Comments on: Numbers Don’t Add Up – Only 347 New Drugs Approved By FDA For 30 Million Americans With Rare Disease in Past 25 Years? http://addiandcassi.com/numbers-dont-add-up-only-344-new-drugs-approved-by-fda-for-30-million-americans-with-rare-disease-in-past-25-years/ Treatments & Cures for Niemann Pick Type C - Childhood Alzheimer's Wed, 28 Jul 2010 20:36:32 +0000 http://wordpress.org/?v=2.9.1 hourly 1 By: Robert Rooney http://addiandcassi.com/numbers-dont-add-up-only-344-new-drugs-approved-by-fda-for-30-million-americans-with-rare-disease-in-past-25-years/comment-page-1/#comment-2439 Robert Rooney Mon, 15 Feb 2010 16:42:51 +0000 http://addiandcassi.com/wordpress/?p=2545#comment-2439 Really interesting piece.. And while I can appreciate your frustration I would have to clarify that beneath the surface of the 347 Approved Orphan Treatments by the FDA there have been over 2000 Orphan Designations which refers to the number of treatments in the pipeline for treating rare diseases which have been reviewed by the FDA for potential Market Authorization. This represents the promise and level of activity into the development of future orphan treatments, not to mention the treatments which are undergoing research and have not yet been submitted for orphan designation. Therefore one would have to say that while the point remains true that we are a long way from having final treatments for the 7k-8k rare diseases - the Orphan Drug act has in fact been a success in redressing the gap when one takes into account the huge factors involved in stimulating the drug development process for an often small cohort of patients. The bottlenecks are still in the usual places: Getting sufficient numbers of patients into Clinical Trials to move the drug development phase forward, finding the funding to move research beyond the pre-clinical phase and sufficiently stimulating and supporting the Biotech industry so that they have the necessary experience and motivation to deliver quality dossiers and safe treatments for review. Really interesting piece.. And while I can appreciate your frustration I would have to clarify that beneath the surface of the 347 Approved Orphan Treatments by the FDA there have been over 2000 Orphan Designations which refers to the number of treatments in the pipeline for treating rare diseases which have been reviewed by the FDA for potential Market Authorization. This represents the promise and level of activity into the development of future orphan treatments, not to mention the treatments which are undergoing research and have not yet been submitted for orphan designation. Therefore one would have to say that while the point remains true that we are a long way from having final treatments for the 7k-8k rare diseases – the Orphan Drug act has in fact been a success in redressing the gap when one takes into account the huge factors involved in stimulating the drug development process for an often small cohort of patients.

The bottlenecks are still in the usual places: Getting sufficient numbers of patients into Clinical Trials to move the drug development phase forward, finding the funding to move research beyond the pre-clinical phase and sufficiently stimulating and supporting the Biotech industry so that they have the necessary experience and motivation to deliver quality dossiers and safe treatments for review.

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