Home > FDA Basics > New FDA Basics Video Available about Orphan Drugs and the Fight Against Rare Diseases

New FDA Basics Video Available about Orphan Drugs and the Fight Against Rare Diseases

As part of FDA Basics, FDA has posted a new video with Dr. Tim Cote, director of FDA’s Office of Orphan Products Development.  Dr. Cote discusses how FDA’s Orphan Drugs program helps in the fight against rare diseases.  View the video below.

  1. Eric McMillan
    June 22, 2010 at 9:02 pm | #1

    I think it is a collective effort in fighting this.
    A campaign spending time with to save the kids future.

  2. Al Musella
    June 23, 2010 at 7:27 pm | #2

    I run a brain tumor foundation.
    We saw amazing results presented at the ASCO conference recently for the treatment of Glioblastoma multioforme (a rare disease – about 20,000 a year).
    What makes these announcements so interesting is the lack of side effects/ toxicity, and some pretty amazing survivals.
    For example, the ICT-107 vaccine trial (19 GBM patients) reported no grade 3 or 4 toxicity, and 2 year survival of 80%. PFS was 17.7 months compared to historical control of 6.9 months. This is with 3 simple injections with no side effects. Yet we are being told it is many years before patients can get access.
    Another example: results of a large study of the Novo TTF device – in 237 patients randomized to the device alone or best of standard care, showed a slight edge in survival, but drastically lower side effects. It was approved in Europe, but USA patients can not get access..
    How do we help speed these things up?

  3. MLM Leads
    July 26, 2010 at 3:13 am | #3

    I just watched the video and how it captured my heart to know how kind is FDA Basics to help save kids. Keep it up.

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  4. August 11, 2011 at 3:35 pm | #6

    A friend of mine has a rare decease, and it’s really frustrating to see what trouble she has to overcome to get her medicine. Perhaps the government should spend more on facilitating medication.

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  5. August 19, 2011 at 3:15 am | #9

    Anytime someone is involved on this level it gives us all hope.

  6. Katy Perry Cupcake Costume
    September 26, 2011 at 9:51 pm | #10

    Rare diseases!? Why don’t you invite Dr. House… :) I really liked watching Dr. Cote discussing how FDA’s Orphan Drugs program is helping in the fight against rare diseases.

  7. Panoraview
    October 1, 2011 at 4:37 pm | #11

    While there are people like these, there is hope.

  8. October 12, 2011 at 3:51 pm | #12

    Great interview well answers by doctor.

  9. October 12, 2011 at 10:12 pm | #13

    Dr Cote is a respected leader in his field, I hope this progresses as fast as it can.

  10. October 24, 2011 at 10:46 am | #14

    very good article as I am in Spain medical support will further investigate these rare diseases to fight them because even if the cases are few, if not ill will continue to investigate the emerging

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    February 27, 2012 at 4:41 pm | #19

    We must support the research of diseases, even qeule diseases affect a minimum percentage of the population, for individuals and families is very hard.

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    January 10, 2013 at 5:24 pm | #26

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