Why FDA Supports a Flexible Approach to Drug Development

FDA Voice, Margaret A. Hamburg M.D., February 6, 2014

We all know that just as every person is different, so too is every disease and every drug. And so we weren’t surprised by the results of a new study published in the Journal of the American Medical Association. The study found that FDA used a range of clinical trial evidence when approving 188 novel therapeutic drugs for 208 indications (uses) between 2005 and 2012. These results are entirely consistent with our regulatory mandate. We believe varying approaches to clinical studies to support drug approval is good news, not bad. Read more…

Feb. 27th Rare Disease Congressional Caucus Briefing

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Rare Disease Legislative Advocates, National Organization for Rare Disorders & Jonah’s Just Begun Foundation to Cure Sanfilippo, in coordination with
Rare Disease Congressional Caucus Co-Chairs
Representatives Leonard Lance (R-NJ) and Joe Crowley (D-NY),
hosted a briefing featuring

Special Guest Jonny Lee Miller, who stars as Sherlock Holmes in the hit TV show Elementary,
will help us unlock the mystery of the
“Science Behind Rare Disease Policy”

Thursday February 27, 2014
12:00 noon – 1:30 pm
Capitol Visitors Center, Room: HVC – 201

Lunch Provided by event sponsors Genzyme & Shire

Moderator:  Kay Holcombe, Senior Policy Analyst, Genzyme

Panelists:

  • P.J. Brooks, Ph.D., Health Science Administrator – Office of Rare Diseases Research & Division of Metabolism and Health Effects, NIAAA,  NIH
  • Marshall Summar, MD, NORD Board Member, Division Chief, Genetics and Metabolism at Children’s National Medical Center and Faculty, Children’s Research Institute, Center for Genetic Medicine Research
  • Emil Kakkis, MD, PhD, President EveryLife Foundation for Rare Diseases
  • Hugh Hempel, Founder, Addi & Cassi Fund
  • Jill Wood, CEO, Jonah’s Just Begun to Cure Sanfilippo
  • Jonny Lee Miller, Actor, Patient Advocate


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Briefing Sponsors:

logos for breifing

May RDLA Luncheon & Conference Call

Advocates Luncheon & Conference Call
Tuesday, May 14, 2013
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4.16 RDLA Meeting & Conference Call

Please join us for an the next RDLA  Meeting & Conference Call
Tuesday, April 16, 2013
2:00 – 3:30 pm Eastern

750 9th Street, NW, Washington, DC
6th floor Office of Akerman Senterfitt – Washington Conference Room

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The Origin of RDLA by Emil Kakkis, MD PhD

RDLA, the Rare Disease Legislative Advocates: the story behind an association developed to help all rare disease groups succeed

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