Orphan drug regulations

The final option is the Centralized Procedure. This is available for a defined list of product types (included in an Annex to the relevant Regulation) and for Orphan Drugs as defined by the Orphan Drug Committee. This procedure involves the appointment of... 

Anon. (1992h). Orphan Drug Regulations 21 CFR PART 316. http //www.fda.gov/orphan/about/odreg.htm... 

Orphan Nuclear Receptor PXR-Mediated Gene Regulation in Drug Metabolism and Endobiotic Homeostasis... 

Xie, W., Uppal, H., Saini, S.P., Mu, Y., Little, J.M., Radominska-Pandya, A. and Zemaitis, M.A. (2004) Orphan nuclear receptor-mediated xenobiotic regulation in drug metabolism. Drug Discovery Today, 9, 442-449. 

The success of the various special-case areas of drug development regulation and performance, such as orphan drugs, cancer, and AIDS drugs, and the pediatric exclusivity extension could be a guide to how to approach - and what to avoid -in creating larger facilitatory approaches in the future. 

Lasagna L. Who will adopt the orphan drug Regulation 1979 3 27-32. 

The expected private benefit depends on a number of market characteristics and public policies, including market size, intellectual property rights, price regulation, and the magnitude of prizes for innovation. Some public policies, such as the Orphan Drug Act, may affect both the costs and the expected private benefits of R D investment. 

CUssold, D. B. 1995. Prescription for The Orphan Drug-Act The Impact of the FDA s 1992 Regulations and the Latest Congressional Proposals for Reform. Food and Drug Law Journal 50(1) 125-147. 

The orphan drug regulations allow a sponsor of an orphan-designated drug a period of marketing exclusivity, free from Continued on next page)... 

G. Other considerations Serono conducted the comparative study to demonstrate that Rebif is clinically superior to Avonex. No other path was available for Serono to have the product licensed by the FDA before mid-2003 because Avonex carried an orphan drug designation based on its superior safety profile over Betaseron, the first interferon beta to be licensed for multiple sclerosis. Based on the results of the Rebif versus Avonex study, the FDA determined that Rebif is clinically superior to Avonex. While recognizing that the safety profile associated with Rebif is not as favorable as the safety profile of Avonex, the FDA has determined that the severity and frequency of such adverse events do not render Rebif unUcensable under Section 35 of the Public Health Service Act. Further, under the orphan drug regulations, if Serono demonstrates that Rebif is superior to Avonex based on efficacy, Serono does not have to show that Rebif is safer than, or as safe as, Avonex. 

RIN 0905-AB55 Orphan-Drug Regulations Agency Food and Drug Administration, HHS Action Final Rule... 

Wang H, LeCluyse EL. Role of orphan nuclear receptors in the regulation of drug-metabolising enzymes. Clin Pharmacokinet 2003 42 1331-1357. 

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