

The Power of Partnerships

December 12-13, 2012, Sheraton Four Points Hotel, Washington, DC

The BIO Patient and Health Advocacy Summit will bring together leaders from national health advocacy organizations and the biotechnology industry for two days of robust programming and networking opportunities. This Summit will provide thought-provoking panel discussions on timely healthcare policy issues, as well as curriculum geared toward non-profit advocacy organizations, including capacity building, advocacy training, and maximization of social media tools.

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RareVoice Awards Gala, Washington DC

On November 14, 2012, RDLA hosted its inaugural RareVoice Awards Gala to honor and thank Members of Congress, Congressional Staff and Patient Advocates for their advocacy to ensure The Food and Drug Administration Safety and Innovation Act (FDASIA), S. 3187 included provisions that spur the development of lifesaving treatments for rare disease patients.  Abbey Meyers, Founder of the National Organization for Rare Disorders, Inc. (NORD) was honored with a lifetime achievement RareVoice Award for her work to pass the Orphan Drug Act. In her honor, the award has been named an “Abbey.”

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