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Executive Summary

GPIA FUNDING ORPHAN DISEASE DATA BASE WITH $25,000 PER YEAR over a three year period, Abbey Meyers of the Tourettes Syndrome Assn., reported at a Sept. 5 meeting of the Pharmaceutical Mfrs. Assn. Commission on Drugs for Rare Diseases. Meyers, who will become exec director of the Natl. Organization for Rare Disorders in December, noted that the data base currently includes information on 190 rare diseases. She said completion of the program within three years is contingent on obtaining adequate funding beyond that provided by the Generic Pharmaceutical Industry Assn. When it is completed, the data base is expected to include data on 5,000-6,000 diseases. The orphan disease data base is accessible through CompuServe under the Health-Com section. Each entry for a disease includes synonyms for and a general description of the disease, symptomatology, etiology, affected population, standard and investigational treatments, and available resources, including clinics and voluntary assns. Meyers said an average of 800 people per month now access the data base. It became operational during the summer. FDAer Steve Groft, PharmD, noted that additional resource material on orphan diseases is available through the Natl. Information Center for Orphan Drugs and Rare Diseases. The center has prepared a resource of educational materials entitled Rare Diseases: A Resource Directory. The publication, which is now in draft form, contains the history and purpose of specific rare disease organizations and lists the printed and audiovisual material available through the various private sector and govt. centers. FDA Acting Director of the Office of Orphan Products Stephen Fredd, MD, noted that 52.3 mil. has been allocated to FDA for its orphan grants program for fiscal year 1985. He said the agency expects to award 21 grants. Fredd also noted that FDA anticipates awarding a contract by Sept. 30 for a literature search on neonatal drugs. He said FDA will examine the labeling for dopamine, theophylline, nitroprusside, phenobarbitol, calcium chloride, ampicillin, and caffeine to determine if relabeling is necessary with respect to indications for neonates. In an update on proposals submitted to PMA for orphan sponsorship, PMA Commission Exec Director John Adams noted that a company had just agreed to support animal studies on the potential efficacy of tizanidine in the treatment of trigeminal neuralgia. With regard to a proposal submitted on the use of tetrathiomolydbate in Wilson's Disease, the Commission agreed to try to find a sponsor despite the fact the proposal is in the early stages and toxicology tests have not yet been conducted. Adams said a proposal for gamma hydroxybutyrate, for use in diseases of the central nervous system, has been funded, and a sponsor is still being sought for sulfur hexafluoride, a gas used for retinal detachments.

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