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Biotech / Medical : Stressgen (VSE: SSB) -- Ignore unavailable to you. Want to Upgrade?

To: Heat Shock who wrote (159)	4/4/2001 5:50:40 PM
From: Heat Shock	Read Replies (1) \| Respond to of 236

To all: good post on orphan drug status by beararn over on stockhouse.ca: Orphan Drug Status is extremely positive....faster to market, permits off-label use, FDA is partner, marketing application can go in anytime and drug can be sold in parallel with phase 3 (if marketing approved), partners more interested, Many countries have ODS and approval process is very quick when ODS achieved in US (ie. faster to market internationally). Some basic questions about orphan drug designation are answered at the following FDA site. It would appear that the trial process is substantially shortened. It would also appear that the financial incentives are not primarily in the grants, as that is not at a high level. It also confirms that while the trial process must continue, the drug can be made available to the public in a couple of ways...here it mentions compassionate basis, and in the regs sited earlier it mentions open protocols (same thing). fda.gov Are orphan products approved faster than other drugs? Historically the approval time for orphan products as a group has been considerably shorter than the approval time for all drugs. This is due to the fact that many orphan products receive expedited review because they are for serious or life-threatening disease. How much money is available for grants? The current annual budget for funding grants is approximately $12 million. Clinical trials are awarded grants from $100,000 to $200,000 per year in direct costs for up to 3 years. The annual RFA will provide more up-to-date information on dollar limits What is the process to obtain a particular orphan drug? If an orphan product has been approved for marketing, it will be available through the normal pharmaceutical supply channels. If the product has not been approved, the sponsor may make the product available on a compassionate use basis. For contact information on sponsors of orphan products, contact the Office of Orphan Products Development. The following FDA site lists orphan drugs approved for marketing. fda.gov the following site lists cancer drug approvals by FDA, done year by year. It is a valuable way to keep track of the competition, gold standards, etc. fda.gov Tons of info here, but here is the first paragraph..lots of interesting nuggets to follow up fda.gov ORPHAN DRUG REGULATIONS AGENCY: Food and Drug Administration, HHS. ACTION: Final rule. SUMMARY: The Food and Drug Administration (FDA) is issuing final regulations to implement section 2 of the Orphan Drug Act, which consists of four sections added to the Federal Food, Drug, and Cosmetic Act (the act). In the FEDERAL REGISTER of January 29, 1991 (56 FR3338), the agency proposed regulations to implement this section of the Orphan Drug Act. The Orphan Drug Act directs FDA to provide written recommendations on studies required for approval of a marketing application for an orphan drug. It also provides for the designation of drugs, including antibiotics and biological products, as orphan drugs when certain conditions are met, and it provides conditions under which a sponsor of an approved orphan drug enjoys exclusive approval for that drug for the orphan indication for 7 years following the date of the drug's marketing approval. Finally, section 2 of the Orphan Drug Act encourages sponsors to make orphan drugs available for treatment on an "open protocol" basis before the drug has been approved for general marketing. This action will benefit consumers by encouraging manufacturers to develop and make available to patients drugs for diseases and conditions that are rare in the United State