To: FDADockets@OHRM-MAIL@FDAOC From: "Robert Thompson" Certify: N Subject: FDAMA Docket #98-339D Date: Thursday, September 10, 1998 at 8:05:40 am EDT Attached: None Sirs, The FDA Modernization Act is critical for those of us who are suffering with terminal diseases, such as ALS - we need speedy access to safe and effective treatments for this deadly disease. The long process of various clinical trials and then evaluation by the FDA creates an untenable situation in which many who could be helped slip further into the grasp of this disease. Perhaps this is not so critical with many other diseases but when you "Know" you are going to die within 2-5 years the FDA process only makes it harder to handle. This is especially true when there are drugs that are safe, shown to be effective in clinical trials, but approval is not forthcoming - drugs like myotrophin. In case you haven't noticed, we will die without the drug - more people have died from Viagra than from myotrophin, we need access and we need it now! On the 17th of August, Dr. Hiroshi Mitsumoto of the Cleveland Clinic Foundation made some very important comments at the CDER Stakeholder's Meeting, please take these comments into serious consideration: *Because ALS is the most devastating neurological disease there should be no higher priority for all FDA centers especially CDER and CBER in expediting development of review of drugs for treating serious and rapidly fatal diseases such as ALS. *Equal benefit for ALL serious and life-threatening diseases from accelerated approval. *For truly life threatening diseases, such as ALS, the FDA can expedite availability of therapies by providing greater authority to approve drugs that strongly suggest effectiveness, as stated by law *The FDA can permit greater use of Phase IV post approval confirmatory trials, to make these therapies more accessible to those in great need and provide better evidence to substantiate effectiveness. *For diseases that have no surrogate markers, such as ALS, but is relentlessly progressive and always fatal a need for placebo controls in Phase III trials should be reassessed. *The FDA should consider efficacy relative to safety. Large exposure to a drug that has shown minimal side effects should weigh heavily even if there is only a small benefit, particularly in diseases such as ALS where long-term exposure is most likely not an issue. *Given the great deference the FDA places on the Advisory Panel decision, it is absolutely critical that true experts of the actual disease be represented on these panels. My life and the lifes of thousands of ALS patients are literally in your hands, don't destroy our hope by denying us access to the drugs that can offer us hope and help. Respectfully, Robert J. Thompson Diagnosed w/ALS 4 Jun 98 ______________________________________________________ Get Your Private, Free Email at http://www.hotmail.com