Dedicated to facilitating the development of therapies for the diagnosis,
treatment and prevention of rare and devastating diseases.
RESOURCES
21st Century Cures Act as it Impacts the Rare Disease Community
(Kelly Austin, PhD, Sandra K. Heibel, PhD, and Marlene E. Haffner, MD, MPH)
National Institute of Health (NIH),
Office of Rare Diseases
National Organization for Rare Diseases (NORD)
US Food and Drug Administration / Orphan Drug Act
2009 interview with
Marlene E. Haffner, MD, MPH
2006 Nature Publishing
Group article
International Conference
on Rare Diseases
"FDA works to speed up
'orphan drug' process"
by Lorna Benson,
Minnesota Public Radio (see below)
Presentation Dates & Powerpoint Slides
2016 dates will be updated shortly - If you have further interest in upcoming
dates, please email us mhaffner@mhaffner.com
September 23, 2015 - Orphan Products and Drug Development 2015
As Presented To GLG
September 17, 2015 - Regulatory Market Update: Changes and Differences
Orphan Drug Summit 2015, Copenhagen, Denmark
August 5, 2015 - How to translate initiatives into realities
III Latin American Congress Of Pharmacy
And Industrial Biochemistry
April 2014 - Regulatory Considerations for the development
of Substantial Evidence of Effectiveness
World Orphan Drug Conference, Washington, DC
November 2013 - International Conference on Rare Diseases,
St Petersburg, Russia
October 2013 - Advance the Evaluation and Development
Orphan Drug Summit, Copenhagen, Denmark
May 2013 - A panel discussion on the Commercialization of Orphan Drugs
Drugs Research & Commercialization Conference, Boston, MA
February 2013 - The First Indian Summit on Neglected and Rare Diseases
Lucknow, India
September 2012 - Orphan Drug Global Incentives and Regulatory Framework
Orphan Drug Summit, Copenhagen, Denmark.
June 2012 - Challenges in the Designation of Drugs for Rare Disorders
DIA (Drug Information Association) meeting, Philadelphia, PA.
April 2012 - Working with Companies: Developing your Orphan Drug
International Business Forum, San Francisco, CA.
February, 2012 - Past Present and Future of orphan drugs
Madrid, Spain.
November 2011 - Women in Bio Seminar.
Issues in Orphan Drug Development
October 2011 - History of the Orphan Drug Act and Contemporary Issues.
1st annual DIA Orphan Drugs Rare Diseases Summit,
Washington, DC
September 2011 - How to Navigate the Biotech Regulatory Process
University of Arizona, Center for Inovation
September 2011 - Innovative Therapies for Rare Diseases, Opportunities and Challenges
Canadian Organization for Rare Disorders (CORD), Toronto, Canada
June 2011 - Overcoming Orphan Drug Designation Challenges.
DIA (Drug Information Association) Annual meeting, Chicago, IL.
April 2011 - What is Subsetting relative to Orphan Drug Products,
World Orphan Drug Congress, Washington, DC.
April 2011 - The Drug Development Process.
The Keck Graduate Institute, Claremont, CA.
March 2011 - Past, Present & Future of Orphan Drugs
for Children with Genetic Diseases.
Genetic Diseases of Children Conference, New York, New York.
February 2011 - Let’s Talk About Orphan Drugs.
Critical Path Institute, Tucson, Arizona.
January 2011 - I am a Drug, Where is my Disease?!
Dalhousie University, Halifax, Canada.