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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.
I think it is a collective effort in fighting this.
A campaign spending time with to save the kids future.
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?
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.
This web site has received lots of extremely helpful details on it. Thanks for informing me!
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.
Anytime someone is involved on this level it gives us all hope.
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.
While there are people like these, there is hope.
Great interview well answers by doctor.
Dr Cote is a respected leader in his field, I hope this progresses as fast as it can.
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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This is nice interview and people can get more information from this.
The most frequent mistake is to set up your organization on a individual profile and you see this a good deal on Facebook. It’s simple to spot since you have friends as an alternative of ‘likes’. Personal profiles are for people and they have pals, pages are for corporations which people can then like.
We must support the research of diseases, even qeule diseases affect a minimum percentage of the population, for individuals and families is very hard.
Very good article and a worth while cause.
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It’s simple to spot since you have friends as an alternative of ‘likes’. Does one offer guest writers to write content for yourself?
A great and well informed video.